New study validates importance of mutations in specific gene in AML therapy

Scientists at the University of California, San Francisco have discovered that mutations in a gene called FLT3 could hold the key to treating the most common type of leukemia in humans, acute myeloid leukemia (AML). A new study validates certain activating mutations in the gene as targets for AML therapy. “These mutations are critically important for the survival of leukemia cells that harbor them,” Neil Shah, who led the research, said. “Our results also identify drug-resistant mutations in FLT3 that represent high-value targets for future drug development, and will hopefully rekindle interest in developing potent FLT3 inhibitors for the treatment of acute myeloid leukemia.” The study concludes that current drugs that target the FTL3 genes fail not due to a lack of precision, but a lack of power. Working with AC220, the first clinically-active FLT3 inhibitor, they were able to achieve deep remissions in all eight patients who participated, although all eventually relapsed.