Molecular Genetic Pathways as Therapeutic Targets in Acute Myeloid Leukemia

The heterogeneity of acute myeloid leukemia (AML) results from a complex network of cytogenetic aberrations and molecular mutations. These genetic markers are the basis for the categorization of cases within distinct subgroups and are highly relevant for the prediction of prognosis and for therapeutic decisions in AML. Clinical variances within distinct genetically defined subgroups could in part be linked to the interaction of diverse mutation classes, and the subdivision of normal karyotype AML on the basis of recurrent molecular mutations gains increasing relevance for therapeutic decisions. In parallel to these important insights in the complexity of the genetic networks in AML, a variety of diverse new compounds is being investigated in preclinical and clinical studies. These approaches aim to develop targeted treatment concepts that are based on interference with molecular genetic or epigenetic mechanisms. This review provides an overview on the most relevant genetic markers, which serve as basis for targeted therapy approaches now or might represent options for such approaches in the future, and summarizes recent results of targeted therapy studies.