The first-in-human, phase Ib clinical trial, in hematological and prostate cancers (NCT00900614), allowed to estimate the maximal tolerated dose and clinical response was observed in several patients and one patient with TP53-mutated AML showed a reduction of blast percentage from 46% to 26% in the bone marrow [94] (Table 1). The gene discussed is TP53; the disease is acute myeloid leukemia.