The safety and antitumor roles of APR-246 were already investigated in a phase I/IIa clinical trial of patients with leukemia, lymphoma or prostate cancer and presented a favorable toxicity profile in patients with p53 mutation, while no clinical response was observed in patients with wild-type p53, according to the predefined response criteria [47, 48], which nicely corroborates the mechanism proposed here and its relevance for future clinical approaches. The gene discussed is TP53; the disease is leukemia.