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17-AAG Treatment of Diseases Sensitive to c-KIT Down Regulation

This invention describes the use of 17-allylamino-17-demethoxygeldanamycin (17-AAG), a derivative of geldanamycin, which inhibits mutated KIT protein kinase activity (the product of proto-oncogene c-KIT). This kinase has been identified as the protein responsible for transformation of certain human cell types into pathologic cells. The invention is predicated on the discovery of a new method of inhibiting the activity of a mutated, constitutively active form of the tyrosine kinase, KIT. The method involves the administration of 17-AAG to a cell expressing the mutated KIT protein, whereby the activity level of KIT in the cell is reduced. The invention may prove to be useful for treating diseases such as mastocytosis, gastrointestinal stromal tumors (GIST), mast cell leukemia, myelogenenous leukemia, and testicular cancer, all of which are associated with mutations in the c-KIT proto-oncogene. Gerard Fumo(NCI)➽ more inventions... Leonard Neckers (NCI)➽ more inventions... US Application No. PCT/US03/31962 Admin. Licensing Specialist (ALS), Email: Phone: E-256-2002/0 Updated:May 1, 2003
Leonard Neckers
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