In a phase 2 study of patients with TP53-mutated MDS and AML, APR-246 plus azacitidine had a side-effect profile similar to that which is expected with azacitidine monotherapy and resulted in an overall response rate of 71% with 44% achieving CR and a median OS of 10.8 months (103). The gene discussed is TP53; the disease is acute myeloid leukemia.