Sorafenib showed a significant antileukemic activity in relapsed or refractory pediatric AML, inducing a reduction by more than 50% of bone marrow blasts and, in combination with clofarabine and cytarabine, it achieved a complete remission in 8 out 12 patients, including both wild type and mutated FLT3 (18). The gene discussed is FLT3; the disease is acute myeloid leukemia.