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ASH 2024: New Leukemia Txs, Fewer Blood Clots With GLP-1 Rxs
Children’s Disorders: Major Progress in B-Cell Acute Lymphoblastic Leukemia (B-ALL), Immune Thrombocytopenic Purpura (ITP)
While B-ALL is the most common childhood cancer and one of the most treatable, some patients face grim outcomes after they relapse following chemotherapy, said Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute.
A new study reports that adding the targeted cancer drug blinatumomab (Blincyto) to chemotherapy boosted disease-free survival in standard-risk pediatric patients. “They definitively demonstrate a benefit with the addition of this immunotherapeutic drug, achieving 97% disease-free survival at 3 years on the blinatumomab plus chemotherapy arm compared to 90% for the control arm with standard therapies alone,” Dunbar said. “This trial will establish the addition of blinatumomab for childhood B-ALL as standard of care.”
A reporter asked Dunbar about the risk for severe immune activation syndrome. “These immune cell engagers can result in cytokine release syndrome and other severe immune activation consequences,” she said. “However, it appears that children seem to be less susceptible to those, at least in terms of severity, than adults. In this study, the complications that occurred didn’t result in mortality and were easily treatable. So that was not a major drawback to the addition of this drug.”
The blinatumomab study is sponsored by Children’s Oncology Group.
In ITP, thrombopoietin (TPO) agonists such as eltrombopag (Promacta) are a mainstay of second- or third-line treatment in children and adults with severe cases, Dunbar said. “However, TPO agonists are generally only given after months to years of failures of corticosteroids, IVIG [intravenous immunoglobulin], or splenectomy.”
In the phase 3, randomized, controlled PINES trial, researchers explored whether the drug could improve outcomes in children with untreated or very recent-onset severe ITP vs standard of care.
“The children treated with eltrombopag had double the response rate with a much lower need for rescue therapies,” Dunbar said. The percentage of patients who received rescue therapy was 19% in the eltrombopag arm (15/78) vs 46% in the control arm (18/39, P = .002).
“Given the potential short- and long-term consequences of corticosteroids and other standard treatments in children, this study is encouraging and will likely result in a change in the standard of care for pediatric ITP,” Dunbar said.
The eltrombopag study is sponsored by the ITP Consortium of North America and funded by Novartis.
Fewer Blood Clots: Another Big Benefit for Weight Loss Drugs?
Mikkael A. Sekeres, MD, MS, of the Sylvester Comprehensive Cancer Center at the University of Miami in Florida, highlighted an analysis of whether GLP-1 receptor agonists, initially approved as type 2 diabetes treatments, affect the risk for VTE.
Researchers tracked patients with type 2 diabetes — 366,369 who received the drugs and 290,219 who took dipeptidyl peptidase 4 inhibitors. The patients who took GLP-1 agonists “had lower rates of venous thromboembolic events after 1 year,” Sekeres said. “The risk reduction was actually pretty substantial.”
In these patients, the risk for VTE was 18% lower (hazard ratio [HR], 0.82; 95% CI, 0.77-0.88), and there were 22% and 15% reductions in pulmonary embolisms and deep venous thrombosis, respectively (HR, 0.78; 95% CI, 0.71-0.86 and HR, 0.85; 95% CI, 0.79-0.92).
Drug Regimen Improves Outcomes in Chronic Lymphocytic Leukemia (CLL)
An interim analysis of an open-label, randomized, phase 3 trial of patients with untreated CLL “demonstrated superior progression-free survival with acalabrutinib and venetoclax versus what we consider more classic chemotherapy of fludarabine, Cytoxan [cyclophosphamide], and rituximab or bendamustine and rituximab,” Sekeres said. “Similar findings were seen with acalabrutinib, venetoclax, and obinutuzumab vs that classic chemotherapy.”
Overall response rates were 93% for both the acalabrutinib/venetoclax regimens vs 75% for bendamustine/rituximab, Sekeres noted, and overall survival was higher for acalabrutinib/venetoclax vs the two classic chemotherapy regimens (HR, 0.33; P < .0001).
However, Sekeres questioned the value of comparing acalabrutinib/venetoclax with classical chemotherapy regimens. “A lot of times we have a lot of new, really good, really effective therapy to offer to patients that isn’t as toxic as previous chemotherapy.”
In contrast, fludarabine, cyclophosphamide, and rituximab are “your grandmother’s or your grandfather’s chemotherapy. It’s pretty toxic stuff,” he said.
Sekeres said it would have been better to compare acalabrutinib/venetoclax with a Bruton tyrosine kinase inhibitor–based regimen.
The German CLL Study Group is listed as the trial’s sponsor, and AstraZeneca is a collaborator. Dunbar disclosed research funding from Novartis. Sekeres had no relevant disclosures.
A version of this article appeared on Medscape.com.
Children’s Disorders: Major Progress in B-Cell Acute Lymphoblastic Leukemia (B-ALL), Immune Thrombocytopenic Purpura (ITP)
While B-ALL is the most common childhood cancer and one of the most treatable, some patients face grim outcomes after they relapse following chemotherapy, said Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute.
A new study reports that adding the targeted cancer drug blinatumomab (Blincyto) to chemotherapy boosted disease-free survival in standard-risk pediatric patients. “They definitively demonstrate a benefit with the addition of this immunotherapeutic drug, achieving 97% disease-free survival at 3 years on the blinatumomab plus chemotherapy arm compared to 90% for the control arm with standard therapies alone,” Dunbar said. “This trial will establish the addition of blinatumomab for childhood B-ALL as standard of care.”
A reporter asked Dunbar about the risk for severe immune activation syndrome. “These immune cell engagers can result in cytokine release syndrome and other severe immune activation consequences,” she said. “However, it appears that children seem to be less susceptible to those, at least in terms of severity, than adults. In this study, the complications that occurred didn’t result in mortality and were easily treatable. So that was not a major drawback to the addition of this drug.”
The blinatumomab study is sponsored by Children’s Oncology Group.
In ITP, thrombopoietin (TPO) agonists such as eltrombopag (Promacta) are a mainstay of second- or third-line treatment in children and adults with severe cases, Dunbar said. “However, TPO agonists are generally only given after months to years of failures of corticosteroids, IVIG [intravenous immunoglobulin], or splenectomy.”
In the phase 3, randomized, controlled PINES trial, researchers explored whether the drug could improve outcomes in children with untreated or very recent-onset severe ITP vs standard of care.
“The children treated with eltrombopag had double the response rate with a much lower need for rescue therapies,” Dunbar said. The percentage of patients who received rescue therapy was 19% in the eltrombopag arm (15/78) vs 46% in the control arm (18/39, P = .002).
“Given the potential short- and long-term consequences of corticosteroids and other standard treatments in children, this study is encouraging and will likely result in a change in the standard of care for pediatric ITP,” Dunbar said.
The eltrombopag study is sponsored by the ITP Consortium of North America and funded by Novartis.
Fewer Blood Clots: Another Big Benefit for Weight Loss Drugs?
Mikkael A. Sekeres, MD, MS, of the Sylvester Comprehensive Cancer Center at the University of Miami in Florida, highlighted an analysis of whether GLP-1 receptor agonists, initially approved as type 2 diabetes treatments, affect the risk for VTE.
Researchers tracked patients with type 2 diabetes — 366,369 who received the drugs and 290,219 who took dipeptidyl peptidase 4 inhibitors. The patients who took GLP-1 agonists “had lower rates of venous thromboembolic events after 1 year,” Sekeres said. “The risk reduction was actually pretty substantial.”
In these patients, the risk for VTE was 18% lower (hazard ratio [HR], 0.82; 95% CI, 0.77-0.88), and there were 22% and 15% reductions in pulmonary embolisms and deep venous thrombosis, respectively (HR, 0.78; 95% CI, 0.71-0.86 and HR, 0.85; 95% CI, 0.79-0.92).
Drug Regimen Improves Outcomes in Chronic Lymphocytic Leukemia (CLL)
An interim analysis of an open-label, randomized, phase 3 trial of patients with untreated CLL “demonstrated superior progression-free survival with acalabrutinib and venetoclax versus what we consider more classic chemotherapy of fludarabine, Cytoxan [cyclophosphamide], and rituximab or bendamustine and rituximab,” Sekeres said. “Similar findings were seen with acalabrutinib, venetoclax, and obinutuzumab vs that classic chemotherapy.”
Overall response rates were 93% for both the acalabrutinib/venetoclax regimens vs 75% for bendamustine/rituximab, Sekeres noted, and overall survival was higher for acalabrutinib/venetoclax vs the two classic chemotherapy regimens (HR, 0.33; P < .0001).
However, Sekeres questioned the value of comparing acalabrutinib/venetoclax with classical chemotherapy regimens. “A lot of times we have a lot of new, really good, really effective therapy to offer to patients that isn’t as toxic as previous chemotherapy.”
In contrast, fludarabine, cyclophosphamide, and rituximab are “your grandmother’s or your grandfather’s chemotherapy. It’s pretty toxic stuff,” he said.
Sekeres said it would have been better to compare acalabrutinib/venetoclax with a Bruton tyrosine kinase inhibitor–based regimen.
The German CLL Study Group is listed as the trial’s sponsor, and AstraZeneca is a collaborator. Dunbar disclosed research funding from Novartis. Sekeres had no relevant disclosures.
A version of this article appeared on Medscape.com.
Children’s Disorders: Major Progress in B-Cell Acute Lymphoblastic Leukemia (B-ALL), Immune Thrombocytopenic Purpura (ITP)
While B-ALL is the most common childhood cancer and one of the most treatable, some patients face grim outcomes after they relapse following chemotherapy, said Cynthia E. Dunbar, MD, chief of the Translational Stem Cell Biology Branch at the National Heart, Lung, and Blood Institute.
A new study reports that adding the targeted cancer drug blinatumomab (Blincyto) to chemotherapy boosted disease-free survival in standard-risk pediatric patients. “They definitively demonstrate a benefit with the addition of this immunotherapeutic drug, achieving 97% disease-free survival at 3 years on the blinatumomab plus chemotherapy arm compared to 90% for the control arm with standard therapies alone,” Dunbar said. “This trial will establish the addition of blinatumomab for childhood B-ALL as standard of care.”
A reporter asked Dunbar about the risk for severe immune activation syndrome. “These immune cell engagers can result in cytokine release syndrome and other severe immune activation consequences,” she said. “However, it appears that children seem to be less susceptible to those, at least in terms of severity, than adults. In this study, the complications that occurred didn’t result in mortality and were easily treatable. So that was not a major drawback to the addition of this drug.”
The blinatumomab study is sponsored by Children’s Oncology Group.
In ITP, thrombopoietin (TPO) agonists such as eltrombopag (Promacta) are a mainstay of second- or third-line treatment in children and adults with severe cases, Dunbar said. “However, TPO agonists are generally only given after months to years of failures of corticosteroids, IVIG [intravenous immunoglobulin], or splenectomy.”
In the phase 3, randomized, controlled PINES trial, researchers explored whether the drug could improve outcomes in children with untreated or very recent-onset severe ITP vs standard of care.
“The children treated with eltrombopag had double the response rate with a much lower need for rescue therapies,” Dunbar said. The percentage of patients who received rescue therapy was 19% in the eltrombopag arm (15/78) vs 46% in the control arm (18/39, P = .002).
“Given the potential short- and long-term consequences of corticosteroids and other standard treatments in children, this study is encouraging and will likely result in a change in the standard of care for pediatric ITP,” Dunbar said.
The eltrombopag study is sponsored by the ITP Consortium of North America and funded by Novartis.
Fewer Blood Clots: Another Big Benefit for Weight Loss Drugs?
Mikkael A. Sekeres, MD, MS, of the Sylvester Comprehensive Cancer Center at the University of Miami in Florida, highlighted an analysis of whether GLP-1 receptor agonists, initially approved as type 2 diabetes treatments, affect the risk for VTE.
Researchers tracked patients with type 2 diabetes — 366,369 who received the drugs and 290,219 who took dipeptidyl peptidase 4 inhibitors. The patients who took GLP-1 agonists “had lower rates of venous thromboembolic events after 1 year,” Sekeres said. “The risk reduction was actually pretty substantial.”
In these patients, the risk for VTE was 18% lower (hazard ratio [HR], 0.82; 95% CI, 0.77-0.88), and there were 22% and 15% reductions in pulmonary embolisms and deep venous thrombosis, respectively (HR, 0.78; 95% CI, 0.71-0.86 and HR, 0.85; 95% CI, 0.79-0.92).
Drug Regimen Improves Outcomes in Chronic Lymphocytic Leukemia (CLL)
An interim analysis of an open-label, randomized, phase 3 trial of patients with untreated CLL “demonstrated superior progression-free survival with acalabrutinib and venetoclax versus what we consider more classic chemotherapy of fludarabine, Cytoxan [cyclophosphamide], and rituximab or bendamustine and rituximab,” Sekeres said. “Similar findings were seen with acalabrutinib, venetoclax, and obinutuzumab vs that classic chemotherapy.”
Overall response rates were 93% for both the acalabrutinib/venetoclax regimens vs 75% for bendamustine/rituximab, Sekeres noted, and overall survival was higher for acalabrutinib/venetoclax vs the two classic chemotherapy regimens (HR, 0.33; P < .0001).
However, Sekeres questioned the value of comparing acalabrutinib/venetoclax with classical chemotherapy regimens. “A lot of times we have a lot of new, really good, really effective therapy to offer to patients that isn’t as toxic as previous chemotherapy.”
In contrast, fludarabine, cyclophosphamide, and rituximab are “your grandmother’s or your grandfather’s chemotherapy. It’s pretty toxic stuff,” he said.
Sekeres said it would have been better to compare acalabrutinib/venetoclax with a Bruton tyrosine kinase inhibitor–based regimen.
The German CLL Study Group is listed as the trial’s sponsor, and AstraZeneca is a collaborator. Dunbar disclosed research funding from Novartis. Sekeres had no relevant disclosures.
A version of this article appeared on Medscape.com.
FROM ASH 2024
New Cancer Vaccines on the Horizon: Renewed Hope or Hype?
Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.
But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.
Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.
In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.
said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.
A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.
“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.
Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.
“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”
Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.
Then: Where We Were
Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.
In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.
Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”
In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”
Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.
In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.
In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.
That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.
In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.
And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.
But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.
When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.
But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.
Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.
“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”
A Turning Point?
Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.
Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.
Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.
Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.
Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.
“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.”
Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.
One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.
But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”
“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”
Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.
“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”
Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.
As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”
A version of this article first appeared on Medscape.com.
Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.
But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.
Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.
In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.
said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.
A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.
“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.
Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.
“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”
Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.
Then: Where We Were
Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.
In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.
Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”
In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”
Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.
In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.
In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.
That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.
In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.
And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.
But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.
When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.
But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.
Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.
“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”
A Turning Point?
Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.
Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.
Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.
Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.
Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.
“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.”
Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.
One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.
But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”
“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”
Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.
“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”
Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.
As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”
A version of this article first appeared on Medscape.com.
Vaccines for treating and preventing cancer have long been considered a holy grail in oncology.
But aside from a few notable exceptions — including the human papillomavirus (HPV) vaccine, which has dramatically reduced the incidence of HPV-related cancers, and a Bacillus Calmette-Guerin vaccine, which helps prevent early-stage bladder cancer recurrence — most have failed to deliver.
Following a string of disappointments over the past decade, recent advances in the immunotherapy space are bringing renewed hope for progress.
In an American Association for Cancer Research (AACR) series earlier in 2024, Catherine J. Wu, MD, predicted big strides for cancer vaccines, especially for personalized vaccines that target patient-specific neoantigens — the proteins that form on cancer cells — as well as vaccines that can treat diverse tumor types.
said Wu, the Lavine Family Chair of Preventative Cancer Therapies at Dana-Farber Cancer Institute and a professor of medicine at Harvard Medical School, both in Boston, Massachusetts.
