Based on the improved understanding of the molecular pathogenesis of AML, new treatment strategies targeting specific molecular defects have been implemented within treatment trials of the German-Austrian AML Study Group (AMLSG), such as FLT3 inhibition in AML with FLT3-ITD (ClinicalTrials.gov Identifier: NCT01477606) [19], KIT-inhibition in CBF-AML (NCT00850382) [17], and the use of gemtuzumab ozogamicin in AML with NPM1 mutations (NCT00893399, EudraCT 2009-011889-28) [18]. The gene discussed is KIT; the disease is acute myeloid leukemia.