New drugs in the treatment of acute myeloid leukaemia
Purpose of review: In the past few years, research in the underlying pathogenic mechanisms of acute myeloid leukaemia (AML) has led to challenging advances in our understanding of the disease. Cytogenetic and molecular aberrations are the most important factors in determining response to chemotherapy as well as long-term outcome and beyond prognostication are potential therapeutic targets. Increased understanding of the pathogenesis of AML spurred the development of new substances in the treatment of AML, especially the creation of small molecules that target the disease on a molecular level. Recent findings: Various new agents are currently investigated within clinical trials. Actually highest response rates are achieved when new molecularly targeted therapies (e.g. tyrosine kinase inhibitors, farnesyltransferase inhibitors, multidrug resistance inhibitors) are combined with standard chemotherapy. Summary: Th e progress of unravelling the pathogenesis in AML and of the discovery of new molecular markers have increased our understanding of leukaemogenesis. Current clinical research focuses to a greater extent on a genotypespecific treatment strategy that may offer a higher grade of effectiveness of the administered therapy and is hoped to be accompanied by a lesser extent of toxicity. Finally, these results
are expected to translate into an increase in cure rates with improved overall survival. Presented here is an overview of molecularly targeted therapies currently being evaluated in AML, with a focus on tyrosine kinase inhibitors, farnesyltransferase inhibitors, multidrug resistance inhibitors, DNA hypomethylation agents and histone deacetylase inhibitors.