The perspectives of the targeted therapy in acute myeloid leukemia

Abstract

Results of therapy of acute myeloid leukemia (AML) patients are still unsatisfactory. Since the progress in conventional chemotherapeutics is likely approaching its limits, novel targeted therapies are attractive candidates to improve treatment results. In principle, these drugs must target the critical pathogenetic pathway in the disease. Owing to the large heterogeneity of the disease, it is particularly difficult to identify such universal pathogenetic mechanism in AML. However, in recent years we have witnessed rapid progress in molecular diagnostics, characterization of molecular pathways, signaling cascades and epigenetic changes that shape liczthe leukemic phenotypes and facilitate individualized risk assessment and individualized therapeutic strategy. These studies also characterized certain genetic lesions, whose protein products are targetable. The model example of the effective, targeted treatment in AML is the use of the all-trans retinoid acid in acute promyelocytic leukemia with t(15;17)/PML-RARA. The promising results of studies addressing the role of targeted drugs in the individual subtypes of AML raise hope that these approach will find its place in the clinical practice and improve the results of treatment in AML patients.
Hematologia 2011; 2, 1: 23–32