Patients with acute myeloid leukemia and an activating mutation in FLT3 respond to a small-molecule FLT3 tyrosine kinase inhibitor, PKC412
Novartis (Switzerland) · Memorial Sloan Kettering Cancer Center · +2 more institutions
Abstract
Leukemic cells from 30% of patients with acute myeloid leukemia (AML) have an activating mutation in the FLT3 (fms-like tyrosine kinase) gene, which represents a target for drug therapy. We treated 20 patients, each with mutant FLT3 relapsed/refractory AML or high-grade myelodysplastic syndrome and not believed to be candidates for chemotherapy, with an FLT3 tyrosine kinase inhibitor, PKC412 (N-benzoylstaurosporine), at a dose of 75 mg 3 times daily by mouth. The drug was generally well tolerated, although 2 patients developed fatal pulmonary events of unclear etiology. The peripheral blast count decreased by 50% in 14 patients (70%). Seven patients (35%) experienced a greater than 2-log reduction in…
Citation impact
- FWCI
- 20.20
- Percentile
- 100%
- References
- 24
Authors
15- RMRichard M. StoneCorresponding
Novartis (Switzerland), Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Dana-Farber Cancer Institute
- DJDaniel J. DeAngelo
Novartis (Switzerland), Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Dana-Farber Cancer Institute
- VMVirginia M. Klimek
Novartis (Switzerland), Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Dana-Farber Cancer Institute
- IGIlene Galinsky
Novartis (Switzerland), Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Dana-Farber Cancer Institute
- EEEli Estey
Novartis (Switzerland), Memorial Sloan Kettering Cancer Center, The University of Texas MD Anderson Cancer Center, Dana-Farber Cancer Institute
Topics & keywords
- Fms-Like Tyrosine Kinase 3
- Myeloid leukemia
- Tyrosine-kinase inhibitor
- Midostaurin
- Cancer research
- Small molecule
- CD135
- Mutation
- Good health and well-being