TP53 mutant (mTP53) myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) represent a molecular cohort with the poorest outcomes, as explained here by David Sallman, MD, of the Moffitt Cancer Center, Tampa, FL. Hypomethylating agents are the current standard of care for these patients; however, an alternative treatment is needed. Speaking at the American Society of Hematology (ASH) 2018 Annual Meeting and Exposition in San Diego, CA, Dr Sallman discusses the encouraging data from the Phase Ib/II trial of APR-246, a novel, small molecule that selectively induces apoptosis in mTP53 cancer cells, in combination with azacitidine.