Skip to main content
An official website of the United States government
Government Funding Lapse
Because of a lapse in government funding, the information on this website may not be up to date, transactions submitted via the website may not be processed, and the agency may not be able to respond to inquiries until appropriations are enacted.

The NIH Clinical Center (the research hospital of NIH) is open. For more details about its operating status, please visit cc.nih.gov.

Updates regarding government operating status and resumption of normal operations can be found at opm.gov.

IDH1 inhibitor KY100001

An orally available inhibitor of isocitrate dehydrogenase type 1 (IDH1; IDH-1; IDH1 [NADP+] soluble) mutant forms, with potential antineoplastic activity. Upon administration, IDH1 inhibitor KY100001 specifically binds to and inhibits certain mutant forms of IDH1, thereby inhibiting the formation of the oncometabolite 2-hydroxyglutarate (2HG) from alpha-ketoglutarate (a-KG). This prevents 2HG-mediated signaling and leads to both an induction of cellular differentiation and an inhibition of cellular proliferation in tumor cells expressing IDH1 mutations. IDH1 mutations are highly expressed in certain malignancies; they initiate and drive cancer growth by both blocking cell differentiation and catalyzing the formation of 2HG.
Check for active clinical trials using this agentView this agent in the NCI Thesaurus
Synonym:mutant IDH1 inhibitor KY100001
mutant-selective IDH1 inhibitor KY100001
Code name:KY 100001
KY-100001
KY100001
Search NCI's Drug Dictionary
Please enter up to 30 characters for your search