The key role of IDH mutations in blocking HPC differentiation and promoting leukemic growth was further and more strongly supported by the observation that a small molecule (AGI-6780) acting as a potent and specific inhibitor of mutant IDH2 suppressed the growth of patient-derived leukemia cells and induced cell differentiation of IDH2MUT leukemic cells [18]. This evidence concerns the gene IDH2 and leukemia.