A prime example is a personalized, messenger RNA (mRNA)–based vaccine designed to prevent melanoma recurrence. The mRNA-4157 vaccine encodes up to 34 different patient-specific neoantigens.
“This is one of the most exciting developments in modern cancer therapy,” said Lawrence Young, a virologist and professor of molecular oncology at the University of Warwick, Coventry, England, who commented on the investigational vaccine via the UK-based Science Media Centre.
Other promising options are on the horizon as well. In August, BioNTech announced a phase 1 global trial to study BNT116 — a vaccine to treat non–small cell lung cancer (NSCLC). BNT116, like mRNA-4157, targets specific antigens in the lung cancer cells.
“This technology is the next big phase of cancer treatment,” Siow Ming Lee, MD, a consultant medical oncologist at University College London Hospitals in England, which is leading the UK trial for the lung cancer and melanoma vaccines, told The Guardian. “We are now entering this very exciting new era of mRNA-based immunotherapy clinical trials to investigate the treatment of lung cancer.”
Still, these predictions have a familiar ring. While the prospects are exciting, delivering on them is another story. There are simply no guarantees these strategies will work as hoped.
Then: Where We Were
Cancer vaccine research began to ramp up in the 2000s, and in 2006, the first-generation HPV vaccine, Gardasil, was approved. Gardasil prevents infection from four strains of HPV that cause about 80% of cervical cancer cases.
In 2010, the Food and Drug Administration approved sipuleucel-T, the first therapeutic cancer vaccine, which improved overall survival in patients with hormone-refractory prostate cancer.
Researchers predicted this approval would “pave the way for developing innovative, next generation of vaccines with enhanced antitumor potency.”
In a 2015 AACR research forecast report, Drew Pardoll, MD, PhD, co-director of the Cancer Immunology and Hematopoiesis Program at Johns Hopkins University, Baltimore, Maryland, said that “we can expect to see encouraging results from studies using cancer vaccines.”
Despite the excitement surrounding cancer vaccines alongside a few successes, the next decade brought a longer string of late-phase disappointments.
In 2016, the phase 3 ACT IV trial of a therapeutic vaccine to treat glioblastoma multiforme (CDX-110) was terminated after it failed to demonstrate improved survival.
In 2017, a phase 3 trial of the therapeutic pancreatic cancer vaccine, GVAX, was stopped early for lack of efficacy.
That year, an attenuated Listeria monocytogenes vaccine to treat pancreatic cancer and mesothelioma also failed to come to fruition. In late 2017, concerns over listeria infections prompted Aduro Biotech to cancel its listeria-based cancer treatment program.
In 2018, a phase 3 trial of belagenpumatucel-L, a therapeutic NSCLC vaccine, failed to demonstrate a significant improvement in survival and further study was discontinued.
And in 2019, a vaccine targeting MAGE-A3, a cancer-testis antigen present in multiple tumor types, failed to meet endpoints for improved survival in a phase 3 trial, leading to discontinuation of the vaccine program.
But these disappointments and failures are normal parts of medical research and drug development and have allowed for incremental advances that helped fuel renewed interest and hope for cancer vaccines, when the timing was right, explained vaccine pioneer Larry W. Kwak, MD, PhD, deputy director of the Comprehensive Cancer Center at City of Hope, Duarte, California.
When it comes to vaccine progress, timing makes a difference. In 2011, Kwak and colleagues published promising phase 3 trial results on a personalized vaccine. The vaccine was a patient-specific tumor-derived antigen for patients with follicular lymphoma in their first remission following chemotherapy. Patients who received the vaccine demonstrated significantly longer disease-free survival.
But, at the time, personalized vaccines faced strong headwinds due, largely, to high costs, and commercial interest failed to materialize. “That’s been the major hurdle for a long time,” said Kwak.
Now, however, interest has returned alongside advances in technology and research. The big shift has been the emergence of lower-cost rapid-production mRNA and DNA platforms and a better understanding of how vaccines and potent immune stimulants, like checkpoint inhibitors, can work together to improve outcomes, he explained.
“The timing wasn’t right” back then, Kwak noted. “Now, it’s a different environment and a different time.”
A Turning Point?
Indeed, a decade later, cancer vaccine development appears to be headed in a more promising direction.
Among key cancer vaccines to watch is the mRNA-4157 vaccine, developed by Merck and Moderna, designed to prevent melanoma recurrence. In a recent phase 2 study, patients receiving the mRNA-4157 vaccine alongside pembrolizumab had nearly half the risk for melanoma recurrence or death at 3 years compared with those receiving pembrolizumab alone. Investigators are now evaluating the vaccine in a global phase 3 study in patients with high-risk, stage IIB to IV melanoma following surgery.
Another one to watch is the BNT116 NSCLC vaccine from BioNTech. This vaccine presents the immune system with NSCLC tumor markers to encourage the body to fight cancer cells expressing those markers while ignoring healthy cells. BioNTech also launched a global clinical trial for its vaccine this year.
Other notables include a pancreatic cancer mRNA vaccine, which has shown promising early results in a small trial of 16 patients. Of 16 patients who received the vaccine alongside chemotherapy and after surgery and immunotherapy, 8 responded. Of these eight, six remained recurrence free at 3 years. Investigators noted that the vaccine appeared to stimulate a durable T-cell response in patients who responded.
Kwak has also continued his work on lymphoma vaccines. In August, his team published promising first-in-human data on the use of personalized neoantigen vaccines as an early intervention in untreated patients with lymphoplasmacytic lymphoma. Among nine asymptomatic patients who received the vaccine, all achieved stable disease or better, with no dose-limiting toxicities. One patient had a minor response, and the median time to progression was greater than 72 months.
“The current setting is more for advanced disease,” Kwak explained. “It’s a tougher task, but combined with checkpoint blockade, it may be potent enough to work.”
Still, caution is important. Despite early promise, it’s too soon to tell which, if any, of these investigational vaccines will pan out in the long run. Like investigational drugs, cancer vaccines may show big promising initially but then fail in larger trials.
One key to success, according to Kwak, is to design trials so that even negative results will inform next steps.
But, he noted, failures in large clinical trials will “put a chilling effect on cancer vaccine research again.”
“That’s what keeps me up at night,” he said. “We know the science is fundamentally sound and we have seen glimpses over decades of research that cancer vaccines can work, so it’s really just a matter of tweaking things to optimize trial design.”
Companies tend to design trials to test if a vaccine works or not, without trying to understand why, he said.
“What we need to do is design those so that we can learn from negative results,” he said. That’s what he and his colleagues attempted to do in their recent trial. “We didn’t just look at clinical results; we’re interrogating the actual tumor environment to understand what worked and didn’t and how to tweak that for the next trial.”
Kwak and his colleagues found, for instance, that the vaccine had a greater effect on B cell–derived tumor cells than on cells of plasma origin, so “the most rational design for the next iteration is to combine the vaccine with agents that work directly against plasma cells,” he explained.
As for what’s next, Kwak said: “We’re just focused on trying to do good science and understand. We’ve seen glimpses of success. That’s where we are.”
A version of this article first appeared on Medscape.com.
Watch That Attitude: Is There Ageism in Healthcare?
People are living longer in Europe. Life expectancy increased on the continent by around 12 years between 1960 and 2022. And despite slower progress during the COVID-19 pandemic, the trend appears to be continuing.
Not only are Europeans living longer, their fertility rates are declining. This means that the number of people aged 75-84 years is projected to grow in Europe a full 56.1% by 2050, while the population younger than 55 years is expected to fall by 13.5%.
This means that attitudes toward age need to change, and fast — even among healthcare professionals.
Healthcare Is Not Exempt From Ageist Attitudes
A systematic review published in the journal PLOS ONE in 2020 found that age was a determinant factor in dictating who received certain medical procedures or treatments. For example, a study of 9105 hospitalized patients found that healthcare providers were significantly more likely to withhold life-sustaining treatments from older patients. Another study found evidence that older people are excluded from clinical trials, even when the trials are for diseases that appear later in life, like Parkinson’s.
“In healthcare, there are different levels of ageism,” explained Hannah Swift, PhD, reader in social and organizational psychology at the University of Kent in the United Kingdom.
Ageism is embedded in the laws, rules, and practices of institutions, she explained. This became especially obvious during the pandemic, when health professionals had to decide who to treat, possibly using age as a proxy for making some of these decisions, she said.
“When you categorize people, you might be using stereotypes, assumptions, and expectations about age and that age group to make those decisions, and that’s where errors can occur.”
She added that ageist attitudes also become apparent at the interpersonal level by using patronizing language or offering unnecessary help to older people based on assumptions about their cognitive and physical abilities.
“Older age is often wrongly associated with declining levels of health and activity,” said Ittay Mannheim, PhD, guest postdoctoral researcher on aging and ageism at the Open University of the Netherlands. “However, older adults are a very diverse group, varying widely in many aspects, including health conditions. This stereotype can influence how healthcare professionals interact with them, assuming frailty or memory issues simply based on age. It’s important to recognize that being older doesn’t necessarily mean being ill.”
Mannheim’s research found that healthcare professionals often stand in the way of older people using technology-based treatments due to negative attitudes towards age. “So, actually, a barrier to using these technologies could be that healthcare professionals don’t think that someone can use it or won’t even offer it because someone looks old or is old,” he said.
The Impacts
Discrimination impacts the physical, mental, and social well-being of its victims. This includes attitudes towards age.
The PLOS ONE review of research on the global reach of ageism found that experienced or self-determined ageism was associated with significantly worse health outcomes across all countries examined. The same research team calculated that an estimated 6.3 million cases of depression worldwide are linked to ageism.
Other research has found that exposure to negative age stereotyping impacts willingness to adopt a healthy lifestyle in addition to increasing the risk for cardiovascular events.
What Can Be Done?
“Healthcare professionals frequently interact with older adults at their most vulnerable, which can reinforce negative stereotypes of older people being vulnerable or ill,” said Swift. “However, not all older adults fit these stereotypes. Many can live well and independently. Perhaps healthcare education should include reminders of the diverse experiences of older individuals rather than solely focusing on the moments when they require help.”
Research indicates that although progress has been made in geriatric training and the care of older individuals by healthcare education institutions, improved education and training are still needed at all levels of geriatric healthcare, including hospital administrators, physicians, nurses, personal caregivers, and associated health professions.
“Generally speaking, what healthcare professionals learn about aging tends to focus more on the biological aspects,” said Mannheim. “However, they may not fully understand what it means to be old or how to interact with older individuals, especially regarding technology. It is important to raise awareness about ageism because, in my experience working with healthcare professionals, even a single workshop on ageism can have a profound impact. Participants often respond with surprise, saying something like, ‘Wow, I never thought about this before.’”
Mannheim said that training healthcare providers to understand the aging process better could help to reduce any biases they might have and better prepare them to respond more adequately to the needs of older patients.
“We cannot devalue the lives of older people simply because they are older. It is crucial for all of us, especially governments, to acknowledge our responsibility to protect and promote human rights for individuals of all ages. If we fail to do this, the strategies we’ve witnessed during this pandemic will be repeated in the future,” said Nena Georgantzi, PhD, Barcelona-based human rights manager at AGE Platform Europe, an EU network of organizations of and for older people.
A version of this article appeared on Medscape.com.
People are living longer in Europe. Life expectancy increased on the continent by around 12 years between 1960 and 2022. And despite slower progress during the COVID-19 pandemic, the trend appears to be continuing.
Not only are Europeans living longer, their fertility rates are declining. This means that the number of people aged 75-84 years is projected to grow in Europe a full 56.1% by 2050, while the population younger than 55 years is expected to fall by 13.5%.
This means that attitudes toward age need to change, and fast — even among healthcare professionals.
Healthcare Is Not Exempt From Ageist Attitudes
A systematic review published in the journal PLOS ONE in 2020 found that age was a determinant factor in dictating who received certain medical procedures or treatments. For example, a study of 9105 hospitalized patients found that healthcare providers were significantly more likely to withhold life-sustaining treatments from older patients. Another study found evidence that older people are excluded from clinical trials, even when the trials are for diseases that appear later in life, like Parkinson’s.
“In healthcare, there are different levels of ageism,” explained Hannah Swift, PhD, reader in social and organizational psychology at the University of Kent in the United Kingdom.
Ageism is embedded in the laws, rules, and practices of institutions, she explained. This became especially obvious during the pandemic, when health professionals had to decide who to treat, possibly using age as a proxy for making some of these decisions, she said.
“When you categorize people, you might be using stereotypes, assumptions, and expectations about age and that age group to make those decisions, and that’s where errors can occur.”
She added that ageist attitudes also become apparent at the interpersonal level by using patronizing language or offering unnecessary help to older people based on assumptions about their cognitive and physical abilities.
“Older age is often wrongly associated with declining levels of health and activity,” said Ittay Mannheim, PhD, guest postdoctoral researcher on aging and ageism at the Open University of the Netherlands. “However, older adults are a very diverse group, varying widely in many aspects, including health conditions. This stereotype can influence how healthcare professionals interact with them, assuming frailty or memory issues simply based on age. It’s important to recognize that being older doesn’t necessarily mean being ill.”
Mannheim’s research found that healthcare professionals often stand in the way of older people using technology-based treatments due to negative attitudes towards age. “So, actually, a barrier to using these technologies could be that healthcare professionals don’t think that someone can use it or won’t even offer it because someone looks old or is old,” he said.
The Impacts
Discrimination impacts the physical, mental, and social well-being of its victims. This includes attitudes towards age.
The PLOS ONE review of research on the global reach of ageism found that experienced or self-determined ageism was associated with significantly worse health outcomes across all countries examined. The same research team calculated that an estimated 6.3 million cases of depression worldwide are linked to ageism.
Other research has found that exposure to negative age stereotyping impacts willingness to adopt a healthy lifestyle in addition to increasing the risk for cardiovascular events.
What Can Be Done?
“Healthcare professionals frequently interact with older adults at their most vulnerable, which can reinforce negative stereotypes of older people being vulnerable or ill,” said Swift. “However, not all older adults fit these stereotypes. Many can live well and independently. Perhaps healthcare education should include reminders of the diverse experiences of older individuals rather than solely focusing on the moments when they require help.”
Research indicates that although progress has been made in geriatric training and the care of older individuals by healthcare education institutions, improved education and training are still needed at all levels of geriatric healthcare, including hospital administrators, physicians, nurses, personal caregivers, and associated health professions.
“Generally speaking, what healthcare professionals learn about aging tends to focus more on the biological aspects,” said Mannheim. “However, they may not fully understand what it means to be old or how to interact with older individuals, especially regarding technology. It is important to raise awareness about ageism because, in my experience working with healthcare professionals, even a single workshop on ageism can have a profound impact. Participants often respond with surprise, saying something like, ‘Wow, I never thought about this before.’”
Mannheim said that training healthcare providers to understand the aging process better could help to reduce any biases they might have and better prepare them to respond more adequately to the needs of older patients.
“We cannot devalue the lives of older people simply because they are older. It is crucial for all of us, especially governments, to acknowledge our responsibility to protect and promote human rights for individuals of all ages. If we fail to do this, the strategies we’ve witnessed during this pandemic will be repeated in the future,” said Nena Georgantzi, PhD, Barcelona-based human rights manager at AGE Platform Europe, an EU network of organizations of and for older people.
A version of this article appeared on Medscape.com.
People are living longer in Europe. Life expectancy increased on the continent by around 12 years between 1960 and 2022. And despite slower progress during the COVID-19 pandemic, the trend appears to be continuing.
Not only are Europeans living longer, their fertility rates are declining. This means that the number of people aged 75-84 years is projected to grow in Europe a full 56.1% by 2050, while the population younger than 55 years is expected to fall by 13.5%.
This means that attitudes toward age need to change, and fast — even among healthcare professionals.
