Ivosidenib efficiently killed IDH1R132 mutant cancer cell lines at low nanomolar IC50 in vitro, drastically and rapidly reduced 2-HG levels in a mouse xenograft model derived from HT-1080 fibrosarcoma cell line and led to the differentiation of IDH1-mutated AML cells from patients ex vivo [95]. This evidence concerns the gene IDH1 and acute myeloid leukemia.