Personalized medicine in hematology using the example of acute myelogenous leukemia

In recent years, advances in molecular genetic characterization of leukemias and lymphomas have started to facilitate targeted therapies. For example, the use of tyrosine kinase inhibitors (TKI) targeting BCR-ABL1 have revolutionized the treatment of chronic myeloid leukemia (CML). In contrast, the treatment of acute myeloid leukemia (AML) has not changed significantly over the last 40 years, but novel insights into leukemia biology have recently started to move things forward. While AML is an extremely heterogeneous disease with outcomes greatly varying according to the AML genotype, recent progress in understanding the biology and the identification of the mutations driving leukemogenesis have opened new avenues for molecular therapeutic approaches. A number of prognostic and predictive molecular markers and pathways have been identified that can serve as therapeutic targets, such as the activation of the fms-like tyrosine kinase 3 (FLT3) receptor tyrosine kinase or aberrant DNA methylation patterns caused by mutations in distinct epigenetic modifiers. In addition, targeted therapy with monoclonal antibodies and novel small molecule kinase inhibitors are promising strategies that might help to further improve the cure rates in AML. In this review, we will highlight recent efforts on targeted therapies in AML, give an outlook on novel and/or upcoming therapeutic options, and briefly summarize current strategies in other hematologic malignancies.