Novel therapeutic strategies for AML in 2012

During the last four decades, much progress has been made in understanding the molecular pathogenesis of acute myeloid leukemia and in identifying prognostic factors predictive of outcome. However, progress in therapy has been much slower. Since the initial description of the combination of an anthracycline and cytarabine for induction, few major advances have changed the standard of care. Furthermore, these few advances apply to younger patients and those with inherently more favorable disease biology. Intensification of post-remission cytarabine improves the cure rate of patients in first complete remission (CR). Daunorubicin dose intensification improves outcome in younger patients. Finally, allogeneic hematopoietic cell transplantation is an effective strategy for many patients in first CR. The discovery of drugs with novel mechanisms of action which are directly at specific molecular targets is among the most exciting areas of research and holds great promise for the development of effective treatment.