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OXY-1 is a small molecule mitochondrial uncoupler and a derivative of niclosamide ethanolamine (NEN). It is being investigated for its potential to treat and prevent hepatic metastasis in colon and pancreatic cancers by targeting cancer cell metabolism. By promoting mitochondrial uncoupling, OXY-1 increases the flux of pyruvate into the mitochondria, antagonizing the Warburg effect (aerobic glycolysis) and leading to the complete oxidation of glucose. This process activates AMP-activated protein kinase (AMPK) and reduces the NADPH/NADP ratio, which in turn inhibits cell proliferation, induces cell cycle arrest, and reduces cell migration and clonogenicity. Preclinical studies in mouse models have demonstrated that oral administration of OXY-1 can significantly reduce tumor size and incidence in the liver.
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