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FC531 is a small molecule inhibitor of carbonic anhydrase IX (CA9/CAIX), a transmembrane protein that plays a critical role in maintaining neutral intracellular pH (pHi) under hypoxic stress conditions. Developed by researchers at the Indiana University School of Medicine, FC531 is being investigated as a therapeutic strategy to overcome hypoxia-mediated drug resistance in FLT3/ITD-mutated acute myeloid leukemia (AML). By inhibiting CA9, FC531 induces significant intracellular acidification and apoptosis in leukemic cells residing in hypoxic bone marrow niches. Preclinical studies have demonstrated that FC531 exhibits dose-dependent growth inhibitory effects and acts synergistically with chemotherapy agents such as cytarabine to reduce leukemic burden in xenograft models.
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