Healthcare Is Not Exempt From Ageist Attitudes
A systematic review published in the journal PLOS ONE in 2020 found that age was a determinant factor in dictating who received certain medical procedures or treatments. For example, a study of 9105 hospitalized patients found that healthcare providers were significantly more likely to withhold life-sustaining treatments from older patients. Another study found evidence that older people are excluded from clinical trials, even when the trials are for diseases that appear later in life, like Parkinson’s.
“In healthcare, there are different levels of ageism,” explained Hannah Swift, PhD, reader in social and organizational psychology at the University of Kent in the United Kingdom.
Ageism is embedded in the laws, rules, and practices of institutions, she explained. This became especially obvious during the pandemic, when health professionals had to decide who to treat, possibly using age as a proxy for making some of these decisions, she said.
“When you categorize people, you might be using stereotypes, assumptions, and expectations about age and that age group to make those decisions, and that’s where errors can occur.”
She added that ageist attitudes also become apparent at the interpersonal level by using patronizing language or offering unnecessary help to older people based on assumptions about their cognitive and physical abilities.
“Older age is often wrongly associated with declining levels of health and activity,” said Ittay Mannheim, PhD, guest postdoctoral researcher on aging and ageism at the Open University of the Netherlands. “However, older adults are a very diverse group, varying widely in many aspects, including health conditions. This stereotype can influence how healthcare professionals interact with them, assuming frailty or memory issues simply based on age. It’s important to recognize that being older doesn’t necessarily mean being ill.”
Mannheim’s research found that healthcare professionals often stand in the way of older people using technology-based treatments due to negative attitudes towards age. “So, actually, a barrier to using these technologies could be that healthcare professionals don’t think that someone can use it or won’t even offer it because someone looks old or is old,” he said.
The Impacts
Discrimination impacts the physical, mental, and social well-being of its victims. This includes attitudes towards age.
The PLOS ONE review of research on the global reach of ageism found that experienced or self-determined ageism was associated with significantly worse health outcomes across all countries examined. The same research team calculated that an estimated 6.3 million cases of depression worldwide are linked to ageism.
Other research has found that exposure to negative age stereotyping impacts willingness to adopt a healthy lifestyle in addition to increasing the risk for cardiovascular events.
What Can Be Done?
“Healthcare professionals frequently interact with older adults at their most vulnerable, which can reinforce negative stereotypes of older people being vulnerable or ill,” said Swift. “However, not all older adults fit these stereotypes. Many can live well and independently. Perhaps healthcare education should include reminders of the diverse experiences of older individuals rather than solely focusing on the moments when they require help.”
Research indicates that although progress has been made in geriatric training and the care of older individuals by healthcare education institutions, improved education and training are still needed at all levels of geriatric healthcare, including hospital administrators, physicians, nurses, personal caregivers, and associated health professions.
“Generally speaking, what healthcare professionals learn about aging tends to focus more on the biological aspects,” said Mannheim. “However, they may not fully understand what it means to be old or how to interact with older individuals, especially regarding technology. It is important to raise awareness about ageism because, in my experience working with healthcare professionals, even a single workshop on ageism can have a profound impact. Participants often respond with surprise, saying something like, ‘Wow, I never thought about this before.’”
Mannheim said that training healthcare providers to understand the aging process better could help to reduce any biases they might have and better prepare them to respond more adequately to the needs of older patients.
“We cannot devalue the lives of older people simply because they are older. It is crucial for all of us, especially governments, to acknowledge our responsibility to protect and promote human rights for individuals of all ages. If we fail to do this, the strategies we’ve witnessed during this pandemic will be repeated in the future,” said Nena Georgantzi, PhD, Barcelona-based human rights manager at AGE Platform Europe, an EU network of organizations of and for older people.
A version of this article appeared on Medscape.com.
Accelerated Approval of New Frontline TKI Use in CML Raises Questions
In October, the US Food and Drug Administration (FDA) granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed Philadelphia chromosome–positive CML in chronic phase.
Asciminib is one of the six tyrosine kinase inhibitor (TKI) drugs used for CML, a class that began with the introduction of imatinib (Gleevec) in 2001. By 2016, researchers reported that TKIs had helped make life expectancy in patients with CML approach that of the general population. Physicians and patients now have several options of second-generation TKI drugs that also can be used in newly diagnosed patients, along with the option to begin with the more affordable option of imatinib.
The FDA in 1992 instituted the accelerated approval pathway to try to speed market drugs for serious conditions that fill unmet medical needs.
The agency and companies essentially make bets on promising study results, often using surrogate markers, to allow sales of medicines while waiting for evidence from confirmatory studies. For example, the FDA in August used the accelerated approval process to clear the first T-cell receptor gene therapy for certain advanced forms of sarcoma, a form of cancer with limited treatment options.
The next accelerated approval of a cancer drug was the indication for asciminib as a frontline therapy. The FDA also used accelerated approval for the initial clearance of asciminib in 2021 for use in CML previously treated with two or more TKIs. By 2022, Novartis presented sufficient evidence of the drug’s merit to win full approval for the drug in this use.
The timeline is longer for the expected confirmatory research for asciminib as a frontline therapy, with a 2028 deadline set for this work. The data presented to date on asciminib have not persuaded some oncologists on the need for the speedy approval of frontline use.
“This boils down to a drug that looks as if it’s just as good as other second-generation TKIs,” Mikkael A. Sekeres, MD, MS, chief of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami. “I don’t know how they could use the accelerated approval mechanism to get this through.”
Sekeres, a former chair of the Oncologic Drugs Advisory Committee, explored concerns and challenges involved with the use of the accelerated approval process in his 2022 book, Drugs and the FDA: Safety, Efficacy, and the Public’s Trust.
“The intent of the accelerated approval mechanism is that you’re bringing a new therapy to treat a serious disease in a way that others haven’t previously, where there aren’t existing options,” Sekeres said.
This is a markedly different situation that exists for CML, where medicines have improved dramatically in the 21st century, unlike many other forms of cancer treated by hematologists.
“As someone who specializes in treating people with leukemia, I’d be happy every clinic day of my life if all of my patients came in with chronic phase, chronic myeloid leukemia,” instead of other cancers lacking these robust treatment options, he said.
With CML, physicians select among TKIs considering side effects and other health conditions patients have, including weighing the impact of financial toxicity in some cases, he said.
“If I have a patient with lower risk chronic phase, chronic myeloid leukemia, I’m treating them with imatinib,” Sekeres said.
Questions About Surrogate Endpoints
Sekeres is not alone in questioning the use of the speedier FDA pathway for a new indication for a TKI in CML.
“Where is the ‘unmet need’ justifying an accelerated approval in this setting?” Timothée Olivier, MD, who is affiliated with both the Hôpitaux universitaires de Genève and the VK Prasad Laboratory funded by Vinay Prasad, MD, MPH, wrote in a November 3 post on X.
Olivier, Prasad, and coauthors in a September correspondence to the American Journal of Hematology raised questions about the study design for a key asciminib study, ASC4FIRST. They noted what they consider a weakness with the endpoints used.
“Molecular milestones like the 48-week MMR [major molecular response] are often used in clinical trials due to their convenience and shorter timeline for assessment,” they wrote. “However, these milestones are not definitive indicators of long-term survival or overall clinical benefit.”
There has been rising concern in recent years about the evidence gap between initial accelerated approvals and the completion of studies that show whether these promising therapies actually help patients live longer or better. Researchers including Bishal Gyawali, MD, PhD, a Medscape Medical News contributor, also have questioned the degree of reliance on surrogate endpoints in accelerated approvals.
In response, the FDA’s Cancer Division and the US Congress have taken steps to try to force drugmakers to more quickly answer the key question in accelerated approvals: Does this medicine produce the expected benefits? For example, the FDA in March appears to have turned down a bid for accelerated approval of a lymphoma drug due to concerns about the timing of completion of confirmatory research.
The use of accelerated approval will continue to be a balancing act, due in part to demand for newer agents, Ravi Bhatia, MD, of the O’Neal Comprehensive Cancer Center at The University of Alabama at Birmingham, told this news organization.
“Accelerated approval of agents for up-front treatment of CML does not appear well justified, given the high degree of efficacy of existing agents,” said Bhatia, vice chair of the National Comprehensive Cancer Network’s Clinical Practice Guidelines in Oncology Panel for Chronic Myeloid Leukemia.
“On the other hand, there is greater urgency for developing agents for patients who have failed existing agents and patients with advanced phase disease, and the use of accelerated approval may be justified in this setting,” Bhatia said.
In an interview with this news organization, Richard A. Larson, MD, a professor in the Department of Hematology/Oncology at The University of Chicago, who is an ASC4FIRST investigator, noted the 96-week follow-up data from the trial will be presented at the annual meeting of the American Society of Hematology in December in San Diego.
Larson said data from this trial will show continued benefit with the frontline use of asciminib. Larson also is an author of a New England Journal of Medicine article in May about the ASC4FIRST trial.
“The data speak for themselves, that asciminib is at least as effective or more so and at least as well tolerated as what’s already on the market,” Larson said. “So their argument, at the end of the day, really boils down to the cost of a new drug and whether we need a new drug.”
From the point of view of patients with cancer, the answer to that is clear, he said.
“If you talk to cancer patients, they’d like to see new drugs become available as quickly as possible. And I think that was the original rationale for the accelerated approval pathway, that a drug that has been shown to be safe and effective in a prospective clinical trial could get accelerated approval based on a surrogate endpoint.”
The remarkable success seen in developing TKI drugs for CML creates difficulties in testing later entrants in this class due to their prolonged survival, Larson said.
“If you look on a population basis, the overall survival of newly diagnosed CML patients with all of these therapeutic options available to them now approximate that of the non-CML population.”
“For most anticancer drugs, the FDA would like to see an overall survival benefit, but patients with newly diagnosed CML are surviving 20 or 30 years, and they’re not dying at an accelerated rate the way they were. So it’d be impractical to require a clinical trial to show a survival benefit, a randomized trial.”
“That’s where the use of a surrogate endpoint, which is the major molecular response at 1 year, has been so valuable, gets the drugs approved, gets them into patients far earlier than if there was a survival end point requirement,” he said.
Larson reported ties with AbbVie, Amgen, Astellas, Celgene, Cellectis, Curis, CVS Caremark, Daiichi Sankyo, ImmunoGen, Jazz, MorphoSys, Rigel, Servier, Forty Seven/Gilead, Novartis, and Rafael Pharmaceuticals. Sekeres disclosed relationships with BMS, Kurome, and Novartis Advisory Boards. Bhatia reported no relevant disclosures.
A version of this article appeared on Medscape.com.
In October, the US Food and Drug Administration (FDA) granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed Philadelphia chromosome–positive CML in chronic phase.
Asciminib is one of the six tyrosine kinase inhibitor (TKI) drugs used for CML, a class that began with the introduction of imatinib (Gleevec) in 2001. By 2016, researchers reported that TKIs had helped make life expectancy in patients with CML approach that of the general population. Physicians and patients now have several options of second-generation TKI drugs that also can be used in newly diagnosed patients, along with the option to begin with the more affordable option of imatinib.
The FDA in 1992 instituted the accelerated approval pathway to try to speed market drugs for serious conditions that fill unmet medical needs.
The agency and companies essentially make bets on promising study results, often using surrogate markers, to allow sales of medicines while waiting for evidence from confirmatory studies. For example, the FDA in August used the accelerated approval process to clear the first T-cell receptor gene therapy for certain advanced forms of sarcoma, a form of cancer with limited treatment options.
The next accelerated approval of a cancer drug was the indication for asciminib as a frontline therapy. The FDA also used accelerated approval for the initial clearance of asciminib in 2021 for use in CML previously treated with two or more TKIs. By 2022, Novartis presented sufficient evidence of the drug’s merit to win full approval for the drug in this use.
The timeline is longer for the expected confirmatory research for asciminib as a frontline therapy, with a 2028 deadline set for this work. The data presented to date on asciminib have not persuaded some oncologists on the need for the speedy approval of frontline use.
“This boils down to a drug that looks as if it’s just as good as other second-generation TKIs,” Mikkael A. Sekeres, MD, MS, chief of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami. “I don’t know how they could use the accelerated approval mechanism to get this through.”
Sekeres, a former chair of the Oncologic Drugs Advisory Committee, explored concerns and challenges involved with the use of the accelerated approval process in his 2022 book, Drugs and the FDA: Safety, Efficacy, and the Public’s Trust.
“The intent of the accelerated approval mechanism is that you’re bringing a new therapy to treat a serious disease in a way that others haven’t previously, where there aren’t existing options,” Sekeres said.
This is a markedly different situation that exists for CML, where medicines have improved dramatically in the 21st century, unlike many other forms of cancer treated by hematologists.
“As someone who specializes in treating people with leukemia, I’d be happy every clinic day of my life if all of my patients came in with chronic phase, chronic myeloid leukemia,” instead of other cancers lacking these robust treatment options, he said.
With CML, physicians select among TKIs considering side effects and other health conditions patients have, including weighing the impact of financial toxicity in some cases, he said.
“If I have a patient with lower risk chronic phase, chronic myeloid leukemia, I’m treating them with imatinib,” Sekeres said.
Questions About Surrogate Endpoints
Sekeres is not alone in questioning the use of the speedier FDA pathway for a new indication for a TKI in CML.
“Where is the ‘unmet need’ justifying an accelerated approval in this setting?” Timothée Olivier, MD, who is affiliated with both the Hôpitaux universitaires de Genève and the VK Prasad Laboratory funded by Vinay Prasad, MD, MPH, wrote in a November 3 post on X.
Olivier, Prasad, and coauthors in a September correspondence to the American Journal of Hematology raised questions about the study design for a key asciminib study, ASC4FIRST. They noted what they consider a weakness with the endpoints used.
“Molecular milestones like the 48-week MMR [major molecular response] are often used in clinical trials due to their convenience and shorter timeline for assessment,” they wrote. “However, these milestones are not definitive indicators of long-term survival or overall clinical benefit.”
There has been rising concern in recent years about the evidence gap between initial accelerated approvals and the completion of studies that show whether these promising therapies actually help patients live longer or better. Researchers including Bishal Gyawali, MD, PhD, a Medscape Medical News contributor, also have questioned the degree of reliance on surrogate endpoints in accelerated approvals.
In response, the FDA’s Cancer Division and the US Congress have taken steps to try to force drugmakers to more quickly answer the key question in accelerated approvals: Does this medicine produce the expected benefits? For example, the FDA in March appears to have turned down a bid for accelerated approval of a lymphoma drug due to concerns about the timing of completion of confirmatory research.
The use of accelerated approval will continue to be a balancing act, due in part to demand for newer agents, Ravi Bhatia, MD, of the O’Neal Comprehensive Cancer Center at The University of Alabama at Birmingham, told this news organization.
“Accelerated approval of agents for up-front treatment of CML does not appear well justified, given the high degree of efficacy of existing agents,” said Bhatia, vice chair of the National Comprehensive Cancer Network’s Clinical Practice Guidelines in Oncology Panel for Chronic Myeloid Leukemia.
“On the other hand, there is greater urgency for developing agents for patients who have failed existing agents and patients with advanced phase disease, and the use of accelerated approval may be justified in this setting,” Bhatia said.
In an interview with this news organization, Richard A. Larson, MD, a professor in the Department of Hematology/Oncology at The University of Chicago, who is an ASC4FIRST investigator, noted the 96-week follow-up data from the trial will be presented at the annual meeting of the American Society of Hematology in December in San Diego.
Larson said data from this trial will show continued benefit with the frontline use of asciminib. Larson also is an author of a New England Journal of Medicine article in May about the ASC4FIRST trial.
“The data speak for themselves, that asciminib is at least as effective or more so and at least as well tolerated as what’s already on the market,” Larson said. “So their argument, at the end of the day, really boils down to the cost of a new drug and whether we need a new drug.”
From the point of view of patients with cancer, the answer to that is clear, he said.
