The capacity of the new NPM1 inhibitor to triggerstem cell apoptosis has been translated to ex vivo and in vivo efficacy in models of acute myeloidleukemia (AML).19 Our results support phenotypicdrug discovery as a valuable approach to develop active moleculesin a pathologically relevant model, which together with the validationof the target protein involved in the disease, contributes to enhanceinnovation and deliver truly novel therapeutics for unmet medicalneeds.20,21. The gene discussed is NPM1; the disease is acute myeloid leukemia.