Researchers from The Cancer Genome Atlas (TCGA) Research Network analyzed 178 primary cervical cancers, and found > 70% had genomic alteration in 1 or both of 2 important cell signaling pathways. They also found that a subset of tumors showed no evidence of HPV infection.

“This aspect of the research is one of the most intriguing findings to come out of the TCGA program, which has been looking at more than 30 tumor types over the past decade,” said Jean-Claude Zenklusen, PhD, director of the TCGA program office.

The researchers found several instances of amplification of genes that code for known immune targets, which may predict responsiveness to immunotherapy. They also identified several novel mutated genes. Particularly interesting, the researchers say, was the identification of a unique set of 8 cervical cancers that showed molecular similarities to endometrial cancers; the cancers were mainly HPV negative. That finding “confirms that not all cervical cancers are related to HPV infection and that a small percentage of cervical tumors may be due to strictly genetic or other factors,” said Zenklusen.

Researchers from The Cancer Genome Atlas (TCGA) Research Network analyzed 178 primary cervical cancers, and found > 70% had genomic alteration in 1 or both of 2 important cell signaling pathways. They also found that a subset of tumors showed no evidence of HPV infection.

“This aspect of the research is one of the most intriguing findings to come out of the TCGA program, which has been looking at more than 30 tumor types over the past decade,” said Jean-Claude Zenklusen, PhD, director of the TCGA program office.

The researchers found several instances of amplification of genes that code for known immune targets, which may predict responsiveness to immunotherapy. They also identified several novel mutated genes. Particularly interesting, the researchers say, was the identification of a unique set of 8 cervical cancers that showed molecular similarities to endometrial cancers; the cancers were mainly HPV negative. That finding “confirms that not all cervical cancers are related to HPV infection and that a small percentage of cervical tumors may be due to strictly genetic or other factors,” said Zenklusen.

Researchers from The Cancer Genome Atlas (TCGA) Research Network analyzed 178 primary cervical cancers, and found > 70% had genomic alteration in 1 or both of 2 important cell signaling pathways. They also found that a subset of tumors showed no evidence of HPV infection.

“This aspect of the research is one of the most intriguing findings to come out of the TCGA program, which has been looking at more than 30 tumor types over the past decade,” said Jean-Claude Zenklusen, PhD, director of the TCGA program office.

The researchers found several instances of amplification of genes that code for known immune targets, which may predict responsiveness to immunotherapy. They also identified several novel mutated genes. Particularly interesting, the researchers say, was the identification of a unique set of 8 cervical cancers that showed molecular similarities to endometrial cancers; the cancers were mainly HPV negative. That finding “confirms that not all cervical cancers are related to HPV infection and that a small percentage of cervical tumors may be due to strictly genetic or other factors,” said Zenklusen.

Photo by Daniel Gay

New research suggests assays used to screen donated blood for the Zika virus are more sensitive than assays used to help physicians diagnose Zika infection.

The study showed that donor screening assays could detect Zika virus RNA with greater sensitivity than diagnostic real-time polymerase chain reaction (RT-PCR) assays.

However, the evidence also indicated that increasing the volume of blood analyzed can increase the sensitivity of diagnostic assays.

This research was published in Transfusion in a special issue focusing on Zika and other transfusion-transmitted viruses.

The researchers compared 17 nucleic acid amplification technology assays used at 11 different labs. (Some of the 17 assays were actually the same assays used at different labs.)

One of the donor screening assays was the Procleix Zika virus assay, which was co-developed by Hologic, Inc. and Grifols Diagnostic Solutions, Inc. and used at Hologic.

The other donor screening assay was the cobas Zika test, which was developed by Roche Molecular Systems, Inc. and used in the company’s lab.

The RT-PCR diagnostic assays included the US Centers for Disease Control and Prevention’s (CDC) Singleplex (1087, 4481) and Trioplex assays—both low input (LI) and high input (HI)—which were used at the CDC labs in Puerto Rico (PR) and Fort Collins (FC).

Modified versions of the CDC’s assays were also used at the Blood Systems Research Institute (BSRI) in San Francisco, the University of California (UC) Davis, the Institut Louis Malarde (ILM) in French Polynesia, and 2 labs in Brazil—Fundação Pró-Sangue and Laboratório Richet.

The US Food and Drug Administration (FDA) used its own RT-PCR test, and the Etablissement Francais du Sang (EFS) in France used the Altona RealStar ZIKV RT-PCR assay.

Results

The various assays were used on plasma samples positive for 2 different strains of Zika virus—1 from Brazil and 1 from French Polynesia—as well as Zika-negative control samples.

The researchers found the donor screening assays provided comparable sensitivity and were more sensitive than each of the diagnostic RT-PCR assays.

