Discoveries
'Targeted' drug shows promise against form of leukemia
Gleevec, a drug that has made headlines for its effectiveness against chronic myelogenous leukemia, may soon be joined by other medications that work by targeting cancer cells' vulnerabilities.
Senior author James Griffin, MD, and lead author Ellen Weisberg, PhD
A particularly promising one is PKC412, which, in a recent study by Dana-Farber scientists, killed certain acute myelogenous leukemia (AML) cells in both test-tube samples and mice. The drug works by zeroing in on a structure in leukemia cells called the FLT3 tyrosine kinase receptor. When the receptor is defective, as it is in many AML cells, the cells grow out of control and fail to perform normally.
"By finding a drug that inhibits the defective form of FLT3, you can specifically target those leukemia cells," says the study's lead author, Ellen Weisberg, PhD, of Dana-Farber's Medical Oncology Department.
Weisberg and her colleagues, including senior author James Griffin, MD, chair of Medical Oncology, screened dozens of compounds before finding that PKC412 fit the bill. The success of the drug in laboratory and animal studies sets the stage for a clinical trial in which PKC412 is being tested in some patients whose AML has resisted other treatments. The trial is under way at Dana-Farber/Partners CancerCare, Memorial Sloan-Kettering Cancer Center in New York, and M.D. Anderson Cancer Center in Houston, under the direction of DFCI's Richard Stone, MD.