“If you talk to cancer patients, they’d like to see new drugs become available as quickly as possible. And I think that was the original rationale for the accelerated approval pathway, that a drug that has been shown to be safe and effective in a prospective clinical trial could get accelerated approval based on a surrogate endpoint.”
The remarkable success seen in developing TKI drugs for CML creates difficulties in testing later entrants in this class due to their prolonged survival, Larson said.
“If you look on a population basis, the overall survival of newly diagnosed CML patients with all of these therapeutic options available to them now approximate that of the non-CML population.”
“For most anticancer drugs, the FDA would like to see an overall survival benefit, but patients with newly diagnosed CML are surviving 20 or 30 years, and they’re not dying at an accelerated rate the way they were. So it’d be impractical to require a clinical trial to show a survival benefit, a randomized trial.”
“That’s where the use of a surrogate endpoint, which is the major molecular response at 1 year, has been so valuable, gets the drugs approved, gets them into patients far earlier than if there was a survival end point requirement,” he said.
Larson reported ties with AbbVie, Amgen, Astellas, Celgene, Cellectis, Curis, CVS Caremark, Daiichi Sankyo, ImmunoGen, Jazz, MorphoSys, Rigel, Servier, Forty Seven/Gilead, Novartis, and Rafael Pharmaceuticals. Sekeres disclosed relationships with BMS, Kurome, and Novartis Advisory Boards. Bhatia reported no relevant disclosures.
A version of this article appeared on Medscape.com.
In October, the US Food and Drug Administration (FDA) granted accelerated approval to asciminib (Scemblix, Novartis AG) for adult patients with newly diagnosed Philadelphia chromosome–positive CML in chronic phase.
Asciminib is one of the six tyrosine kinase inhibitor (TKI) drugs used for CML, a class that began with the introduction of imatinib (Gleevec) in 2001. By 2016, researchers reported that TKIs had helped make life expectancy in patients with CML approach that of the general population. Physicians and patients now have several options of second-generation TKI drugs that also can be used in newly diagnosed patients, along with the option to begin with the more affordable option of imatinib.
The FDA in 1992 instituted the accelerated approval pathway to try to speed market drugs for serious conditions that fill unmet medical needs.
The agency and companies essentially make bets on promising study results, often using surrogate markers, to allow sales of medicines while waiting for evidence from confirmatory studies. For example, the FDA in August used the accelerated approval process to clear the first T-cell receptor gene therapy for certain advanced forms of sarcoma, a form of cancer with limited treatment options.
The next accelerated approval of a cancer drug was the indication for asciminib as a frontline therapy. The FDA also used accelerated approval for the initial clearance of asciminib in 2021 for use in CML previously treated with two or more TKIs. By 2022, Novartis presented sufficient evidence of the drug’s merit to win full approval for the drug in this use.
The timeline is longer for the expected confirmatory research for asciminib as a frontline therapy, with a 2028 deadline set for this work. The data presented to date on asciminib have not persuaded some oncologists on the need for the speedy approval of frontline use.
“This boils down to a drug that looks as if it’s just as good as other second-generation TKIs,” Mikkael A. Sekeres, MD, MS, chief of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami Health System, Miami. “I don’t know how they could use the accelerated approval mechanism to get this through.”
Sekeres, a former chair of the Oncologic Drugs Advisory Committee, explored concerns and challenges involved with the use of the accelerated approval process in his 2022 book, Drugs and the FDA: Safety, Efficacy, and the Public’s Trust.
“The intent of the accelerated approval mechanism is that you’re bringing a new therapy to treat a serious disease in a way that others haven’t previously, where there aren’t existing options,” Sekeres said.
This is a markedly different situation that exists for CML, where medicines have improved dramatically in the 21st century, unlike many other forms of cancer treated by hematologists.
“As someone who specializes in treating people with leukemia, I’d be happy every clinic day of my life if all of my patients came in with chronic phase, chronic myeloid leukemia,” instead of other cancers lacking these robust treatment options, he said.
With CML, physicians select among TKIs considering side effects and other health conditions patients have, including weighing the impact of financial toxicity in some cases, he said.
“If I have a patient with lower risk chronic phase, chronic myeloid leukemia, I’m treating them with imatinib,” Sekeres said.
Questions About Surrogate Endpoints
Sekeres is not alone in questioning the use of the speedier FDA pathway for a new indication for a TKI in CML.
“Where is the ‘unmet need’ justifying an accelerated approval in this setting?” Timothée Olivier, MD, who is affiliated with both the Hôpitaux universitaires de Genève and the VK Prasad Laboratory funded by Vinay Prasad, MD, MPH, wrote in a November 3 post on X.
Olivier, Prasad, and coauthors in a September correspondence to the American Journal of Hematology raised questions about the study design for a key asciminib study, ASC4FIRST. They noted what they consider a weakness with the endpoints used.
“Molecular milestones like the 48-week MMR [major molecular response] are often used in clinical trials due to their convenience and shorter timeline for assessment,” they wrote. “However, these milestones are not definitive indicators of long-term survival or overall clinical benefit.”
There has been rising concern in recent years about the evidence gap between initial accelerated approvals and the completion of studies that show whether these promising therapies actually help patients live longer or better. Researchers including Bishal Gyawali, MD, PhD, a Medscape Medical News contributor, also have questioned the degree of reliance on surrogate endpoints in accelerated approvals.
In response, the FDA’s Cancer Division and the US Congress have taken steps to try to force drugmakers to more quickly answer the key question in accelerated approvals: Does this medicine produce the expected benefits? For example, the FDA in March appears to have turned down a bid for accelerated approval of a lymphoma drug due to concerns about the timing of completion of confirmatory research.
The use of accelerated approval will continue to be a balancing act, due in part to demand for newer agents, Ravi Bhatia, MD, of the O’Neal Comprehensive Cancer Center at The University of Alabama at Birmingham, told this news organization.
“Accelerated approval of agents for up-front treatment of CML does not appear well justified, given the high degree of efficacy of existing agents,” said Bhatia, vice chair of the National Comprehensive Cancer Network’s Clinical Practice Guidelines in Oncology Panel for Chronic Myeloid Leukemia.
“On the other hand, there is greater urgency for developing agents for patients who have failed existing agents and patients with advanced phase disease, and the use of accelerated approval may be justified in this setting,” Bhatia said.
In an interview with this news organization, Richard A. Larson, MD, a professor in the Department of Hematology/Oncology at The University of Chicago, who is an ASC4FIRST investigator, noted the 96-week follow-up data from the trial will be presented at the annual meeting of the American Society of Hematology in December in San Diego.
Larson said data from this trial will show continued benefit with the frontline use of asciminib. Larson also is an author of a New England Journal of Medicine article in May about the ASC4FIRST trial.
“The data speak for themselves, that asciminib is at least as effective or more so and at least as well tolerated as what’s already on the market,” Larson said. “So their argument, at the end of the day, really boils down to the cost of a new drug and whether we need a new drug.”
From the point of view of patients with cancer, the answer to that is clear, he said.
“If you talk to cancer patients, they’d like to see new drugs become available as quickly as possible. And I think that was the original rationale for the accelerated approval pathway, that a drug that has been shown to be safe and effective in a prospective clinical trial could get accelerated approval based on a surrogate endpoint.”
The remarkable success seen in developing TKI drugs for CML creates difficulties in testing later entrants in this class due to their prolonged survival, Larson said.
“If you look on a population basis, the overall survival of newly diagnosed CML patients with all of these therapeutic options available to them now approximate that of the non-CML population.”
“For most anticancer drugs, the FDA would like to see an overall survival benefit, but patients with newly diagnosed CML are surviving 20 or 30 years, and they’re not dying at an accelerated rate the way they were. So it’d be impractical to require a clinical trial to show a survival benefit, a randomized trial.”
“That’s where the use of a surrogate endpoint, which is the major molecular response at 1 year, has been so valuable, gets the drugs approved, gets them into patients far earlier than if there was a survival end point requirement,” he said.
Larson reported ties with AbbVie, Amgen, Astellas, Celgene, Cellectis, Curis, CVS Caremark, Daiichi Sankyo, ImmunoGen, Jazz, MorphoSys, Rigel, Servier, Forty Seven/Gilead, Novartis, and Rafael Pharmaceuticals. Sekeres disclosed relationships with BMS, Kurome, and Novartis Advisory Boards. Bhatia reported no relevant disclosures.
A version of this article appeared on Medscape.com.
Inside the Patient-Oncologist Bond: Why It’s Often So Strong
Rose Gerber was 39, mother to a third grader and a kindergartener, when the diagnosis came: Advanced HER2-positive breast cancer.
“On one of my first or second appointments, I took in a little picture of Alexander and Isabella,” Gerber said. Gerber showed her oncologist the picture and told her: “I’ll do anything. I just want to be there for them.”
That was 21 years ago. Today, her current cancer status is “no evidence of disease.”
Over the past 2 decades, Gerber has gotten to be there for her children. Her youngest is now a television producer and her oldest, a CPA.
In that time,
“I’ve seen multiple physicians over my 21 years, but my oncologist has always been the focal point, guiding me in the right direction,” Gerber said in an interview.
Over the years, Jaga guided Gerber through a range of treatment decisions, including a Herceptin clinical trial that the mom of two views as lifesaving. Jaga often took on the role of both doctor and therapist, even providing comfort in the smaller moments when Gerber would fret about her weight gain.
The oncologist-patient “bond is very, very, very special,” said Gerber, who now works as director of patient advocacy and education at the Community Oncology Alliance.
Gerber isn’t alone in calling out the depth of the oncologist-patient bond.
Over years, sometimes decades, patients and oncologists can experience a whole world together: The treatment successes, relapses, uncertainties, and tough calls. As a result, a deep therapeutic alliance often develops. And with each new hurdle or decision, that collaborative, human connection between doctor and patient continues to form new layers.
“It’s like a shared bonding experience over trauma, like strangers trapped on a subway and then we get out, and we’re now on the other side, celebrating together,” said Saad Khan, MD, an associate professor of medicine (oncology) at Stanford University in California.
Connecting Through Stress
Although studies exploring the oncologist-patient bond are limited, some research suggests that a strong therapeutic alliance between patients and oncologists not only provides a foundation for quality care but can also help improve patients’ quality of life, protect against suicidal ideation, and increase treatment adherence.
Because of how stressful and frightening a cancer diagnosis can be, creating “a trusting, uninterrupted, almost sacred environment for them” is paramount for Khan. “I have no doubt that the most important part of their treatment is that they find an oncologist in whom they have total confidence,” Khan wrote in a blog.
The stress that patients with cancer experience is well documented, but oncologists take on a lot themselves and can also experience intense stress (.
“I consider my patient’s battles to be my battles,” Khan wrote.
The stress can start with the daily schedule. Oncologists often have a high volume of patients and tend to spend more time with each individual than most.
According to a 2023 survey, oncologists see about 68 patients a week, on average, but some oncologists, like Khan, have many more. Khan typically sees 20-30 patients a day and continues to care for many over years.
The survey also found that oncologists tend to spend a lot of time with their patients. Compared with other physicians, oncologists are two times more likely to spend at least 25 minutes with each patient.
With this kind of patient volume and time, Khan said, “you’re going to be exhausted.”
What can compound the exhaustion are the occasions oncologists need to deliver bad news — this treatment isn’t working, your cancer has come roaring back and, perhaps the hardest, we have no therapeutic options left. The end-of-life conversations, in particular, can be heartbreaking, especially when a patient is young and not ready to stop trying.
“It can be hard for doctors to discuss the end of life,” Don Dizon, MD, director of the Pelvic Malignancies Program at Lifespan Cancer Institute and director of Medical Oncology at Rhode Island Hospital, Providence, wrote in a column in 2023. Instead, it can be tempting and is often easier to focus on the next treatment, “instilling hope that there’s more that can be done,” even if doing more will only do harm.
In the face of these challenging decisions, growing a personal connection with patients over time can help keep oncologists going.
“We’re not just chemotherapy salesmen,” Khan said in an interview. “We get to know their social support network, who’s going to be driving them [to and from appointments], where they go on vacation, their cat’s name, who their neighbors are.”
A ‘Special Relationship’
Ralph V. Boccia, MD, is often asked what he does.
The next question that often comes — “Why do I do what I do?” — is Boccia’s favorite.
“Someone needs to take these patients through their journey,” Boccia, the founder of The Center for Cancer and Blood Disorders, Bethesda, Maryland, typically responds. He also often notes that “it is a special relationship you develop with the patient and their families.”
Boccia thinks about one long-term patient who captures this bond.
Joan Pinson, 70, was diagnosed with multiple myeloma about 25 years ago, when patients’ average survival was about 4 years.
Over a quarter century, Pinson has pivoted to different treatments, amid multiple relapses and remissions. Throughout most of this cancer journey, Boccia has been her primary oncologist, performing a stem cell transplant in 2000 and steering her to six clinical trials.
Her last relapse was 2 years ago, and since then she has been doing well on oral chemotherapy.
“Every time I relapsed, by the next appointment, he’d say, ‘here is what we are going to do,’ ” Pinson recalled. “I never worried, I never panicked. I knew he would take care of me.”
Over the years, Pinson and Boccia have shared many personal moments, sometimes by accident. One special moment happened early on in Pinson’s cancer journey. During an appointment, Boccia had “one ear to the phone” as his wife was about to deliver their first baby, Pinson recalled.
Later, Pinson met that child as a young man working in Boccia’s lab. She has also met Boccia’s wife, a nurse, when she filled in one day in the chemotherapy room.
Boccia now also treats Pinson’s husband who has prostate cancer, and he ruled out cancer when Pinson’s son, now in his 40s, had some worrisome symptoms.
More than 2 decades ago, Pinson told Boccia her goal was to see her youngest child graduate from high school. Now, six grandsons later, she has lived far beyond that goal.
“He has kept me alive,” said Pinson.
The Dying Patient
Harsha Vyas, MD, FACP, remembers the first encounter his office had with a 29-year-old woman referred with a diagnosis of stage IV breast cancer.
After just 15 minutes in the waiting room, the woman announced she was leaving. Although office staff assured the woman that she was next, the patient walked out.
Several months later, Vyas was called for an inpatient consult. It was the same woman.
Her lungs were full of fluid, and she was struggling to breathe, said Vyas, president and CEO of the Cancer Center of Middle Georgia, Dublin, and assistant professor at Augusta University in Georgia.
The woman, a single mother, told Vyas about her three young kids at home and asked him, “Doc, do something, please help me,” he recalled.
“Absolutely,” Vyas told her. But he had to be brutally honest about her prognosis and firm that she needed to follow his instructions. “You have a breast cancer I cannot cure,” he said. “All I can do is control the disease.”
From that first day, until the day she died, she came to every appointment and followed the treatment plan Vyas laid out.
For about 2 years, she responded well to treatment. And as the time passed and the trust grew, she began to open up to him. She showed him pictures. She talked about her children and being a mother.
“I’ve got to get my kids in a better place. I’m going to be there for them,” he recalled her saying.
Vyas admired her resourcefulness. She held down a part-time job, working retail and at a local restaurant. She figured out childcare so she could get to her chemotherapy appointments every 3 weeks and manage the copays.
Several years later, when she knew she was approaching the end of her life, she asked Vyas a question that hit hard.
“Doc, I don’t want to die and my kids find me dead. What can we do about it?”
Vyas, who has three daughters, imagined how traumatic this would be for a child. She and Vyas made the shared decision to cease treatment and begin home hospice. When the end was approaching, a hospice worker took over, waiting for bodily functions to cease.
When news of a death comes, “I say a little prayer, it’s almost like a send-off for that soul. That helps me absorb the news ... and let it go.”
But when the bond grows strong over time, as with his patient with breast cancer, Vyas said, “a piece of her is still with me.”
Khan had no relevant disclosures. Boccia and Vyas had no disclosures.