So the team compared results with the 2 donor screening assays combined to results with the RT-PCR assays, which they grouped into 9 categories based on similar intended applications, methodologies, and results.

The 95% limit of detection (LOD₉₅) and 50% limit of detection (LOD₅₀) for the assays were as follows.

The researchers noted that the donor screening assays were about 10-fold to 100-fold more sensitive than the standard input RT-PCR assays.

However, the CDC’s assays were performed with low and high inputs of plasma. And increasing the sample input volume increased the limit of detection by 10-fold to 30-fold.

“The results of this study, that evaluated 17 Zika virus assays in 11 laboratories and documented excellent sensitivity of the 2 donor screening assays manufactured by Roche and Grifols, were critical to support the decision by the US Food and Drug Administration and blood industry to implement investigational screening of donors in Puerto Rico in April 2016 and the entire US by the end of 2016,” said study author Michael Busch, MD, PhD, of BSRI.

“Given the sensitivity of these assays, the FDA approved clinical trials using individual donation screening and rescinded earlier policies precluding transfusion of blood collected in Puerto Rico and deferral from donation by donors who had traveled to Zika risk countries throughout the US. This screening has detected over 350 infected blood donations in Puerto Rico and dozens of infected donations in the continental US.”

Photo by Daniel Gay

New research suggests assays used to screen donated blood for the Zika virus are more sensitive than assays used to help physicians diagnose Zika infection.

The study showed that donor screening assays could detect Zika virus RNA with greater sensitivity than diagnostic real-time polymerase chain reaction (RT-PCR) assays.

However, the evidence also indicated that increasing the volume of blood analyzed can increase the sensitivity of diagnostic assays.

This research was published in Transfusion in a special issue focusing on Zika and other transfusion-transmitted viruses.

The researchers compared 17 nucleic acid amplification technology assays used at 11 different labs. (Some of the 17 assays were actually the same assays used at different labs.)

One of the donor screening assays was the Procleix Zika virus assay, which was co-developed by Hologic, Inc. and Grifols Diagnostic Solutions, Inc. and used at Hologic.

The other donor screening assay was the cobas Zika test, which was developed by Roche Molecular Systems, Inc. and used in the company’s lab.

The RT-PCR diagnostic assays included the US Centers for Disease Control and Prevention’s (CDC) Singleplex (1087, 4481) and Trioplex assays—both low input (LI) and high input (HI)—which were used at the CDC labs in Puerto Rico (PR) and Fort Collins (FC).

Modified versions of the CDC’s assays were also used at the Blood Systems Research Institute (BSRI) in San Francisco, the University of California (UC) Davis, the Institut Louis Malarde (ILM) in French Polynesia, and 2 labs in Brazil—Fundação Pró-Sangue and Laboratório Richet.

The US Food and Drug Administration (FDA) used its own RT-PCR test, and the Etablissement Francais du Sang (EFS) in France used the Altona RealStar ZIKV RT-PCR assay.

Results

The various assays were used on plasma samples positive for 2 different strains of Zika virus—1 from Brazil and 1 from French Polynesia—as well as Zika-negative control samples.

The researchers found the donor screening assays provided comparable sensitivity and were more sensitive than each of the diagnostic RT-PCR assays.

So the team compared results with the 2 donor screening assays combined to results with the RT-PCR assays, which they grouped into 9 categories based on similar intended applications, methodologies, and results.

The 95% limit of detection (LOD₉₅) and 50% limit of detection (LOD₅₀) for the assays were as follows.

The researchers noted that the donor screening assays were about 10-fold to 100-fold more sensitive than the standard input RT-PCR assays.

However, the CDC’s assays were performed with low and high inputs of plasma. And increasing the sample input volume increased the limit of detection by 10-fold to 30-fold.

“The results of this study, that evaluated 17 Zika virus assays in 11 laboratories and documented excellent sensitivity of the 2 donor screening assays manufactured by Roche and Grifols, were critical to support the decision by the US Food and Drug Administration and blood industry to implement investigational screening of donors in Puerto Rico in April 2016 and the entire US by the end of 2016,” said study author Michael Busch, MD, PhD, of BSRI.

“Given the sensitivity of these assays, the FDA approved clinical trials using individual donation screening and rescinded earlier policies precluding transfusion of blood collected in Puerto Rico and deferral from donation by donors who had traveled to Zika risk countries throughout the US. This screening has detected over 350 infected blood donations in Puerto Rico and dozens of infected donations in the continental US.”

Photo by Daniel Gay

New research suggests assays used to screen donated blood for the Zika virus are more sensitive than assays used to help physicians diagnose Zika infection.

The study showed that donor screening assays could detect Zika virus RNA with greater sensitivity than diagnostic real-time polymerase chain reaction (RT-PCR) assays.

