Hotspot genetic alterations that confer the enzymes isocitrate dehydrogenase (IDH) 1 and 2 with neomorphic activity to produce the oncometabolite D-2-hydroxyglutarate are common in several cancer types, including acute myeloid leukaemia, cholangiocarcinoma, chondrosarcoma and glioma. Herein, Pirozzi and Yan describe the current understanding of the biological, pathogenetic and prognostic implications of IDH mutations in these cancers. They also review the available preclinical and clinical data on the various therapeutic strategies that are being pursued for IDH-mutant cancers and discuss whether treatment approaches will converge or be context dependent.
- Christopher J. Pirozzi
- Hai Yan
