HMS-101 is a small-molecule inhibitor of mutant IDH1 which reduced 2-HG level, affected proliferation and the ERK (extracellular signal-regulated kinase) signaling pathway, and inhibited colony formation of IDH1-mut murine cells and primary AML cells cultured ex vivo without affecting normal bone marrow cells [77]. Here, IDH1 is linked to acute myeloid leukemia.