However, the evidence also indicated that increasing the volume of blood analyzed can increase the sensitivity of diagnostic assays.

This research was published in Transfusion in a special issue focusing on Zika and other transfusion-transmitted viruses.

The researchers compared 17 nucleic acid amplification technology assays used at 11 different labs. (Some of the 17 assays were actually the same assays used at different labs.)

One of the donor screening assays was the Procleix Zika virus assay, which was co-developed by Hologic, Inc. and Grifols Diagnostic Solutions, Inc. and used at Hologic.

The other donor screening assay was the cobas Zika test, which was developed by Roche Molecular Systems, Inc. and used in the company’s lab.

The RT-PCR diagnostic assays included the US Centers for Disease Control and Prevention’s (CDC) Singleplex (1087, 4481) and Trioplex assays—both low input (LI) and high input (HI)—which were used at the CDC labs in Puerto Rico (PR) and Fort Collins (FC).

Modified versions of the CDC’s assays were also used at the Blood Systems Research Institute (BSRI) in San Francisco, the University of California (UC) Davis, the Institut Louis Malarde (ILM) in French Polynesia, and 2 labs in Brazil—Fundação Pró-Sangue and Laboratório Richet.

The US Food and Drug Administration (FDA) used its own RT-PCR test, and the Etablissement Francais du Sang (EFS) in France used the Altona RealStar ZIKV RT-PCR assay.

Results

The various assays were used on plasma samples positive for 2 different strains of Zika virus—1 from Brazil and 1 from French Polynesia—as well as Zika-negative control samples.

The researchers found the donor screening assays provided comparable sensitivity and were more sensitive than each of the diagnostic RT-PCR assays.

So the team compared results with the 2 donor screening assays combined to results with the RT-PCR assays, which they grouped into 9 categories based on similar intended applications, methodologies, and results.

The 95% limit of detection (LOD₉₅) and 50% limit of detection (LOD₅₀) for the assays were as follows.

The researchers noted that the donor screening assays were about 10-fold to 100-fold more sensitive than the standard input RT-PCR assays.

However, the CDC’s assays were performed with low and high inputs of plasma. And increasing the sample input volume increased the limit of detection by 10-fold to 30-fold.

“The results of this study, that evaluated 17 Zika virus assays in 11 laboratories and documented excellent sensitivity of the 2 donor screening assays manufactured by Roche and Grifols, were critical to support the decision by the US Food and Drug Administration and blood industry to implement investigational screening of donors in Puerto Rico in April 2016 and the entire US by the end of 2016,” said study author Michael Busch, MD, PhD, of BSRI.

“Given the sensitivity of these assays, the FDA approved clinical trials using individual donation screening and rescinded earlier policies precluding transfusion of blood collected in Puerto Rico and deferral from donation by donors who had traveled to Zika risk countries throughout the US. This screening has detected over 350 infected blood donations in Puerto Rico and dozens of infected donations in the continental US.”

Photo by Chad McNeeley

MARSEILLE—An investigational drug has successfully treated a severe case of transplant-associated thrombotic microangiopathy (TA-TMA), according to a presentation at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation.

The drug is OMS721, a monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

The patient received OMS721, which is being developed by Omeros Corporation, under a compassionate-use protocol.

Marco Zecca, MD, of the Fondazione IRCCS Policlinico San Matteo in Italy, and his colleagues provided details on this patient in a poster presented at the meeting (Physician Poster Abstracts-Day 1, abstract A437).

The female patient had undergone a hematopoietic stem cell transplant to treat Diamond‐Blackfan anemia. At age 14, she received a transplant from an HLA-compatible, unrelated donor.

Seven months later, she was diagnosed with TA-TMA. The patient was initially treated with eculizumab but had to stop taking the drug after she developed acute pulmonary edema.

She was then treated with plasma exchange but experienced a TA-TMA relapse at 11 months. The patient was again treated with eculizumab and again had to discontinue the drug after developing acute pulmonary edema.

The patient’s condition continued to worsen, and she soon required hemodialysis 3 times a week as well as daily platelet transfusions.

Dr Zecca requested OMS721 as compassionate-use treatment for the patient, and Omeros complied.

Two months after starting OMS721, the patient was able to discontinue hemodialysis and decrease her platelet transfusion requirements. She did not experience any adverse events related to OMS721.

Recently, the patient’s dose was tapered, but she developed a viral infection that reactivated her TA-TMA.

A return to the original dose of OMS721 was successful. Now, the patient no longer requires dialysis or transfusions.

“This patient had severe TMA that I believe would have caused her death,” Dr Zecca said. “Her positive response to OMS721 treatment, both initially and following her virus-induced relapse during tapering, was impressive.”

