NPC/MIPE Toxicity Screen of IDH1 Inhibitors against Cholangiocarcinoma Cell Lines

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This project aims to repurpose approved drugs and other small molecules with well-characterized mechanisms of action for IDH1 anticancer indications by examining toxicity of these small molecules against ICC mutant cell lines. During this period, the project team screened NCGC's small molecule libraries, and identified a number of hit demonstrating selective toxicity against IDH1 mutant cell lines. Focusing on selected candidates, medicinal chemistry optimization has led to improved physiochemical and therapeutic properties, with the ultimate goal of developing an orally available pre-clinical candidate. Advanced characterization of these molecules is underway to further profile their activity and the mechanism of action responsible for the selective lethality in the context of IDH1 mutant ICC cell lines.