Optimizing Next-Generation AML Therapy: Activity of Mutant IDH2 Inhibitor AG-221 in Preclinical Models

Cancer Discov. 2017 May;7(5):459-461. doi: 10.1158/2159-8290.CD-17-0270.


<b/> AG-221 or enasidenib is a first-in-class selective inhibitor of mutated isocitrate dehydrogenase 2 (IDH2) with early demonstrated clinical efficacy in acute myeloid leukemia as a single agent, yet with persistence of mutant IDH2 clones. Two articles in this issue of Cancer Discovery provide further insight into the biological activity of AG-221 in promoting differentiation of IDH2-mutant cells and reversing aberrant DNA methylation over time, and demonstrating preclinical activity in combination with a targeted FLT3 kinase inhibitor to eliminate IDH2-mutant clones. Cancer Discov; 7(5); 459-61. ©2017 AACR.See related article by Yen et al., p. 478See related article by Shih et al., p. 494.

Publication types

  • Comment

MeSH terms

  • Aminopyridines
  • Humans
  • Isocitrate Dehydrogenase / antagonists & inhibitors*
  • Leukemia, Myeloid, Acute / genetics
  • Mutation*
  • Triazines


  • Aminopyridines
  • Triazines
  • enasidenib
  • Isocitrate Dehydrogenase