In a first-in-human study, p53 was targeted via APR-246, which restores the transcriptional function of mutant p53 in patients with hematologic malignances and prostate cancer; biologic effects such as cell cycle arrest and early signals of apoptosis were observed, and one patient with p53-mutated AML in this study demonstrated a reduced blast percentage [111]. The gene discussed is TP53; the disease is prostate cancer.