A version of this article appeared on Medscape.com.
Rose Gerber was 39, mother to a third grader and a kindergartener, when the diagnosis came: Advanced HER2-positive breast cancer.
“On one of my first or second appointments, I took in a little picture of Alexander and Isabella,” Gerber said. Gerber showed her oncologist the picture and told her: “I’ll do anything. I just want to be there for them.”
That was 21 years ago. Today, her current cancer status is “no evidence of disease.”
Over the past 2 decades, Gerber has gotten to be there for her children. Her youngest is now a television producer and her oldest, a CPA.
In that time,
“I’ve seen multiple physicians over my 21 years, but my oncologist has always been the focal point, guiding me in the right direction,” Gerber said in an interview.
Over the years, Jaga guided Gerber through a range of treatment decisions, including a Herceptin clinical trial that the mom of two views as lifesaving. Jaga often took on the role of both doctor and therapist, even providing comfort in the smaller moments when Gerber would fret about her weight gain.
The oncologist-patient “bond is very, very, very special,” said Gerber, who now works as director of patient advocacy and education at the Community Oncology Alliance.
Gerber isn’t alone in calling out the depth of the oncologist-patient bond.
Over years, sometimes decades, patients and oncologists can experience a whole world together: The treatment successes, relapses, uncertainties, and tough calls. As a result, a deep therapeutic alliance often develops. And with each new hurdle or decision, that collaborative, human connection between doctor and patient continues to form new layers.
“It’s like a shared bonding experience over trauma, like strangers trapped on a subway and then we get out, and we’re now on the other side, celebrating together,” said Saad Khan, MD, an associate professor of medicine (oncology) at Stanford University in California.
Connecting Through Stress
Although studies exploring the oncologist-patient bond are limited, some research suggests that a strong therapeutic alliance between patients and oncologists not only provides a foundation for quality care but can also help improve patients’ quality of life, protect against suicidal ideation, and increase treatment adherence.
Because of how stressful and frightening a cancer diagnosis can be, creating “a trusting, uninterrupted, almost sacred environment for them” is paramount for Khan. “I have no doubt that the most important part of their treatment is that they find an oncologist in whom they have total confidence,” Khan wrote in a blog.
The stress that patients with cancer experience is well documented, but oncologists take on a lot themselves and can also experience intense stress (.
“I consider my patient’s battles to be my battles,” Khan wrote.
The stress can start with the daily schedule. Oncologists often have a high volume of patients and tend to spend more time with each individual than most.
According to a 2023 survey, oncologists see about 68 patients a week, on average, but some oncologists, like Khan, have many more. Khan typically sees 20-30 patients a day and continues to care for many over years.
The survey also found that oncologists tend to spend a lot of time with their patients. Compared with other physicians, oncologists are two times more likely to spend at least 25 minutes with each patient.
With this kind of patient volume and time, Khan said, “you’re going to be exhausted.”
What can compound the exhaustion are the occasions oncologists need to deliver bad news — this treatment isn’t working, your cancer has come roaring back and, perhaps the hardest, we have no therapeutic options left. The end-of-life conversations, in particular, can be heartbreaking, especially when a patient is young and not ready to stop trying.
“It can be hard for doctors to discuss the end of life,” Don Dizon, MD, director of the Pelvic Malignancies Program at Lifespan Cancer Institute and director of Medical Oncology at Rhode Island Hospital, Providence, wrote in a column in 2023. Instead, it can be tempting and is often easier to focus on the next treatment, “instilling hope that there’s more that can be done,” even if doing more will only do harm.
In the face of these challenging decisions, growing a personal connection with patients over time can help keep oncologists going.
“We’re not just chemotherapy salesmen,” Khan said in an interview. “We get to know their social support network, who’s going to be driving them [to and from appointments], where they go on vacation, their cat’s name, who their neighbors are.”
A ‘Special Relationship’
Ralph V. Boccia, MD, is often asked what he does.
The next question that often comes — “Why do I do what I do?” — is Boccia’s favorite.
“Someone needs to take these patients through their journey,” Boccia, the founder of The Center for Cancer and Blood Disorders, Bethesda, Maryland, typically responds. He also often notes that “it is a special relationship you develop with the patient and their families.”
Boccia thinks about one long-term patient who captures this bond.
Joan Pinson, 70, was diagnosed with multiple myeloma about 25 years ago, when patients’ average survival was about 4 years.
Over a quarter century, Pinson has pivoted to different treatments, amid multiple relapses and remissions. Throughout most of this cancer journey, Boccia has been her primary oncologist, performing a stem cell transplant in 2000 and steering her to six clinical trials.
Her last relapse was 2 years ago, and since then she has been doing well on oral chemotherapy.
“Every time I relapsed, by the next appointment, he’d say, ‘here is what we are going to do,’ ” Pinson recalled. “I never worried, I never panicked. I knew he would take care of me.”
Over the years, Pinson and Boccia have shared many personal moments, sometimes by accident. One special moment happened early on in Pinson’s cancer journey. During an appointment, Boccia had “one ear to the phone” as his wife was about to deliver their first baby, Pinson recalled.
Later, Pinson met that child as a young man working in Boccia’s lab. She has also met Boccia’s wife, a nurse, when she filled in one day in the chemotherapy room.
Boccia now also treats Pinson’s husband who has prostate cancer, and he ruled out cancer when Pinson’s son, now in his 40s, had some worrisome symptoms.
More than 2 decades ago, Pinson told Boccia her goal was to see her youngest child graduate from high school. Now, six grandsons later, she has lived far beyond that goal.
“He has kept me alive,” said Pinson.
The Dying Patient
Harsha Vyas, MD, FACP, remembers the first encounter his office had with a 29-year-old woman referred with a diagnosis of stage IV breast cancer.
After just 15 minutes in the waiting room, the woman announced she was leaving. Although office staff assured the woman that she was next, the patient walked out.
Several months later, Vyas was called for an inpatient consult. It was the same woman.
Her lungs were full of fluid, and she was struggling to breathe, said Vyas, president and CEO of the Cancer Center of Middle Georgia, Dublin, and assistant professor at Augusta University in Georgia.
The woman, a single mother, told Vyas about her three young kids at home and asked him, “Doc, do something, please help me,” he recalled.
“Absolutely,” Vyas told her. But he had to be brutally honest about her prognosis and firm that she needed to follow his instructions. “You have a breast cancer I cannot cure,” he said. “All I can do is control the disease.”
From that first day, until the day she died, she came to every appointment and followed the treatment plan Vyas laid out.
For about 2 years, she responded well to treatment. And as the time passed and the trust grew, she began to open up to him. She showed him pictures. She talked about her children and being a mother.
“I’ve got to get my kids in a better place. I’m going to be there for them,” he recalled her saying.
Vyas admired her resourcefulness. She held down a part-time job, working retail and at a local restaurant. She figured out childcare so she could get to her chemotherapy appointments every 3 weeks and manage the copays.
Several years later, when she knew she was approaching the end of her life, she asked Vyas a question that hit hard.
“Doc, I don’t want to die and my kids find me dead. What can we do about it?”
Vyas, who has three daughters, imagined how traumatic this would be for a child. She and Vyas made the shared decision to cease treatment and begin home hospice. When the end was approaching, a hospice worker took over, waiting for bodily functions to cease.
When news of a death comes, “I say a little prayer, it’s almost like a send-off for that soul. That helps me absorb the news ... and let it go.”
But when the bond grows strong over time, as with his patient with breast cancer, Vyas said, “a piece of her is still with me.”
Khan had no relevant disclosures. Boccia and Vyas had no disclosures.
A version of this article appeared on Medscape.com.
Rose Gerber was 39, mother to a third grader and a kindergartener, when the diagnosis came: Advanced HER2-positive breast cancer.
“On one of my first or second appointments, I took in a little picture of Alexander and Isabella,” Gerber said. Gerber showed her oncologist the picture and told her: “I’ll do anything. I just want to be there for them.”
That was 21 years ago. Today, her current cancer status is “no evidence of disease.”
Over the past 2 decades, Gerber has gotten to be there for her children. Her youngest is now a television producer and her oldest, a CPA.
In that time,
“I’ve seen multiple physicians over my 21 years, but my oncologist has always been the focal point, guiding me in the right direction,” Gerber said in an interview.
Over the years, Jaga guided Gerber through a range of treatment decisions, including a Herceptin clinical trial that the mom of two views as lifesaving. Jaga often took on the role of both doctor and therapist, even providing comfort in the smaller moments when Gerber would fret about her weight gain.
The oncologist-patient “bond is very, very, very special,” said Gerber, who now works as director of patient advocacy and education at the Community Oncology Alliance.
Gerber isn’t alone in calling out the depth of the oncologist-patient bond.
Over years, sometimes decades, patients and oncologists can experience a whole world together: The treatment successes, relapses, uncertainties, and tough calls. As a result, a deep therapeutic alliance often develops. And with each new hurdle or decision, that collaborative, human connection between doctor and patient continues to form new layers.
“It’s like a shared bonding experience over trauma, like strangers trapped on a subway and then we get out, and we’re now on the other side, celebrating together,” said Saad Khan, MD, an associate professor of medicine (oncology) at Stanford University in California.
Connecting Through Stress
Although studies exploring the oncologist-patient bond are limited, some research suggests that a strong therapeutic alliance between patients and oncologists not only provides a foundation for quality care but can also help improve patients’ quality of life, protect against suicidal ideation, and increase treatment adherence.
Because of how stressful and frightening a cancer diagnosis can be, creating “a trusting, uninterrupted, almost sacred environment for them” is paramount for Khan. “I have no doubt that the most important part of their treatment is that they find an oncologist in whom they have total confidence,” Khan wrote in a blog.
The stress that patients with cancer experience is well documented, but oncologists take on a lot themselves and can also experience intense stress (.
“I consider my patient’s battles to be my battles,” Khan wrote.
The stress can start with the daily schedule. Oncologists often have a high volume of patients and tend to spend more time with each individual than most.
According to a 2023 survey, oncologists see about 68 patients a week, on average, but some oncologists, like Khan, have many more. Khan typically sees 20-30 patients a day and continues to care for many over years.
The survey also found that oncologists tend to spend a lot of time with their patients. Compared with other physicians, oncologists are two times more likely to spend at least 25 minutes with each patient.
With this kind of patient volume and time, Khan said, “you’re going to be exhausted.”
What can compound the exhaustion are the occasions oncologists need to deliver bad news — this treatment isn’t working, your cancer has come roaring back and, perhaps the hardest, we have no therapeutic options left. The end-of-life conversations, in particular, can be heartbreaking, especially when a patient is young and not ready to stop trying.
“It can be hard for doctors to discuss the end of life,” Don Dizon, MD, director of the Pelvic Malignancies Program at Lifespan Cancer Institute and director of Medical Oncology at Rhode Island Hospital, Providence, wrote in a column in 2023. Instead, it can be tempting and is often easier to focus on the next treatment, “instilling hope that there’s more that can be done,” even if doing more will only do harm.
In the face of these challenging decisions, growing a personal connection with patients over time can help keep oncologists going.
“We’re not just chemotherapy salesmen,” Khan said in an interview. “We get to know their social support network, who’s going to be driving them [to and from appointments], where they go on vacation, their cat’s name, who their neighbors are.”
A ‘Special Relationship’
Ralph V. Boccia, MD, is often asked what he does.
The next question that often comes — “Why do I do what I do?” — is Boccia’s favorite.
“Someone needs to take these patients through their journey,” Boccia, the founder of The Center for Cancer and Blood Disorders, Bethesda, Maryland, typically responds. He also often notes that “it is a special relationship you develop with the patient and their families.”
Boccia thinks about one long-term patient who captures this bond.
Joan Pinson, 70, was diagnosed with multiple myeloma about 25 years ago, when patients’ average survival was about 4 years.
Over a quarter century, Pinson has pivoted to different treatments, amid multiple relapses and remissions. Throughout most of this cancer journey, Boccia has been her primary oncologist, performing a stem cell transplant in 2000 and steering her to six clinical trials.
Her last relapse was 2 years ago, and since then she has been doing well on oral chemotherapy.
“Every time I relapsed, by the next appointment, he’d say, ‘here is what we are going to do,’ ” Pinson recalled. “I never worried, I never panicked. I knew he would take care of me.”
Over the years, Pinson and Boccia have shared many personal moments, sometimes by accident. One special moment happened early on in Pinson’s cancer journey. During an appointment, Boccia had “one ear to the phone” as his wife was about to deliver their first baby, Pinson recalled.
Later, Pinson met that child as a young man working in Boccia’s lab. She has also met Boccia’s wife, a nurse, when she filled in one day in the chemotherapy room.
Boccia now also treats Pinson’s husband who has prostate cancer, and he ruled out cancer when Pinson’s son, now in his 40s, had some worrisome symptoms.
More than 2 decades ago, Pinson told Boccia her goal was to see her youngest child graduate from high school. Now, six grandsons later, she has lived far beyond that goal.
“He has kept me alive,” said Pinson.
The Dying Patient
Harsha Vyas, MD, FACP, remembers the first encounter his office had with a 29-year-old woman referred with a diagnosis of stage IV breast cancer.
After just 15 minutes in the waiting room, the woman announced she was leaving. Although office staff assured the woman that she was next, the patient walked out.
Several months later, Vyas was called for an inpatient consult. It was the same woman.
Her lungs were full of fluid, and she was struggling to breathe, said Vyas, president and CEO of the Cancer Center of Middle Georgia, Dublin, and assistant professor at Augusta University in Georgia.
The woman, a single mother, told Vyas about her three young kids at home and asked him, “Doc, do something, please help me,” he recalled.
“Absolutely,” Vyas told her. But he had to be brutally honest about her prognosis and firm that she needed to follow his instructions. “You have a breast cancer I cannot cure,” he said. “All I can do is control the disease.”
From that first day, until the day she died, she came to every appointment and followed the treatment plan Vyas laid out.
For about 2 years, she responded well to treatment. And as the time passed and the trust grew, she began to open up to him. She showed him pictures. She talked about her children and being a mother.
“I’ve got to get my kids in a better place. I’m going to be there for them,” he recalled her saying.
Vyas admired her resourcefulness. She held down a part-time job, working retail and at a local restaurant. She figured out childcare so she could get to her chemotherapy appointments every 3 weeks and manage the copays.
Several years later, when she knew she was approaching the end of her life, she asked Vyas a question that hit hard.
“Doc, I don’t want to die and my kids find me dead. What can we do about it?”
Vyas, who has three daughters, imagined how traumatic this would be for a child. She and Vyas made the shared decision to cease treatment and begin home hospice. When the end was approaching, a hospice worker took over, waiting for bodily functions to cease.
When news of a death comes, “I say a little prayer, it’s almost like a send-off for that soul. That helps me absorb the news ... and let it go.”
But when the bond grows strong over time, as with his patient with breast cancer, Vyas said, “a piece of her is still with me.”
Khan had no relevant disclosures. Boccia and Vyas had no disclosures.
A version of this article appeared on Medscape.com.
‘Being a Doctor Isn’t Healthy’: Train Your Body to Handle It
Heather K. Schopper, MD, a head and neck surgeon at Penn State Health, Hershey, Pennsylvania, wasn’t long into her career when she began feeling its physical demands. Standing for 12 hours at a time, holding awkward positions for long periods, and working with surgical tables and instruments made for doctors much taller and larger meant severe back, shoulder, and neck pain at the end of every shift.
“You just want to lie down on the floor at the end of the day,” Schopper explained. “The wear and tear of our profession is really challenging.”
Here’s the thing: At the time Schopper wasn’t particularly out of shape. She only knew she needed to build up her body for long days and a long career. What, physically, would that look like?
This was the catalyst for what she calls a “health and fitness journey” that transformed the way she practices.
