Ongoing clinical trials evaluate the efficacy of IDH1/2 inhibitors in MDS and AML-MRC patients (NCT03503409, NCT04603001, NCT04493164, NCT03471260) and their combination with venetoclax, azacitidine and intensive chemotherapy (NCT03471260, NCT03839771). The gene discussed is IDH1; the disease is myelodysplastic syndrome.