Stunning Data for Ambit Biosciences/Astellas Pharma Inc. AML Drug

Published: Dec 11, 2012

The promise shown by Ambit Biosciences' quizartinib, which is partnered with Astellas, as a treatment for acute myeloid leukaemia, has caused a stir at the American Society of Hematology meeting in Atlanta. Final results of a Phase II study of quizartinib were presented which suggest the drug may be an excellent option to treat a subset of patients with treatment-resistant AML. In the study, the majority carried a mutation in a gene called FLT3-ITD and presenting the data at ASH, Mark Levis of Johns Hopkins University, noted that the FLT3 mutation is a "power switch" that leukaemia cells use to spread more aggressively, which helps them to grow back immediately after chemotherapy. He added that the only way to treat this type of mutation "is to find a way to turn the switch off - a feat that has eluded researchers for far too long". While patients with the FLT3-ITD mutation can achieve remission with standard chemotherapy, they tend to relapse very quickly. Furthermore, an important treatment option is a stem cell transplant, but only if the patient is in some form of remission, otherwise, failure rates are high.

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