Preclinical studies have supported the capacity of ivosidenib to exert an inhibitory activity on mutant IDH1-AML cells, as shown by: (i) Low concentrations of ivosidenib are sufficient to inhibit 2-HG levels in mutant IDH1 leukemic cell lines; (ii) treatment of primary mutant IDH1 leukemic blasts with ivosidenib reduces 2-HG production, induces cell differentiation, and reduces cell viability; (iii) in a xenograft model generated with primary human AML cells bearing an IDH1-R132H mutation, ivosidenib markedly decreased 2-HG levels in vivo and induced leukemic cell differentiation [158]. The gene discussed is IDH1; the disease is acute myeloid leukemia.