Recent Developments in Differentiation Therapy of Acute Myeloid Leukemia

This is the authors' abstract. We don't add key points for this paper.

Acute myeloid leukemia (AML) is characterized by clonal expansion of myeloid progenitors blocked at various stages of their differentiation process and drugs that bypass this differentiation block are therapeutically efficient, as supported by retinoic acid and arsenic trioxide in acute promyelocytic leukemia. However, the successful application of differentiation therapy to APL was not translated into clinical benefit for other non-APL subtypes of AML, in which intensive chemotherapy regimens represent the standard of care. However, the development of molecular studies has led to the identification of therapeutic targets (such as mutated proteins, deregulated pathways) have led to the generation of a new category of specific pharmacologic agents. Some of these agents, such as inhibitors of mutant isocitrate dehydrogenase (IDH1 and IDH2), Lysine-specific demethylase-1 (LSD1) and Menin have shown the capacity of inducing leukemic cell differentiation and a significant therapeutic efficacy.