“The results of OMS721 treatment in this challenge-rechallenge scenario underscore the important effects of the drug. Since the poster was produced, her TMA has remained in remission, and we have been able to discontinue her platelet transfusions. Her rapid response has been heartening, and we all are grateful for this remarkable outcome.”

Photo by Chad McNeeley

MARSEILLE—An investigational drug has successfully treated a severe case of transplant-associated thrombotic microangiopathy (TA-TMA), according to a presentation at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation.

The drug is OMS721, a monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

The patient received OMS721, which is being developed by Omeros Corporation, under a compassionate-use protocol.

Marco Zecca, MD, of the Fondazione IRCCS Policlinico San Matteo in Italy, and his colleagues provided details on this patient in a poster presented at the meeting (Physician Poster Abstracts-Day 1, abstract A437).

The female patient had undergone a hematopoietic stem cell transplant to treat Diamond‐Blackfan anemia. At age 14, she received a transplant from an HLA-compatible, unrelated donor.

Seven months later, she was diagnosed with TA-TMA. The patient was initially treated with eculizumab but had to stop taking the drug after she developed acute pulmonary edema.

She was then treated with plasma exchange but experienced a TA-TMA relapse at 11 months. The patient was again treated with eculizumab and again had to discontinue the drug after developing acute pulmonary edema.

The patient’s condition continued to worsen, and she soon required hemodialysis 3 times a week as well as daily platelet transfusions.

Dr Zecca requested OMS721 as compassionate-use treatment for the patient, and Omeros complied.

Two months after starting OMS721, the patient was able to discontinue hemodialysis and decrease her platelet transfusion requirements. She did not experience any adverse events related to OMS721.

Recently, the patient’s dose was tapered, but she developed a viral infection that reactivated her TA-TMA.

A return to the original dose of OMS721 was successful. Now, the patient no longer requires dialysis or transfusions.

“This patient had severe TMA that I believe would have caused her death,” Dr Zecca said. “Her positive response to OMS721 treatment, both initially and following her virus-induced relapse during tapering, was impressive.”

“The results of OMS721 treatment in this challenge-rechallenge scenario underscore the important effects of the drug. Since the poster was produced, her TMA has remained in remission, and we have been able to discontinue her platelet transfusions. Her rapid response has been heartening, and we all are grateful for this remarkable outcome.”

Photo by Chad McNeeley

MARSEILLE—An investigational drug has successfully treated a severe case of transplant-associated thrombotic microangiopathy (TA-TMA), according to a presentation at the 43rd Annual Meeting of the European Society for Blood and Marrow Transplantation.

The drug is OMS721, a monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system.

The patient received OMS721, which is being developed by Omeros Corporation, under a compassionate-use protocol.

Marco Zecca, MD, of the Fondazione IRCCS Policlinico San Matteo in Italy, and his colleagues provided details on this patient in a poster presented at the meeting (Physician Poster Abstracts-Day 1, abstract A437).

The female patient had undergone a hematopoietic stem cell transplant to treat Diamond‐Blackfan anemia. At age 14, she received a transplant from an HLA-compatible, unrelated donor.

Seven months later, she was diagnosed with TA-TMA. The patient was initially treated with eculizumab but had to stop taking the drug after she developed acute pulmonary edema.

She was then treated with plasma exchange but experienced a TA-TMA relapse at 11 months. The patient was again treated with eculizumab and again had to discontinue the drug after developing acute pulmonary edema.

The patient’s condition continued to worsen, and she soon required hemodialysis 3 times a week as well as daily platelet transfusions.

Dr Zecca requested OMS721 as compassionate-use treatment for the patient, and Omeros complied.

Two months after starting OMS721, the patient was able to discontinue hemodialysis and decrease her platelet transfusion requirements. She did not experience any adverse events related to OMS721.

Recently, the patient’s dose was tapered, but she developed a viral infection that reactivated her TA-TMA.

A return to the original dose of OMS721 was successful. Now, the patient no longer requires dialysis or transfusions.

“This patient had severe TMA that I believe would have caused her death,” Dr Zecca said. “Her positive response to OMS721 treatment, both initially and following her virus-induced relapse during tapering, was impressive.”

“The results of OMS721 treatment in this challenge-rechallenge scenario underscore the important effects of the drug. Since the poster was produced, her TMA has remained in remission, and we have been able to discontinue her platelet transfusions. Her rapid response has been heartening, and we all are grateful for this remarkable outcome.”

Photo by Rhoda Baer

The National Comprehensive Cancer Network® (NCCN®) recently launched the NCCN Radiation Therapy Compendium™, which provides a single access point for NCCN recommendations pertaining to radiation therapy.

The compendium provides guidance on all radiation therapy modalities recommended within NCCN guidelines, including intensity modulated radiation therapy, intra-operative radiation therapy, stereotactic radiosurgery/stereotactic body radiotherapy/stereotactic ablative radiotherapy, image-guided radiotherapy, low dose-rate brachytherapy/high dose-rate brachytherapy, radioisotope, and particle therapy.

