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Mutant isocitrate dehydrogenase 2 (IDH2, mitochondrial form) is a cancer-associated variant of the metabolic enzyme normally responsible for catalyzing the oxidative decarboxylation of isocitrate to alpha-ketoglutarate in the mitochondrial tricarboxylic acid (TCA) cycle[2][4][7]. Hotspot missense mutations at arginine residues (most commonly R140 or R172) confer a neomorphic (gain-of-function) enzymatic activity, which allows mutant IDH2 to reduce alpha-ketoglutarate to the oncometabolite D-2-hydroxyglutarate (2-HG), leading to competitive inhibition of alpha-ketoglutarate–dependent dioxygenases[2][3]. This disrupts cell differentiation through extensive epigenetic remodeling and metabolic reprogramming, contributing to leukemogenesis and other cancers[2][3][4]. IDH2 mutations are therapeutic targets in several cancers, with specific inhibitors approved for mutant forms in acute myeloid leukemia and under investigation for other malignancies[5]. Elevated D-2-hydroxyglutarate serves as a biomarker for detection and monitoring of IDH2-mutant activity and response to therapy[5][3].
Inhibition of mutant neomorphic activity (blocks reduction of alpha-ketoglutarate to D-2-hydroxyglutarate; lowers 2-HG levels). Reactivation of normal differentiation in leukemia cells.
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