“Medicine is unique in its physical demands,” said Meghan Wieser, PT, DPT, a doctor of physical therapy at Recharge Health and Fitness in Ellicott City, Maryland. Wieser frequently works with physicians and others in high-stress career environments, and she’s observed the serious toll that physically demanding medical practice can take on the body.
It’s not just about preventing acute or chronic injury, she said. It’s about performing better for longer periods. And every doctor knows the only way to build a more functional body is training.
The Fantasy of Physical Perfection vs the Reality of, Well, Reality
Jordan D. Metzl, MD, is a sports medicine physician at Hospital for Special Surgery (HSS) in New York City. He’s also a lifelong triathlete and marathon runner and has parlayed that passion into an online fitness community of more than 10,000 people called Ironstrength. Through that, Metzl has led free exercise classes in Central Park for years. He doesn’t dabble. Three times a year he leads a boot camp class of more than 1000 people on the flight deck of the USS Intrepid on the Hudson River.
“I get it, being a doctor is all about the hours,” he said. “The time sacrifices get brutal and you have to cut something out, sometimes every day. For a lot of us, that’s exercise.”
Metzl understands it so well that he recently began leading twice-monthly boot camp classes just for his HSS physician colleagues on Wednesday mornings. He says those doctors both want and need that extra boost and will be aggressive about making time for it.
“The better shape you’re in, the better job you’ll do as a physician,” he said. “You’ll feel better when the hours get long. In my own career, I have always been a better doctor when I’m active and in shape.”
Knowledge isn’t really the issue for physicians. Reality is. And reality dictates that doctors have just as much issue with achieving consistency as any patient they prescribe exercise to.
Metzl suggests total body functional training to mimic real-world movement, particularly core and lower body to keep you upright for hours at a time. How do you schedule that? He uses early mornings and weekends to train for his races and run his fitness classes, which is why his primary advice is to focus not on the activity, but on time.
“Schedule full workouts when you can and steal the rest,” he said.
Schopper agrees. “You may not be able to fit in 60 minutes of exercise every day, but 20-30 minutes of intentional movement is key,” she explained. “When you have a day off, prioritize a longer session of something you can’t fit in on workdays.”
Those shorter bouts of exercise might include “bookending” the day with 10 minutes of burpees in the morning and then 10 minutes of bodyweight strength moves like planks, push-ups, and air squats in the evening.
“Bodyweight exercises are low-hanging fruit,” said Wieser. “If you’ve got a short window, aim for something that can shoot your heart rate up quickly.”
You can also throw in “movement snacks” throughout the day — skip the elevator and run up a flight of stairs, walk around during a quick lunch break, or throw in a set of jumping jacks between patients. (Don’t worry — you won’t be dripping sweat when they walk in.)
Remember, the rehab room in the orthopedic wing may have a few dumbbells and exercise bands you can utilize when you have 5 extra minutes in your day. “Any way you can squeeze in extra movement counts,” said Wieser.
Feats of Strength? Neighborhood Sprints? It All Matters
Kissinger Goldman, DO, a Florida-based ER physician, began his dedication to exercise 17 years ago, after a high-cholesterol diagnosis. “Did I have time to exercise in medical school and residency? Yes,” Goldman admitted. “But I didn’t have the same commitment to my health until I received that number. I set about to change everything.”
Goldman follows the approach of dividing up his exercise routine into short or long sessions, depending on his schedule. “If I’m off, I’ll aim for 30 minutes of cardio and 30 minutes of strength and core work,” he explained. “When I have to work, I’ll do a compressed version of that routine as soon as I wake up, and make sure the cardio is very intense — I’ll sprint in my neighborhood, for instance.”
Matt Klein, a doctor of physical therapy and professor at George Fox University in Newberg, Oregon, who has treated many doctors, says that, when pushed for time, just 20 minutes of “heavy” strength training can deliver good results. “The definition of heavy will vary, but aim for a weight that is challenging, whether a beginner or a more experienced exerciser,” he said. “Most doctors won’t have time to go to the gym, so a simple set of dumbbells or kettlebells will work just fine. The easier it is to access, the more likely you are to do it consistently.”
Klein is a fan of strength training with good reason: “Strength is a predictor of chronic disease, so doing some high-level strength training or power training can go a long way,” he said.
The endorphin high and overall sense of improved well-being are an extra bonus. Goldman credits it with ensuring he rarely misses a workout.
Get Hardcore About Sleep
Consider the following passage: “There are clear negative effects of sleep deprivation on performance, including reaction time, accuracy, vigor, submaximal strength, and endurance. Cognitive functions such as judgment and decision-making also suffer.”
Does that sound like how you feel on suboptimal sleep? That’s from an International Journal of Sports Medicine study on the effects of sleep deprivation on athletes.
Athletes aren’t doctors — but when you consider “reaction time, accuracy, endurance, judgment, and decision-making” — doctors could certainly benefit by thinking like athletes.
Schopper is serious about sleep and sets firm boundaries.
“It’s hard,” she admitted. “We want to work, see our families, have fun. But I work hard to say, ‘I’m done,’ and go to bed.”
“Rest is crucial for this job,” agreed Goldman. “If you don’t have adequate sleep, your cortisone levels are going to go up. When you’re exhausted and you’re working, you’re likely to miss something.” Goldman is consistent with early bedtimes around 9:00 or 9:30 PM, and he allows for a bit of “wind-down” time by reading for about 20 minutes before nodding off.
Goldman also sees a link between rest and improved interactions with patients. “There’s a direct correlation between number of hours worked in a row with respect to ‘customer service’ with patients,” he said.
But don’t aim for perfection. Allow some wiggle room for the time you spend asleep, Klein recommends. “We’ve always aimed for 8 hours, but there’s evidence that even 6 or 7 hours can be enough to allow you to recover as needed,” he said. “Optimally, you want that to be uninterrupted, but if not, a 10-minute power nap can help with mental clarity.”
Keep Searching, Keep Trying, Keep Training
Schopper was never, nor has she become, a gym rat. Still, “I knew I needed to build upper body strength,” she said. That meant expanding her fitness possibilities beyond the obvious. She discovered aerial arts — intense workouts using straps and other suspension tools to work every muscle in her body while hanging from the ceiling. Increased strength was a given, but she also seriously increased her range of motion.
For Schopper, the improvements to her lifestyle have been game changers. “I still have long days, but I’m no longer sore and tired after them,” she said. “I sleep better and have more energy. I’m proud of myself for putting the effort into this.”
A journey toward health and fitness may look different for everyone, but (as doctors frequently tell their patients) it’s a path anyone can follow.
“Being a doctor is not necessarily good for your health,” said Klein. “The body can handle the job, however, if you train for it.”
A version of this article first appeared on Medscape.com.
Heather K. Schopper, MD, a head and neck surgeon at Penn State Health, Hershey, Pennsylvania, wasn’t long into her career when she began feeling its physical demands. Standing for 12 hours at a time, holding awkward positions for long periods, and working with surgical tables and instruments made for doctors much taller and larger meant severe back, shoulder, and neck pain at the end of every shift.
“You just want to lie down on the floor at the end of the day,” Schopper explained. “The wear and tear of our profession is really challenging.”
Here’s the thing: At the time Schopper wasn’t particularly out of shape. She only knew she needed to build up her body for long days and a long career. What, physically, would that look like?
This was the catalyst for what she calls a “health and fitness journey” that transformed the way she practices.
“Medicine is unique in its physical demands,” said Meghan Wieser, PT, DPT, a doctor of physical therapy at Recharge Health and Fitness in Ellicott City, Maryland. Wieser frequently works with physicians and others in high-stress career environments, and she’s observed the serious toll that physically demanding medical practice can take on the body.
It’s not just about preventing acute or chronic injury, she said. It’s about performing better for longer periods. And every doctor knows the only way to build a more functional body is training.
The Fantasy of Physical Perfection vs the Reality of, Well, Reality
Jordan D. Metzl, MD, is a sports medicine physician at Hospital for Special Surgery (HSS) in New York City. He’s also a lifelong triathlete and marathon runner and has parlayed that passion into an online fitness community of more than 10,000 people called Ironstrength. Through that, Metzl has led free exercise classes in Central Park for years. He doesn’t dabble. Three times a year he leads a boot camp class of more than 1000 people on the flight deck of the USS Intrepid on the Hudson River.
“I get it, being a doctor is all about the hours,” he said. “The time sacrifices get brutal and you have to cut something out, sometimes every day. For a lot of us, that’s exercise.”
Metzl understands it so well that he recently began leading twice-monthly boot camp classes just for his HSS physician colleagues on Wednesday mornings. He says those doctors both want and need that extra boost and will be aggressive about making time for it.
“The better shape you’re in, the better job you’ll do as a physician,” he said. “You’ll feel better when the hours get long. In my own career, I have always been a better doctor when I’m active and in shape.”
Knowledge isn’t really the issue for physicians. Reality is. And reality dictates that doctors have just as much issue with achieving consistency as any patient they prescribe exercise to.
Metzl suggests total body functional training to mimic real-world movement, particularly core and lower body to keep you upright for hours at a time. How do you schedule that? He uses early mornings and weekends to train for his races and run his fitness classes, which is why his primary advice is to focus not on the activity, but on time.
“Schedule full workouts when you can and steal the rest,” he said.
Schopper agrees. “You may not be able to fit in 60 minutes of exercise every day, but 20-30 minutes of intentional movement is key,” she explained. “When you have a day off, prioritize a longer session of something you can’t fit in on workdays.”
Those shorter bouts of exercise might include “bookending” the day with 10 minutes of burpees in the morning and then 10 minutes of bodyweight strength moves like planks, push-ups, and air squats in the evening.
“Bodyweight exercises are low-hanging fruit,” said Wieser. “If you’ve got a short window, aim for something that can shoot your heart rate up quickly.”
You can also throw in “movement snacks” throughout the day — skip the elevator and run up a flight of stairs, walk around during a quick lunch break, or throw in a set of jumping jacks between patients. (Don’t worry — you won’t be dripping sweat when they walk in.)
Remember, the rehab room in the orthopedic wing may have a few dumbbells and exercise bands you can utilize when you have 5 extra minutes in your day. “Any way you can squeeze in extra movement counts,” said Wieser.
Feats of Strength? Neighborhood Sprints? It All Matters
Kissinger Goldman, DO, a Florida-based ER physician, began his dedication to exercise 17 years ago, after a high-cholesterol diagnosis. “Did I have time to exercise in medical school and residency? Yes,” Goldman admitted. “But I didn’t have the same commitment to my health until I received that number. I set about to change everything.”
Goldman follows the approach of dividing up his exercise routine into short or long sessions, depending on his schedule. “If I’m off, I’ll aim for 30 minutes of cardio and 30 minutes of strength and core work,” he explained. “When I have to work, I’ll do a compressed version of that routine as soon as I wake up, and make sure the cardio is very intense — I’ll sprint in my neighborhood, for instance.”
Matt Klein, a doctor of physical therapy and professor at George Fox University in Newberg, Oregon, who has treated many doctors, says that, when pushed for time, just 20 minutes of “heavy” strength training can deliver good results. “The definition of heavy will vary, but aim for a weight that is challenging, whether a beginner or a more experienced exerciser,” he said. “Most doctors won’t have time to go to the gym, so a simple set of dumbbells or kettlebells will work just fine. The easier it is to access, the more likely you are to do it consistently.”
Klein is a fan of strength training with good reason: “Strength is a predictor of chronic disease, so doing some high-level strength training or power training can go a long way,” he said.
The endorphin high and overall sense of improved well-being are an extra bonus. Goldman credits it with ensuring he rarely misses a workout.
Get Hardcore About Sleep
Consider the following passage: “There are clear negative effects of sleep deprivation on performance, including reaction time, accuracy, vigor, submaximal strength, and endurance. Cognitive functions such as judgment and decision-making also suffer.”
Does that sound like how you feel on suboptimal sleep? That’s from an International Journal of Sports Medicine study on the effects of sleep deprivation on athletes.
Athletes aren’t doctors — but when you consider “reaction time, accuracy, endurance, judgment, and decision-making” — doctors could certainly benefit by thinking like athletes.
Schopper is serious about sleep and sets firm boundaries.
“It’s hard,” she admitted. “We want to work, see our families, have fun. But I work hard to say, ‘I’m done,’ and go to bed.”
“Rest is crucial for this job,” agreed Goldman. “If you don’t have adequate sleep, your cortisone levels are going to go up. When you’re exhausted and you’re working, you’re likely to miss something.” Goldman is consistent with early bedtimes around 9:00 or 9:30 PM, and he allows for a bit of “wind-down” time by reading for about 20 minutes before nodding off.
Goldman also sees a link between rest and improved interactions with patients. “There’s a direct correlation between number of hours worked in a row with respect to ‘customer service’ with patients,” he said.
But don’t aim for perfection. Allow some wiggle room for the time you spend asleep, Klein recommends. “We’ve always aimed for 8 hours, but there’s evidence that even 6 or 7 hours can be enough to allow you to recover as needed,” he said. “Optimally, you want that to be uninterrupted, but if not, a 10-minute power nap can help with mental clarity.”
Keep Searching, Keep Trying, Keep Training
Schopper was never, nor has she become, a gym rat. Still, “I knew I needed to build upper body strength,” she said. That meant expanding her fitness possibilities beyond the obvious. She discovered aerial arts — intense workouts using straps and other suspension tools to work every muscle in her body while hanging from the ceiling. Increased strength was a given, but she also seriously increased her range of motion.
For Schopper, the improvements to her lifestyle have been game changers. “I still have long days, but I’m no longer sore and tired after them,” she said. “I sleep better and have more energy. I’m proud of myself for putting the effort into this.”
A journey toward health and fitness may look different for everyone, but (as doctors frequently tell their patients) it’s a path anyone can follow.
“Being a doctor is not necessarily good for your health,” said Klein. “The body can handle the job, however, if you train for it.”
A version of this article first appeared on Medscape.com.
Heather K. Schopper, MD, a head and neck surgeon at Penn State Health, Hershey, Pennsylvania, wasn’t long into her career when she began feeling its physical demands. Standing for 12 hours at a time, holding awkward positions for long periods, and working with surgical tables and instruments made for doctors much taller and larger meant severe back, shoulder, and neck pain at the end of every shift.
“You just want to lie down on the floor at the end of the day,” Schopper explained. “The wear and tear of our profession is really challenging.”
Here’s the thing: At the time Schopper wasn’t particularly out of shape. She only knew she needed to build up her body for long days and a long career. What, physically, would that look like?
This was the catalyst for what she calls a “health and fitness journey” that transformed the way she practices.
“Medicine is unique in its physical demands,” said Meghan Wieser, PT, DPT, a doctor of physical therapy at Recharge Health and Fitness in Ellicott City, Maryland. Wieser frequently works with physicians and others in high-stress career environments, and she’s observed the serious toll that physically demanding medical practice can take on the body.
It’s not just about preventing acute or chronic injury, she said. It’s about performing better for longer periods. And every doctor knows the only way to build a more functional body is training.
The Fantasy of Physical Perfection vs the Reality of, Well, Reality
Jordan D. Metzl, MD, is a sports medicine physician at Hospital for Special Surgery (HSS) in New York City. He’s also a lifelong triathlete and marathon runner and has parlayed that passion into an online fitness community of more than 10,000 people called Ironstrength. Through that, Metzl has led free exercise classes in Central Park for years. He doesn’t dabble. Three times a year he leads a boot camp class of more than 1000 people on the flight deck of the USS Intrepid on the Hudson River.
“I get it, being a doctor is all about the hours,” he said. “The time sacrifices get brutal and you have to cut something out, sometimes every day. For a lot of us, that’s exercise.”
Metzl understands it so well that he recently began leading twice-monthly boot camp classes just for his HSS physician colleagues on Wednesday mornings. He says those doctors both want and need that extra boost and will be aggressive about making time for it.
