Emerging treatments in acute myeloid leukaemia

Acute myeloid leukaemia (AML) is the most common form of leukaemia in young adults. Although 75-85% of patients will achieve complete remission after induction chemotherapy, the long-term survival is still < 50% at 5 years. Chemotherapy has increased in intensity in recent years and is perceived to have reached the limit of toxicity. Allogeneic bone marrow transplantation, which is undoubtedly the most effective way to prevent relapse, may not add substantial survival benefits. Several new pharmacological approaches to the treatment of AML are now becoming available, with various molecular targets identified, including the farnesylation of RAS family proteins and tyrosine kinases involved in signal transduction and epigenetic methylation. More selective delivery of chemotherapeutic agents is also feasible using humanised monoclonal antibodies, with the intriguing possibility of increasing treatment delivery without increasing the toxicity. However, despite the progress in the rational design of drugs in disorders such as chronic myeloid leukaemia, AML lacks a single specific pathognomic genetic event to act as a drug target. This review discusses the drugs presently under investigation in Phase II or Phase III trials in AML.