New Drug Targets Three Kinds of Leukemia

Just three years after discovering a genetic mutation that causes a trio of leukemias, Howard Hughes Medical Institute (HHMI) researchers have helped move a new leukemia drug into clinical trials.

The Food and Drug Administration approved human clinical trials of the drug based on strong preclinical data and additional studies in mice showing that the drug eliminates clinical manifestation of the leukemias without any significant toxicity. Some of the data that laid the foundation for the clinical trials are now being reported in the April 7, 2008, issue of the journal Cancer Cell by D. Gary Gilliland and colleagues at Brigham and Women's Hospital and Harvard Medical School in Boston. Scientists at TargeGen Inc., in San Diego., and the Mayo Clinic are also coauthors of the article.