Target intelligence / Profile preview

Isocitrate dehydrogenase [NADP], mitochondrial (mutant) (IDH2 (mutant))

Target
IDH2 (mutant)
Molecular classification
Enzyme, Metabolic enzyme, Mitochondrial protein
01

Overview

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].

Other names
Mutant IDH2IDH2 mutantIsocitrate dehydrogenase 2 mutantIsocitrate dehydrogenase [NADP] 2 mutantMutant Isocitrate dehydrogenase 2
02

Mechanism of action

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.

03

Biological functions

Catalysis of oxidative decarboxylation of isocitrate to alpha-ketoglutarate (wild-type function)Neomorphic activity: reduction of alpha-ketoglutarate to D-2-hydroxyglutarate (mutant function)Energy production/intermediary metabolismRegulation of epigenetic processes
04

Disease associations

CancerAcute myeloid leukemia (AML)GliomaCholangiocarcinomaChondrosarcomaMyelodysplastic syndromes (MDS)Angioimmunoblastic T-cell lymphomaD-2-hydroxyglutaric aciduriaOllier and Maffucci syndromes
05

Safety considerations

Potential for differentiation syndrome when mutant IDH inhibitors are used in leukemiaUnintended inhibition of wild-type IDH2 or off-target mitochondrial effectsAltered intermediary metabolism in non-cancer tissues
06

Interacting drugs

Enasidenib (AG-221)

2 more in the full profile.

07

Biomarkers

D-2-hydroxyglutarate (2-HG; elevated in blood/tissues of patients with IDH2 mutations)Mutant IDH2 gene/protein itself (for diagnostic/predictive use)2-HG levels as a pharmacodynamic biomarker for drug efficacy

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