“The better shape you’re in, the better job you’ll do as a physician,” he said. “You’ll feel better when the hours get long. In my own career, I have always been a better doctor when I’m active and in shape.”
Knowledge isn’t really the issue for physicians. Reality is. And reality dictates that doctors have just as much issue with achieving consistency as any patient they prescribe exercise to.
Metzl suggests total body functional training to mimic real-world movement, particularly core and lower body to keep you upright for hours at a time. How do you schedule that? He uses early mornings and weekends to train for his races and run his fitness classes, which is why his primary advice is to focus not on the activity, but on time.
“Schedule full workouts when you can and steal the rest,” he said.
Schopper agrees. “You may not be able to fit in 60 minutes of exercise every day, but 20-30 minutes of intentional movement is key,” she explained. “When you have a day off, prioritize a longer session of something you can’t fit in on workdays.”
Those shorter bouts of exercise might include “bookending” the day with 10 minutes of burpees in the morning and then 10 minutes of bodyweight strength moves like planks, push-ups, and air squats in the evening.
“Bodyweight exercises are low-hanging fruit,” said Wieser. “If you’ve got a short window, aim for something that can shoot your heart rate up quickly.”
You can also throw in “movement snacks” throughout the day — skip the elevator and run up a flight of stairs, walk around during a quick lunch break, or throw in a set of jumping jacks between patients. (Don’t worry — you won’t be dripping sweat when they walk in.)
Remember, the rehab room in the orthopedic wing may have a few dumbbells and exercise bands you can utilize when you have 5 extra minutes in your day. “Any way you can squeeze in extra movement counts,” said Wieser.
Feats of Strength? Neighborhood Sprints? It All Matters
Kissinger Goldman, DO, a Florida-based ER physician, began his dedication to exercise 17 years ago, after a high-cholesterol diagnosis. “Did I have time to exercise in medical school and residency? Yes,” Goldman admitted. “But I didn’t have the same commitment to my health until I received that number. I set about to change everything.”
Goldman follows the approach of dividing up his exercise routine into short or long sessions, depending on his schedule. “If I’m off, I’ll aim for 30 minutes of cardio and 30 minutes of strength and core work,” he explained. “When I have to work, I’ll do a compressed version of that routine as soon as I wake up, and make sure the cardio is very intense — I’ll sprint in my neighborhood, for instance.”
Matt Klein, a doctor of physical therapy and professor at George Fox University in Newberg, Oregon, who has treated many doctors, says that, when pushed for time, just 20 minutes of “heavy” strength training can deliver good results. “The definition of heavy will vary, but aim for a weight that is challenging, whether a beginner or a more experienced exerciser,” he said. “Most doctors won’t have time to go to the gym, so a simple set of dumbbells or kettlebells will work just fine. The easier it is to access, the more likely you are to do it consistently.”
Klein is a fan of strength training with good reason: “Strength is a predictor of chronic disease, so doing some high-level strength training or power training can go a long way,” he said.
The endorphin high and overall sense of improved well-being are an extra bonus. Goldman credits it with ensuring he rarely misses a workout.
Get Hardcore About Sleep
Consider the following passage: “There are clear negative effects of sleep deprivation on performance, including reaction time, accuracy, vigor, submaximal strength, and endurance. Cognitive functions such as judgment and decision-making also suffer.”
Does that sound like how you feel on suboptimal sleep? That’s from an International Journal of Sports Medicine study on the effects of sleep deprivation on athletes.
Athletes aren’t doctors — but when you consider “reaction time, accuracy, endurance, judgment, and decision-making” — doctors could certainly benefit by thinking like athletes.
Schopper is serious about sleep and sets firm boundaries.
“It’s hard,” she admitted. “We want to work, see our families, have fun. But I work hard to say, ‘I’m done,’ and go to bed.”
“Rest is crucial for this job,” agreed Goldman. “If you don’t have adequate sleep, your cortisone levels are going to go up. When you’re exhausted and you’re working, you’re likely to miss something.” Goldman is consistent with early bedtimes around 9:00 or 9:30 PM, and he allows for a bit of “wind-down” time by reading for about 20 minutes before nodding off.
Goldman also sees a link between rest and improved interactions with patients. “There’s a direct correlation between number of hours worked in a row with respect to ‘customer service’ with patients,” he said.
But don’t aim for perfection. Allow some wiggle room for the time you spend asleep, Klein recommends. “We’ve always aimed for 8 hours, but there’s evidence that even 6 or 7 hours can be enough to allow you to recover as needed,” he said. “Optimally, you want that to be uninterrupted, but if not, a 10-minute power nap can help with mental clarity.”
Keep Searching, Keep Trying, Keep Training
Schopper was never, nor has she become, a gym rat. Still, “I knew I needed to build upper body strength,” she said. That meant expanding her fitness possibilities beyond the obvious. She discovered aerial arts — intense workouts using straps and other suspension tools to work every muscle in her body while hanging from the ceiling. Increased strength was a given, but she also seriously increased her range of motion.
For Schopper, the improvements to her lifestyle have been game changers. “I still have long days, but I’m no longer sore and tired after them,” she said. “I sleep better and have more energy. I’m proud of myself for putting the effort into this.”
A journey toward health and fitness may look different for everyone, but (as doctors frequently tell their patients) it’s a path anyone can follow.
“Being a doctor is not necessarily good for your health,” said Klein. “The body can handle the job, however, if you train for it.”
A version of this article first appeared on Medscape.com.
Blood Buddies: Can Mentorship Revive Classical Hematology?
But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.
“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”
Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.
Here’s a closer look at mentor-focused efforts to attract medical students to CH.
How Severe Is the Shortage in CH?
Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.
The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”
If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients.
Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.
“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said.
There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.
Why Does a Shortage Exist?
“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”
As a result, fewer fellows ended up specializing in CH, she said.
The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”
Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”
However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.”
As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”
Why Are Mentors Important?
Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”
As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”
As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”
Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.”
Does Research Support Mentorship in CH?
The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”
Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute.
The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month.
Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.
“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”
Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.”
There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field.
What’s Next for Mentorship in CH?
The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”
Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”
Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.
Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.
A version of this article appeared on Medscape.com.
But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.
“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”
Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.
Here’s a closer look at mentor-focused efforts to attract medical students to CH.
How Severe Is the Shortage in CH?
Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.
The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”
If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients.
Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.
“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said.
There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.
Why Does a Shortage Exist?
“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”
As a result, fewer fellows ended up specializing in CH, she said.
The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”
Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”
However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.”
As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”
Why Are Mentors Important?
Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”
As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”
As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”
Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.”
Does Research Support Mentorship in CH?
The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”
Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute.
The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month.
Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.
“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”
Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.”
There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field.
What’s Next for Mentorship in CH?
The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”
Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”
Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.
Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.
A version of this article appeared on Medscape.com.
But when it comes to turning people on to the idea of a career in classical hematology (CH), there may be no more powerful influence than a mentor who loves their job. That’s why the field is focusing so much on supporting mentors and mentees amid a stark shortage of classical hematologists.
“Mentorship is key for maintaining trainee interest in the field and for providing role models for career growth,” said Rakhi P. Naik, MD, MHS, associate professor of medicine and director of the Hematology Fellowship Track at Johns Hopkins University, Baltimore, Maryland, in an interview. “This collaboration is especially critical because there are so few trainees and so few mentors currently in the field.”
Now there’s new research backing up the power of mentorship, even when it’s only provided virtually, and a brand-new program aims to unite more mentors and mentees.
Here’s a closer look at mentor-focused efforts to attract medical students to CH.
How Severe Is the Shortage in CH?
Patients with conditions treated by classical hematologists are waiting months for appointments at many outpatient centers, with some being forced to wait 6 months or more, said Srikanth Nagalla, MD, chief of benign hematology at Miami Cancer Institute, Florida, in an interview.
The shortage is creating dire problems in the inpatient setting too, Nagalla said. “Serious blood disorders like heparin-induced thrombocytopenia, acute chest syndrome [a complication of sickle cell disease], and thrombotic thrombocytopenic purpura have to be diagnosed and treated in a timely manner. If not, the morbidity and mortality are really high.”
If classical hematologists aren’t available, he said, oncologists and others not trained in hematology will need to cover these patients.
Hematologist Ariela Marshall, MD, associate professor of medicine at the University of Minnesota in Minneapolis, noted in an interview that the CH shortage comes at a time when medical advances and an aging population are boosting the number of patients with noncancerous blood disorders. Older people are at greater risk for blood clots, she said. And lifespans for patients with bleeding and clotting disorders are rising thanks to effective new treatments.
“Because of our larger patient population in CH, we are going to need more classical hematologists to follow them for longer and longer periods of time,” she said.
There’s no sign yet that newly minted physicians will take up the slack in CH. A 2019 study found that just 4.6% of 626 of hematology/oncology fellows said they planned to go into CH, also known as benign hematology, vs 67.1% who expected to treat patients with solid tumors, blood cancer, or both. The rest, 24.6%, planned to work in CH plus the two oncology fields.
Why Does a Shortage Exist?
“The reasons are complex, but one of the most important factors was the combining of the adult hematology and medical oncology training programs by the Accreditation Council for Graduate Medical Education in 1995,” Naik said. “After that time, the majority of fellowship training programs went from having separate programs for hematology and medical oncology to combining the training for the two specialties into one. Because most of these combined training programs resided within Cancer Centers, classical hematology training slowly became de-emphasized.”
As a result, fewer fellows ended up specializing in CH, she said.
The field of CH also appears to suffer from a less than enticing reputation. According to a 2019 study coauthored by Marshall, surveys of thousands of hematology/oncology fellows found that “hematology, particularly benign hematology, was viewed as having poorer income potential, research funding, job availability, and job security than oncology.”
Regarding pay, Marshall said the good news is that many classical hematologists work in academia, where it’s common for pay to be “equitable across hematology/oncology divisions and based more on academic rank and other factors rather than subspecialty within hematology oncology.”
However, she noted, “this may differ at institutions where hematology and oncology are different departments. For example, centers where oncology is its own department, and hematology is part of the department of medicine.”
As for job availability, Naik said that there’s plenty of demand. “In academics, it is clear that there are jobs available everywhere, but trainees are often worried about job prospects in private practice. While classical hematology jobs in private practice are not widely advertised, I can attest that there is no shortage of need,” she said. “Many private practices do not specifically advertise for classical hematologists because they assume that classical hematology experts are not available. But I assure you that every private practice my trainees have ever approached is always ecstatic to hire a classical hematologist.”
Why Are Mentors Important?
Mentorship is crucial to promoting the value of CH as a great career choice in a competitive environment, classical hematologists say. “We can motivate trainees by showing how the disease states themselves are so fascinating and how the treatments are showing great outcomes,” Nagalla said. “We can show positive results, how patient lives can be changed, and how well-respected across the system [we] are.”
As a selling point, classical hematologists like to emphasize that their field requires intensive detective work. “Let’s say a patient comes with anemia, which might have 15 different causes. You get some labs, and then you systemically rule in or rule out most of these on the differential diagnosis,” Nagalla said. “Then once you narrow it down, you get more labs. You keep going to the next step and next step, and so finally you come to a conclusion.”
As for therapy, Marshall said that “while for many cancers there are specific treatment recommendations for patients with a specific cancer type at a specific stage, there is not always a specific treatment recommendation (or a ‘right answer’) for our CH patients. Treatment planning depends strongly on a patient’s preferences, other medical conditions, and a discussion about risks [and] benefits of different treatment options such that two patients with the same condition may choose two different treatment options.”
Marshall also emphasizes to trainees that “CH is a broad field. Physicians and trainees are able to interact and collaborate with physicians in other specialties such as gastroenterology, cardiology, ob/gyn, and surgical specialties.”
Does Research Support Mentorship in CH?
The 2019 study that revealed just 4.6% of fellows planned to go into CH found that “fellows who planned to enter hematology-only careers were significantly more likely to report having clinical training and mentorship experiences in hematology throughout their training relative to fellows with oncology-only or combined hematology/oncology career plans.”
Now there are more data to support mentorships. For a study published in Blood Advances in September 2024, Zoya Qureshy, MD, an internal medicine chief resident at the University of California at San Diego, and colleagues evaluated a year-long external membership program implemented by the American Society of Hematology (ASH) Medical Educators Institute.
The program linked 35 US hematology/oncology fellows (80% female, 46% White, 35% Asian) who were interested in CH to 34 North American faculty members. The pairs were told to meet virtually once a month.
Of 30 mentees and 23 mentors surveyed, 94% and 85%, respectively, said their pairings were good matches. Two thirds of the mentees accepted faculty positions in CH after their mentorships.
“Our study showed that external mentorship in a virtual format is feasible,” Qureshy said in an interview. “Additionally, external mentorship provided benefits such as different perspectives and the opportunity for mentorship for those who may not have it in their field of interest at their home institution.”
Qureshy added that “one strength of our mentorship program was that mentoring pairs were meticulously assigned based on shared interests and background. Many participants cited this common ground as a reason why they thought their mentoring pair was a good match.”
There’s an important caveat: Most of the mentees weren’t new to CH. About 70% had previously worked with a mentor in the CH field, and 86% had previously conducted research in the field.
What’s Next for Mentorship in CH?
The ASH Hematology-Focused Fellowship Training Program Consortium aims to mint 50 new academic hematologists by 2030 through programs at 12 institutions. “Mentorship is an exciting aspect of the program since it allows classical hematology trainees to form a network of peers nationally and also provides access to mentors across institutions,” Naik said. “And as the workforce grows, there will be more and more role models for future trainees to look up to.”
Moving forward, she said, “we hope to inspire even more institutions to adopt hematology training tracks throughout the country.”
Meanwhile, ASH’s new Classical Hematology Advancement Mentorship is taking applications for its debut 2025 program through January 9, 2025. Trainees will meet monthly with mentors both virtually and in person. Applicants must have been in their first or second year of hematology/oncology fellowship training at accredited programs in the United States as of July 15, 2024.
Naik, Marshall, Nagalla, and Qureshy have no relevant disclosures.
A version of this article appeared on Medscape.com.
Trump Nominations for US Health Agencies Spark Controversy, Criticism, Praise
President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:
- Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
- Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
- Fox News contributor Janette Nesheiwat, MD, for surgeon general.
Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS).
Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.
Martin A. Makary
Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool.
As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy.
Makary is also chief medical officer of telehealth platform Sesame.
Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials.
In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.
Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”
Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.
In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.
Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.
Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.
While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.
Janette Nesheiwat
As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.
She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.
Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith.
Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”
While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination.
“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.
David J. Weldon
If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.
After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.
He now practices as an internist in Brevard County, Florida.
In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.
Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.”
But some physicians criticized Weldon for what they called his anti-vaccine views.
A version of this article first appeared on Medscape.com.
President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:
- Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
- Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
- Fox News contributor Janette Nesheiwat, MD, for surgeon general.
Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS).
Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.
Martin A. Makary
Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool.
As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy.
Makary is also chief medical officer of telehealth platform Sesame.
Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials.
In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.
Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”
Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.
In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.
Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.
Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.
While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.
Janette Nesheiwat
As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.
She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.
Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith.
Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”
While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination.
“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.
David J. Weldon
If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.
After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.
He now practices as an internist in Brevard County, Florida.
In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.
Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.”
But some physicians criticized Weldon for what they called his anti-vaccine views.
A version of this article first appeared on Medscape.com.
President-elect Donald Trump’s vision for the nation’s top health agencies is coming into focus with three nominations announced Nov. 22 that drew both criticism and praise:
- Surgeon and health researcher Martin A. Makary, MD, MPH, to lead the US Food and Drug Administration (FDA).
- Former Republican congressman and physician David J. Weldon, MD, for director of the US Centers for Disease Control and Prevention (CDC).
- Fox News contributor Janette Nesheiwat, MD, for surgeon general.
Earlier in November, Trump nominated vaccine skeptic and former presidential candidate Robert F. Kennedy Jr. to lead the US Department of Health and Human Services (HHS).
