Acute myeloid leukemia (AML) is a heterogeneous group of hematopoietic malignancies which are characterized by the blockage of hematopoietic cell differentiation with uncontrolled proliferation and/or impaired apoptosis. Over the past 20 years, there has been tremendous progress in the biological, molecular, and cytogenetic aspects of the disease, accompanied by significant advancements in the treatment of AML patients. For example, all-trans retinoic acid (ATRA) and arsenic trioxide (As2O3) have been used clinically for effective treatments of patients with acute promyelocytic leukemia (APL, a unique subtype of AML) through differentiation and/or apoptosis induction. More intriguingly, these active compounds-based chemical biological studies greatly accelerated our understanding on leukemogenesis and targeted therapy of AML patients. Based on some recent findings mainly from our group, this review attempts to summarize the related advances from Chinese researchers.