“As a single source for all radiation therapy recommendations within the NCCN guidelines, the compendium benefits patients with cancer by assisting providers and payers in making evidence-based treatment and coverage decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

The NCCN Radiation Therapy Compendium™ includes recommendations for the following 24 cancer types:

Acute myeloid leukemia

Anal cancer

B-cell lymphomas

Bladder cancer

Breast cancer

Chronic lymphocytic leukemia/small lymphoblastic lymphoma

Colon cancer

Hepatobiliary cancers

Kidney cancer

Malignant pleural mesothelioma

Melanoma

Multiple myeloma

Neuroendocrine tumors

Non-small cell lung cancer

Occult primary cancer

Pancreatic adenocarcinoma

Penile cancer

Primary cutaneous B-cell lymphomas

Prostate cancer

Rectal cancer

Small cell lung cancer

Soft tissue sarcoma

T-cell lymphomas

Testicular cancer

NCCN said additional cancer types will be published on a rolling basis over the coming months.

Photo by Rhoda Baer

The National Comprehensive Cancer Network® (NCCN®) recently launched the NCCN Radiation Therapy Compendium™, which provides a single access point for NCCN recommendations pertaining to radiation therapy.

The compendium provides guidance on all radiation therapy modalities recommended within NCCN guidelines, including intensity modulated radiation therapy, intra-operative radiation therapy, stereotactic radiosurgery/stereotactic body radiotherapy/stereotactic ablative radiotherapy, image-guided radiotherapy, low dose-rate brachytherapy/high dose-rate brachytherapy, radioisotope, and particle therapy.

“As a single source for all radiation therapy recommendations within the NCCN guidelines, the compendium benefits patients with cancer by assisting providers and payers in making evidence-based treatment and coverage decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

The NCCN Radiation Therapy Compendium™ includes recommendations for the following 24 cancer types:

Acute myeloid leukemia

Anal cancer

B-cell lymphomas

Bladder cancer

Breast cancer

Chronic lymphocytic leukemia/small lymphoblastic lymphoma

Colon cancer

Hepatobiliary cancers

Kidney cancer

Malignant pleural mesothelioma

Melanoma

Multiple myeloma

Neuroendocrine tumors

Non-small cell lung cancer

Occult primary cancer

Pancreatic adenocarcinoma

Penile cancer

Primary cutaneous B-cell lymphomas

Prostate cancer

Rectal cancer

Small cell lung cancer

Soft tissue sarcoma

T-cell lymphomas

Testicular cancer

NCCN said additional cancer types will be published on a rolling basis over the coming months.

Photo by Rhoda Baer

The National Comprehensive Cancer Network® (NCCN®) recently launched the NCCN Radiation Therapy Compendium™, which provides a single access point for NCCN recommendations pertaining to radiation therapy.

The compendium provides guidance on all radiation therapy modalities recommended within NCCN guidelines, including intensity modulated radiation therapy, intra-operative radiation therapy, stereotactic radiosurgery/stereotactic body radiotherapy/stereotactic ablative radiotherapy, image-guided radiotherapy, low dose-rate brachytherapy/high dose-rate brachytherapy, radioisotope, and particle therapy.

“As a single source for all radiation therapy recommendations within the NCCN guidelines, the compendium benefits patients with cancer by assisting providers and payers in making evidence-based treatment and coverage decisions,” said Robert W. Carlson, MD, chief executive officer of NCCN.

The NCCN Radiation Therapy Compendium™ includes recommendations for the following 24 cancer types:

Acute myeloid leukemia

Anal cancer

B-cell lymphomas

Bladder cancer

Breast cancer

Chronic lymphocytic leukemia/small lymphoblastic lymphoma

Colon cancer

Hepatobiliary cancers

Kidney cancer

Malignant pleural mesothelioma

Melanoma

Multiple myeloma

Neuroendocrine tumors

Non-small cell lung cancer

Occult primary cancer

Pancreatic adenocarcinoma

Penile cancer

Primary cutaneous B-cell lymphomas

Prostate cancer

Rectal cancer

Small cell lung cancer

Soft tissue sarcoma

T-cell lymphomas

Testicular cancer

NCCN said additional cancer types will be published on a rolling basis over the coming months.

AML cells

The US Food and Drug Administration (FDA) has granted annamycin orphan designation for the treatment of acute myeloid leukemia (AML).

Annamycin is a liposomal anthracycline under development by Moleculin Biotech, Inc.

The company said it is working with the FDA on an investigative new drug application for a phase 1/2 trial of annamycin in patients with relapsed or refractory AML.

