The safety and efficacy of Enasidenib demonstrated in a phase 1/2 trial in IDH2-mutant advanced myeloid malignancies (including AML and MDS) paved the way for FDA approval of the first small molecule mutIDH inhibitor in cancer and propelled global clinical research efforts for IDH-based targeted cancer treatments (ClinicalTrials.gov NCT01915498) (12). This evidence concerns the gene IDH1 and myelodysplastic syndrome.