Here’s what to know about the latest nominees, who, like Kennedy, must be confirmed by the US Senate.
Martin A. Makary
Currently a professor at the Johns Hopkins School of Medicine and chief of islet transplant surgery at Johns Hopkins Hospital, Makary co-invented in 2006 a surgery checklist that became a widely-used patient safety tool.
As a US FDA commissioner, Makary would preside over a $6.5 billion agency with more than 18,000 employees. The agency, part of HHS, oversees human and animal drugs and vaccines, medical devices, food, tobacco and other products. Some of Makary’s views align closely with those of HHS nominee Kennedy.
Makary is also chief medical officer of telehealth platform Sesame.
Makary was primarily known as a health researcher and author of books about price transparency and the cost of health care until the COVID-19 pandemic, when he became an outspoken critic of the federal response, lambasting restrictions and mandates advocated by the CDC and other public health officials.
In 2023, Makary told the House Select Subcommittee on the COVID Pandemic that federal officials had ignored what he called “natural immunity.” Studies have shown that natural immunity is “at least as effective as vaccinated immunity, and probably better,” testified Makary.
Makary called for an overhaul of the US FDA in a 2021 Fox News opinion, saying that its culture was “defined by counterproductive rigidity and a refusal to adapt.”
Blind Spots, his most recent book, takes on what he calls “medical dogma” and challenges conventional views on subjects ranging from the microbiome to marijuana to cancer prevention, hormone replacement therapy, antibiotics and peanut allergies.
In an interview he posted to X, Makary blames inappropriate use of antibiotics for a variety of childhood illnesses. He cites increases in obesity, learning disabilities, attention deficit disorder, asthma, celiac disease, ulcerative colitis and Crohn’s disease as all potentially causally related to antibiotics given in childhood.
Makary is an advisor to two conservative think tanks, the Foundation for Research on Equal Opportunity, and to Paragon Health Institute, begun in 2021 by two former top officials in the previous Trump administration.
Makary would “cut the bureaucratic red tape at the agency to make sure Americans get the medical cures and treatments they deserve,” Trump said on his social media platform, Truth Social, and in a press release.
While Los Angeles Times owner and physician-entrepreneur Patrick Soon-Shiong, MBBCh, MSc, praised the nomination of Makary (and the two other nominees) as “inspired,” other physicians criticized Makary for his anti-COVID mandate views and “fear-mongering” over COVID vaccine side effects.
Janette Nesheiwat
As surgeon general, Nesheiwat would serve as the top “health communicator in chief” and oversee the 6000 member US Public Health Service Commissioned Corps.
She is a frequent medical contributor to Fox News and serves as a medical director for a group of urgent care clinics in New York. She received her medical degree from the American University of the Caribbean School of Medicine and completed a family medicine residency at the University of Arkansas for Medical Sciences. She is board-certified in family medicine.
Nesheiwat sells vitamin supplements on her website and in December will publish a book on “miracles in medicine” and her Christian faith.
Trump said in a statement that Nesheiwat “is a fierce advocate and strong communicator for preventive medicine and public health. She is committed to ensuring that Americans have access to affordable, quality healthcare, and believes in empowering individuals to take charge of their health to live longer, healthier lives.”
While Nesheiwat was critical of COVID mandates, she voiced more support for COVID vaccines and mask-wearing during the pandemic than her fellow nominees, leading some Trump supporters to criticize her nomination.
“A good appointment, happy about this: I got to know @DoctorJanette during the pandemic, exchanging information. She is very smart, thoughtful, interested in learning, and a compassionate doctor, and…a truly nice person,” noted vaccine researcher Peter Hotez, MD, PhD, said on X.
David J. Weldon
If confirmed, former congressman Weldon would oversee the sprawling CDC, an agency with a roughly $17 billion budget, 15,000 employees or contractors, and numerous centers covering everything from health statistics to vaccines to epidemiology.
After earning his medical degree from the University at Buffalo School of Medicine, Weldon served in the US Army and US Army reserve. The Republican later served for 14 years in Congress representing Florida’s 15th district, which covers the Tampa region.
He now practices as an internist in Brevard County, Florida.
In Congress, Weldon raised concerns about the safety of some vaccines and promoted the false narrative that a former vaccine ingredient, thimerosal, caused autism, the Washington Post reported. Thimerosal has not been used in child vaccines for more than two decades. He also introduced a bill to move vaccine safety oversight from the CDC to an independent agency within HHS.
Trump said in a statement that Weldon “will proudly restore the CDC to its true purpose, and will work to end the Chronic Disease Epidemic.”
But some physicians criticized Weldon for what they called his anti-vaccine views.
A version of this article first appeared on Medscape.com.
ASH 2024 Myeloma Studies: My Top 10 Picks
First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks:
IFM 2017-03 Phase 3 Study
Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.
AQUILA Study
This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.
This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI.
All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed.
Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.
Extended Follow-Up of Anito-Cel in its First In-Human study
Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.
Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.
Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary
This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.
Further Information on Cevostamab, Another Bispecific Option
We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.
Is GPRC5D a Better Target for Car T Rather Than Bispecifics?
Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.
Daratumumab, a Game-Changer for AL Amyloidosis
A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.
Understanding how SMM Progresses to MM
We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323).
The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World
At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).
Delayed Neurotoxicity may not be Just a Consequence of high tumor burden
We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.
I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.
First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks:
IFM 2017-03 Phase 3 Study
Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.
AQUILA Study
This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.
This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI.
All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed.
Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.
Extended Follow-Up of Anito-Cel in its First In-Human study
Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.
Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.
Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary
This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.
Further Information on Cevostamab, Another Bispecific Option
We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.
Is GPRC5D a Better Target for Car T Rather Than Bispecifics?
Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.
Daratumumab, a Game-Changer for AL Amyloidosis
A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.
Understanding how SMM Progresses to MM
We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323).
The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World
At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).
Delayed Neurotoxicity may not be Just a Consequence of high tumor burden
We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.
I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.
First, let me place my selected studies in context by acknowledging my biases. As a clinician, I’m prone to choosing clinical rather than basic science or translational work — even if translational work might well end up exerting a pivotal impact on practice in the future. And now — in no particular order — here are my picks:
IFM 2017-03 Phase 3 Study
Frail patients are underrepresented in most myeloma studies, yet in this randomized trial for newly diagnosed myeloma, exclusively frail patients were enrolled. The trial compared daratumumab/lenalidomide to lenalidomide/dexamethasone, and the most recent follow/up shows a progression-free survival (PFS) (48.5 vs 21 months) and overall survival (OS) (not reached vs 36 months) benefit to daratumumab/lenalidomide. What I see in practice is that anti-CD38 monoclonal antibodies are the best-tolerated drugs in this space and should be the backbone of any regimen for frail patients. Steroids should be omitted as early as possible. Future trials may optimize what to give in addition to the anti-CD38 therapy, and how to adapt/escalate therapy to frailty and clinical status, as lenalidomide remains difficult for such patients to tolerate.
AQUILA Study
This is a randomized, phase 3 comparison of daratumumab to observation for patients with smoldering myeloma. The endpoint was PFS. For context, similar studies done in asymptomatic CLL have shown improved PFS, but not OS, and the authors of such studies have concluded that improvement in PFS alone should not justify a change in approach.
This study shows that daratumumab can improve laboratory markers and reduce progression (60-month PFS rates of 63.1% for daratumumab vs 40.8% with observation). However, several important caveats remain. The protocol only mandated spine/pelvis MRI imaging, not whole-body MRI imaging, and such imaging was only performed once a year, which may not be frequent enough to catch lesions at an earlier stage. These details have important implications, as previous research shows that up to half of lesions can be missed by doing only a spine MRI, as opposed to a whole-body MRI.
All of this means that had more comprehensive imaging been done, many more patients may have been diagnosed with myeloma. Such patients may have been undertreated, and single agent daratumumab, with a response rate of just 63%, may not have been enough. Conversely, some patients may also have been overtreated using this approach, as the protocol allowed patients who had been diagnosed with smoldering myeloma up to 5 years earlier to be enrolled. Many of these people could have had indolent disease for years prior to enrollment and may not have ever progressed.
Further information is needed to help us understand this study better. What was the nature of the progression events: asymptomatic lab changes or morbid end organ damage? Was daratumumab given when patients in the control arm progressed to myeloma? My concern is that if patients in the control arm do not universally receive modern daratumumab-containing therapies when they develop myeloma, then an overall survival advantage may be shown simply because patients in the intervention arm are getting a good drug earlier in the disease, while those in the control arm are not getting a good drug at all. Nevertheless, despite these limitations, it is likely this trial will lead to regulatory approval of daratumumab in this space, and lots of discussions from patients and clinicians alike.
Extended Follow-Up of Anito-Cel in its First In-Human study
Two chimeric antigen receptor therapy (CAR-T) products are currently approved for myeloma. Cilta-cel is clearly effective but is associated with problematic late-onset neurological toxicities. Ide-cel appears much less effective. There is clearly a need for a product that is both effective and safe.
Anito-cel has two relevant abstracts this meeting that show much promise. Extended follow-up of anito-cel from its first in human study shows a promising 27-month PFS of 52%, and with no cases of delayed neurotoxicity. I also eagerly await further information from the registrational single-arm study of anti-cel being presented at ASH 2024, which should (hopefully) lead to its accelerated approval.
Screening for Myeloma for all People With Vertebral Fractures Likely Unnecessary
This elegant study of over 9,000 people with vertebral fractures shows that absolute risks for myeloma were 0.43% and 0.63% in women and men with grade 2-3 fractures, respectively, indicating that there is likely little benefit in evaluating asymptomatic individuals with incidentally discovered vertebral fractures for myeloma, unless other features are present. Spread the word.
Further Information on Cevostamab, Another Bispecific Option
We do need effective treatments for targets beyond just BCMA and GPRC5D. Cevostamab, a bispecific targeting FCRH5, represents another option, with updated data on 167 patients. With an overall response rate of 43% (duration of response, 10 months), and a response rate of about 30% in those with prior bispecific exposure, this data helps us contextualize expected benefits as we look forward to the eventual approval of this drug. The efficacy is relatively modest in those who have already progressed on bispecifics, but cevostamab would still be a welcome addition to our arsenal.
Is GPRC5D a Better Target for Car T Rather Than Bispecifics?
Our currently available GPRC5D bispecific (talquetamab) leads to high rates of skin, oral, and nail toxicity. This drug can also bring significant weight loss. These side effects make me consider that continuous targeting of GPRC5D through a bispecific may not be ideal, and that GPRC5D may be better as a one-time CAR T target. At ASH 2024, we will have 15-month follow-up data from BMS-986393, a GPRC5D CAR T. Response rates for this heavily pretreated population (76% of whom had triple refractory disease) were at 87%, with a median PFS of 14.5 months. Only 6% of patients experienced treatment-related weight loss, and nail (19%), skin (30%), and oral (31%) toxicities were relatively low. I look forward to updated data, as well as data on the resolution of the toxicities seen.
Daratumumab, a Game-Changer for AL Amyloidosis
A truly effective drug given early can change the natural history of disease, even if patients in the control arm only receive the drug later. A case in point is daratumumab. The 5-year survival rate was 76.1% for the daratumumab/cyclophosphamide/bortezomib/dexamethasone arm and 64.7% for cyclophosphamide/bortezomib/dexamethasone arm. This happened despite the fact that 67% of the control arm patients (among those who received therapy) went on to receive daratumumab later in the disease course.
Understanding how SMM Progresses to MM
We often hear that we should treat SMM and not just watch carefully because fractures may suddenly happen, or a patient may end up on dialysis. What this retrospective study tells us that amongst 427 patients with SMM, 42 had progression to myeloma, and amongst those 42, only 1 developed renal dysfunction (unclear if this resolved), and only 1 had lytic lesions that were symptomatic. The remainder were all asymptomatic lab and imaging changes. This is a retrospective study, and one should assume that follow-up was thus highly variable. It does not appear that diffusion weighted whole-body MRI imaging (our most sensitive imaging test) was employed universally or very frequently. Nevertheless, these powerful findings reassure us that, with close observation, morbidity is unlikely. Our group has designed a prospective study incorporating frequent diffusion weighted whole body MRI imaging to formally test this hypothesis (SPOTLIGHT, NCT06212323).
The Underperformance of Daratumumab/Lenalidomide/Dexamethasone in the Real World
At every major meeting I am reminded of the disconnect between real-world efficacy and clinical trial efficacy. Case in point: In this Austrian experience, daratumumab/lenalidomide/dexamethasone led to a PFS of 22.7 months vs 61.9 months in the MAIA trial of daratumumab/lenalidomide/dexamethasone. Such a sobering difference! And if you think this is an isolated experience, even in a US real-world cohort, consider that in a recently published comparative study dara/len/dex underperformed, although the time to next treatment or death was longer (37.8 months).
Delayed Neurotoxicity may not be Just a Consequence of high tumor burden
We currently think that some of the scariest side effects of cilta-cel, such as delayed neurotoxicity, may be a consequence of a high number of cancer cells and may be prevented by better disease control at the time of infusion. This study, a sobering analysis of 52 patients with delayed neurotoxicity occurring after CAR T, included 8 patients (15%) who were not heavily pretreated, and all had less than 5% plasma cells at the time of infusion. None of these patients had extramedullary disease. This result worries me, especially because cilta-cel is being studied and is poised for future approval in earlier line settings. It suggests that this toxicity may not always be a product of disease burden, contrary to our current belief.
I will be paying close attention to these 10 myeloma studies at ASH 2024, where I look forward to meeting you and learning more.Dr. Mohyuddin is assistant professor in the multiple myeloma program at the Huntsman Cancer Institute at the University of Utah, Salt Lake City.
Recognizing Burnout: Why Physicians Often Miss the Signs in Themselves
Summary and Key Highlights
Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.
Key Takeaways:
- Many physicians struggle to identify burnout due to stigma and self-blame.
- Awareness of burnout symptoms is essential for early intervention and healthy coping.
- Seeking support can prevent burnout from worsening and improve quality of life.
Our Editors Also Recommend:
Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’
Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance
Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance
A Transformative Rx for Burnout, Grief, and Illness: Dance
Next Medscape Masters Event:
Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.
Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.
A version of this article first appeared on Medscape.com.
Summary and Key Highlights
Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.
Key Takeaways:
- Many physicians struggle to identify burnout due to stigma and self-blame.
- Awareness of burnout symptoms is essential for early intervention and healthy coping.
- Seeking support can prevent burnout from worsening and improve quality of life.
Our Editors Also Recommend:
Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’
Medscape Hospitalist Burnout & Depression Report 2024: Seeking Progress, Balance
Medscape Physician Lifestyle & Happiness Report 2024: The Ongoing Struggle for Balance
A Transformative Rx for Burnout, Grief, and Illness: Dance
Next Medscape Masters Event:
Stay at the forefront of obesity care. Register for exclusive insights and the latest treatment innovations.
Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.
A version of this article first appeared on Medscape.com.
Summary and Key Highlights
Summary: This section explores why physicians often struggle to recognize burnout within themselves, partly due to stigma and a tendency to focus on productivity over well-being. Dr. Tyra Fainstad shares personal experiences of burnout symptoms, emphasizing the importance of awareness and self-reflection. Recognizing and addressing burnout early can help physicians find healthier coping strategies, avoid productivity traps, and seek support.
Key Takeaways:
- Many physicians struggle to identify burnout due to stigma and self-blame.
- Awareness of burnout symptoms is essential for early intervention and healthy coping.
- Seeking support can prevent burnout from worsening and improve quality of life.
Our Editors Also Recommend:
Medscape Physician Burnout & Depression Report 2024: ‘We Have Much Work to Do’
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Lotte Dyrbye, has disclosed the following relevant financial relationships: Co-inventor of the Well-being Index and its derivatives, which Mayo Clinic has licensed. Dyrbye receives royalties.
A version of this article first appeared on Medscape.com.