Annamycin has already been tested in 6 clinical trials, 3 of which focused on leukemia.

Results from one of these trials, in adults with relapsed/refractory acute lymphoblastic leukemia, were published in Clinical Lymphoma, Myeloma & Leukemia in 2013.

Annamycin has been under development by several other pharmaceutical companies. Moleculin Biotech, Inc. acquired rights and development assets relating to the drug in 2015.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

AML cells

The US Food and Drug Administration (FDA) has granted annamycin orphan designation for the treatment of acute myeloid leukemia (AML).

Annamycin is a liposomal anthracycline under development by Moleculin Biotech, Inc.

The company said it is working with the FDA on an investigative new drug application for a phase 1/2 trial of annamycin in patients with relapsed or refractory AML.

Annamycin has already been tested in 6 clinical trials, 3 of which focused on leukemia.

Results from one of these trials, in adults with relapsed/refractory acute lymphoblastic leukemia, were published in Clinical Lymphoma, Myeloma & Leukemia in 2013.

Annamycin has been under development by several other pharmaceutical companies. Moleculin Biotech, Inc. acquired rights and development assets relating to the drug in 2015.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

AML cells

The US Food and Drug Administration (FDA) has granted annamycin orphan designation for the treatment of acute myeloid leukemia (AML).

Annamycin is a liposomal anthracycline under development by Moleculin Biotech, Inc.

The company said it is working with the FDA on an investigative new drug application for a phase 1/2 trial of annamycin in patients with relapsed or refractory AML.

Annamycin has already been tested in 6 clinical trials, 3 of which focused on leukemia.

Results from one of these trials, in adults with relapsed/refractory acute lymphoblastic leukemia, were published in Clinical Lymphoma, Myeloma & Leukemia in 2013.

Annamycin has been under development by several other pharmaceutical companies. Moleculin Biotech, Inc. acquired rights and development assets relating to the drug in 2015.

About orphan designation

The FDA grants orphan designation to drugs and biologics intended to treat, diagnose, or prevent rare diseases/disorders affecting fewer than 200,000 people in the US.

Orphan designation provides companies with certain incentives to develop products for rare diseases. This includes a 50% tax break on research and development, a fee waiver, access to federal grants, and 7 years of market exclusivity if the product is approved.

Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

©SolStock/iStock

dwatson@frontlinemedcom.com
 

Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

©SolStock/iStock

dwatson@frontlinemedcom.com
 

Overweight or obese children in a cohort study were more likely to have adult nonalcoholic fatty liver disease (NAFLD) and elevated levels of the liver enzyme alanine aminotransferase than were healthy weight children of both sexes, but this association can be mitigated by weight loss in adulthood.

“These findings underscore the importance of both early prevention and lifelong treatment of overweight and obesity to reduce the risk of adverse liver outcome in adulthood,” Yinkun Yan, PhD, an epidemiologist at the Capital Institute of Pediatrics, Beijing, and associates wrote (Pediatrics. 2017;139[4]:e20162738).

©SolStock/iStock

dwatson@frontlinemedcom.com
 

CHICAGO – Guidelines for the management of splanchnic artery aneurysms have been hard to come by because of their rarity, but investigators at Massachusetts General Hospital and Harvard Medical School, both in Boston, have surveyed their 20-year experience to conclude that surveillance is appropriate for most cases of aneurysms smaller than 25 mm, and selective open or endovascular repair is indicated for larger lesions, depending on their location.

“Most of the small splanchnic artery aneurysms (SAAs) of less than 25 mm did not grow or rupture over time and can be observed with axial imaging every 3 years,” Mark F. Conrad, MD, reported at a symposium on vascular surgery sponsored by Northwestern University.

CHICAGO – Guidelines for the management of splanchnic artery aneurysms have been hard to come by because of their rarity, but investigators at Massachusetts General Hospital and Harvard Medical School, both in Boston, have surveyed their 20-year experience to conclude that surveillance is appropriate for most cases of aneurysms smaller than 25 mm, and selective open or endovascular repair is indicated for larger lesions, depending on their location.

“Most of the small splanchnic artery aneurysms (SAAs) of less than 25 mm did not grow or rupture over time and can be observed with axial imaging every 3 years,” Mark F. Conrad, MD, reported at a symposium on vascular surgery sponsored by Northwestern University.

CHICAGO – Guidelines for the management of splanchnic artery aneurysms have been hard to come by because of their rarity, but investigators at Massachusetts General Hospital and Harvard Medical School, both in Boston, have surveyed their 20-year experience to conclude that surveillance is appropriate for most cases of aneurysms smaller than 25 mm, and selective open or endovascular repair is indicated for larger lesions, depending on their location.

