Mutant IDH1 Inhibitors Useful for Treating Cancer
Lead Inventor: Matthew Boxer (NCATS)
Ref. No.: E-189-2016
Abstract: Mutations in isocitrate dehydrogenase 1 (IDH1), an enzyme whose normal function is to convert isocitrate to α-ketoglutarate, are common in a variety of cancers, including acute myeloid leukemia, glioma, cholangiocarcinoma, chondrosarcoma and melanoma. These IDH1 mutants catalyze the production of 2-hydroxyglutarate, an oncometabolite that has been shown to contribute to epigenetic dysregulation in cancer cells. Small molecule inhibitors of several cancer-specific mutant isoforms of IDH1 are available for licensing.
Biochemical, Cellular, and Biophysical Characterization of a Potent Inhibitor of Mutant Isocitrate Dehydrogenase IDH1 • Journal of Biological Chemistry • May 16, 2014 • NCATS Chemical Genomics Center