IACS-010759 is a highly potent and selective small-molecule complex I inhibitor [28] that impairs proliferation and induces apoptosis in brain tumors, acute myeloid leukemia (AML) [29, 30] and NOTCH1-mutated T-cell acute lymphoblastic leukemia [56] although the first clinical trial has shown limited antitumor activity [57]. The gene discussed is NOTCH1; the disease is T-cell acute lymphoblastic leukemia.