The drug sorafenib, which is used to treat kidney and liver cancer, may be effective acute myeloid leukemia patients with specific gene mutations.
Previous studies have shown that sorafenib is especially effective against acute myeloid leukemia cells that have certain mutations known as internal tandem duplication (ITD) mutations of the Fms-like tyrosine kinase 3 (FLT3) gene.
Michael Andreeff, M.D., Ph.D., of the University of Texas M. D. Anderson Cancer Center in Houston and colleagues examined sorafenib's ability to kill leukemia cells that express either the common form or mutant copies of FLT3. They also gave sorafenib to mice bearing leukemia cells with known FLT3 gene mutations and to leukemia patients with and without the mutations.
The researchers found that sorafenib slowed growth and induced cell death in the FLT3 mutant leukemia cells and increased survival of the mice with FLT3 mutant leukemia. Sorafenib reduced the percentage of leukemia cells in blood and marrow in patients with this mutation, but not in patients without the mutation.
"Our findings imply that sorafenib is a potent antileukemic agent in patients with FLT3-ITD mutant [acute myeloid leukemia], a form of [the disease] that responds poorly to traditional chemotherapy," the authors write.
This research was recently published in the Journal of the National Cancer Institute.
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