“Most of the small splanchnic artery aneurysms (SAAs) of less than 25 mm did not grow or rupture over time and can be observed with axial imaging every 3 years,” Mark F. Conrad, MD, reported at a symposium on vascular surgery sponsored by Northwestern University.

ORLANDO—Patients with multiple sclerosis (MS) have a 14-year reduction in median life expectancy from onset of the disease, compared with the general population, according to a 60-year Norwegian study presented at the ACTRIMS 2017 Forum. The standardized mortality ratio (SMR) may be nearly threefold higher in patients with MS, compared with controls, and sevenfold higher in patients who are the youngest at onset.

Approximately 1,388 patients with MS with onset between 1953 and 2012 in Hordaland County, Western Norway, participated in the study. Dr. Grytten and colleagues obtained information from patient records at Haukeland University Hospital, and it was linked to the Causes of Death Registry. They estimated survival from disease onset, adjusted for sex, age, and disease course, using the Kaplan-Meier analyses. In addition, researchers used the SMR to examine mortality and causes of death in MS.

In all, 291 patients died, primarily from MS. The median time to death from MS onset was 41 years for all patients, compared with 55 years for the general population. Women with MS had a 43-year median life expectancy from onset, compared with 56 years in the general female population. Men had a 36-year median life expectancy from MS onset, compared with 50 years in the general male population. Patients with relapsing-remitting MS had a 43-year median life expectancy from onset, and individuals with primary progressive MS had a 26-year median life expectancy from onset.

The SMR was 2.7 in the total MS population. SMR was 2.4 in patients with relapsing-remitting MS and 3.9 in patients with primary progressive MS. In addition, SMR was 2.9 in women with MS and 2.5 in men with MS. Patients with onset at age 20 or younger had an SMR of 7.3. For patients age 21 to 30 at onset, SMR was 4.0. SMR was 2.6 for patients age 31 to 40 at onset. SMR was 1.3 in patients who were age 60 or older at onset.

SMR from disease onset during the period from 1953 to 1974 was 3.1. SMR from disease onset between 1975 and 1996 was 2.6, and SMR from disease onset between 1997 and 2012 was 0.7. Among causes of death, the SMR for cancer was 5.4, that for respiratory causes and infections was 4.5, and that for coronary and cerebrovascular causes was 3.28. The SMR of causes of death indicated serious comorbid disease in MS, said the investigators.

Female patients with MS had a longer median time to death, compared with male patients, but had a higher risk of dying than did the general population. Investigators also observed a decrease in mortality throughout the study, which might be attributed to environmental factors such as lifestyle and treatment.

—Erica Tricarico

Suggested Reading

Grytten Torkildsen N, Lie SA, Aarseth JH, et al. Survival and cause of death in multiple sclerosis: results from a 50-year follow-up in Western Norway. Mult Scler. 2008; 14(9):1191-1198.

ORLANDO—Patients with multiple sclerosis (MS) have a 14-year reduction in median life expectancy from onset of the disease, compared with the general population, according to a 60-year Norwegian study presented at the ACTRIMS 2017 Forum. The standardized mortality ratio (SMR) may be nearly threefold higher in patients with MS, compared with controls, and sevenfold higher in patients who are the youngest at onset.

Approximately 1,388 patients with MS with onset between 1953 and 2012 in Hordaland County, Western Norway, participated in the study. Dr. Grytten and colleagues obtained information from patient records at Haukeland University Hospital, and it was linked to the Causes of Death Registry. They estimated survival from disease onset, adjusted for sex, age, and disease course, using the Kaplan-Meier analyses. In addition, researchers used the SMR to examine mortality and causes of death in MS.

In all, 291 patients died, primarily from MS. The median time to death from MS onset was 41 years for all patients, compared with 55 years for the general population. Women with MS had a 43-year median life expectancy from onset, compared with 56 years in the general female population. Men had a 36-year median life expectancy from MS onset, compared with 50 years in the general male population. Patients with relapsing-remitting MS had a 43-year median life expectancy from onset, and individuals with primary progressive MS had a 26-year median life expectancy from onset.

The SMR was 2.7 in the total MS population. SMR was 2.4 in patients with relapsing-remitting MS and 3.9 in patients with primary progressive MS. In addition, SMR was 2.9 in women with MS and 2.5 in men with MS. Patients with onset at age 20 or younger had an SMR of 7.3. For patients age 21 to 30 at onset, SMR was 4.0. SMR was 2.6 for patients age 31 to 40 at onset. SMR was 1.3 in patients who were age 60 or older at onset.

SMR from disease onset during the period from 1953 to 1974 was 3.1. SMR from disease onset between 1975 and 1996 was 2.6, and SMR from disease onset between 1997 and 2012 was 0.7. Among causes of death, the SMR for cancer was 5.4, that for respiratory causes and infections was 4.5, and that for coronary and cerebrovascular causes was 3.28. The SMR of causes of death indicated serious comorbid disease in MS, said the investigators.

