ORLANDO – The oral multikinase inhibitor midostaurin improved overall survival by 23% when added to standard chemotherapy and given as maintenance therapy for 1 year in newly diagnosed patients with FLT3-mutated acute myeloid leukemia (AML) in the global phase III CALGB 10603/RATIFY trial.
The results struck a chord at the annual meeting of the American Society of Hematology because the benefits of targeted therapy have so far eluded AML patients despite transforming the treatment of other blood cancers. Currently, there are no approved, targeted treatments for AML.
CALGB 10603/RATIFY is the first large, controlled trial to show an overall survival benefit in the roughly 30% of AML patients with a mutation in the FLT3 gene.
“This is exciting because we haven’t had a new treatment in AML for 30 years,” Dr. Robert Hromas of the University of Florida, Gainesville, said while moderating a press conference highlighting the plenary abstract.
The results were a decade in the making after midostaurin failed previously when used in all AML patients rather than the subset with the FLT3 mutation. But the persistence of researchers, the international collaboration, and funding in cancer research paid off, Dr. Hromas said.
Study author Dr. Richard M. Stone, chief of staff at Dana-Farber Cancer Institute in Boston, reviewed the results of CALGB 10603/RATIFY in an interview.
CALGB 10603/RATIFY was sponsored by the Cancer Therapy Evaluation Program. Dr. Stone reported financial relationships with several drug companies including Novartis, which provided the study drug and sponsored the trial outside North America. Dr. Hromas disclosed serving as an uncompensated advisory board member without equity for Cloud Pharmaceuticals.