Two pivotal clinical trials (NCT03072043 and NCT03588078) demonstrated promising efficacy when APR-246 was combined with azacitidine (the first FDA-approved drug for myelodysplastic syndrome (MDS) in patients with TP53-mutated MDS or acute myeloid leukemia (AML) [36,37]. The gene discussed is TP53; the disease is acute myeloid leukemia.