Female patients with MS had a longer median time to death, compared with male patients, but had a higher risk of dying than did the general population. Investigators also observed a decrease in mortality throughout the study, which might be attributed to environmental factors such as lifestyle and treatment.

—Erica Tricarico

Suggested Reading

Grytten Torkildsen N, Lie SA, Aarseth JH, et al. Survival and cause of death in multiple sclerosis: results from a 50-year follow-up in Western Norway. Mult Scler. 2008; 14(9):1191-1198.

ORLANDO—Patients with multiple sclerosis (MS) have a 14-year reduction in median life expectancy from onset of the disease, compared with the general population, according to a 60-year Norwegian study presented at the ACTRIMS 2017 Forum. The standardized mortality ratio (SMR) may be nearly threefold higher in patients with MS, compared with controls, and sevenfold higher in patients who are the youngest at onset.

Approximately 1,388 patients with MS with onset between 1953 and 2012 in Hordaland County, Western Norway, participated in the study. Dr. Grytten and colleagues obtained information from patient records at Haukeland University Hospital, and it was linked to the Causes of Death Registry. They estimated survival from disease onset, adjusted for sex, age, and disease course, using the Kaplan-Meier analyses. In addition, researchers used the SMR to examine mortality and causes of death in MS.

In all, 291 patients died, primarily from MS. The median time to death from MS onset was 41 years for all patients, compared with 55 years for the general population. Women with MS had a 43-year median life expectancy from onset, compared with 56 years in the general female population. Men had a 36-year median life expectancy from MS onset, compared with 50 years in the general male population. Patients with relapsing-remitting MS had a 43-year median life expectancy from onset, and individuals with primary progressive MS had a 26-year median life expectancy from onset.

The SMR was 2.7 in the total MS population. SMR was 2.4 in patients with relapsing-remitting MS and 3.9 in patients with primary progressive MS. In addition, SMR was 2.9 in women with MS and 2.5 in men with MS. Patients with onset at age 20 or younger had an SMR of 7.3. For patients age 21 to 30 at onset, SMR was 4.0. SMR was 2.6 for patients age 31 to 40 at onset. SMR was 1.3 in patients who were age 60 or older at onset.

SMR from disease onset during the period from 1953 to 1974 was 3.1. SMR from disease onset between 1975 and 1996 was 2.6, and SMR from disease onset between 1997 and 2012 was 0.7. Among causes of death, the SMR for cancer was 5.4, that for respiratory causes and infections was 4.5, and that for coronary and cerebrovascular causes was 3.28. The SMR of causes of death indicated serious comorbid disease in MS, said the investigators.

Female patients with MS had a longer median time to death, compared with male patients, but had a higher risk of dying than did the general population. Investigators also observed a decrease in mortality throughout the study, which might be attributed to environmental factors such as lifestyle and treatment.

—Erica Tricarico

Suggested Reading

Grytten Torkildsen N, Lie SA, Aarseth JH, et al. Survival and cause of death in multiple sclerosis: results from a 50-year follow-up in Western Norway. Mult Scler. 2008; 14(9):1191-1198.

PHILADELPHIA – Particularly in adults, there are three conditions that produce symptoms consistent with idiopathic gastroparesis and should be specifically considered in a detailed history conducted before advanced diagnostic tests, according to a clinical update at the Fourth Annual Digestive Diseases: New Advances meeting, held by Rutgers, the State University of New Jersey, and Global Academy for Medical Education.

The three disorders “are very important, because we see them missed all the time,” reported Anthony J. Lembo, MD, director of the GI Motility Laboratory, Beth Israel Deaconess Medical Center, Boston.

PHILADELPHIA – Particularly in adults, there are three conditions that produce symptoms consistent with idiopathic gastroparesis and should be specifically considered in a detailed history conducted before advanced diagnostic tests, according to a clinical update at the Fourth Annual Digestive Diseases: New Advances meeting, held by Rutgers, the State University of New Jersey, and Global Academy for Medical Education.

The three disorders “are very important, because we see them missed all the time,” reported Anthony J. Lembo, MD, director of the GI Motility Laboratory, Beth Israel Deaconess Medical Center, Boston.

PHILADELPHIA – Particularly in adults, there are three conditions that produce symptoms consistent with idiopathic gastroparesis and should be specifically considered in a detailed history conducted before advanced diagnostic tests, according to a clinical update at the Fourth Annual Digestive Diseases: New Advances meeting, held by Rutgers, the State University of New Jersey, and Global Academy for Medical Education.

The three disorders “are very important, because we see them missed all the time,” reported Anthony J. Lembo, MD, director of the GI Motility Laboratory, Beth Israel Deaconess Medical Center, Boston.