Novel therapeutic drugs targeting key molecular defects in acute myeloid leukemia (AML) are being developed, with FMS-like tyrosine kinase 3 (FLT3) representing 1 of the most attractive targets, according to a recent review.

FLT3 mutants resulting from either internal tandem duplication or point mutations possess enhanced kinase activity and cause constitutive activation of signaling. To date, several small molecule inhibitors of FLT3 have been developed. The review focuses on the pathological role of mutant FLT3 in the development of AML, the current status of FLT3 inhibitor development, and mechanisms that cause resistance.