The changing molecular landscape of AML and its impact on treatment

Abstract

Acute myeloid leukemia is the primary acute leukemia affecting adults and until recently had very limited available treatment options. We are now seeing development and redevelopment of targeted therapy in acute myeloid leukemia with subsequent improvements in disease control and overall survival. The development of therapy with molecular targets allows patients who are not candidates for chemotherapy to receive treatment. In this review, we discuss the molecular mutations FLT3 and IDH and the molecular target CD33, recognized for having key roles in the development of acute myeloid leukemia. We reference key clinical trials to further discuss the approved treatments for acute myeloid leukemia that target these molecular mutations. We then discuss ongoing research to develop new drugs and new combinations of therapy to overcome resistance mechanisms and further improve patient outcomes.