Molecular Mechanisms and Targeted Therapies for Acute Myeloid Leukemia

YANG Xue^1,2, WANG Jianxiang¹*

(¹State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China; ²Department of Hematology, Zhongshan Hospital, Fudan University, Shanghai 200030, China))

Abstract:

AML (acute myeloid leukemia) is a group of heterogeneous hematopoietic malignancy. Multiple mechanisms involving both genetics and epigenetics may contribute to leukemogenesis. APL (acute promyelocytic leukemia) is a distinct subtype of AML. The advent of ATRA (all-trans retinoic acid) synergized with ATO (arsenic trioxide) turned most APL from highly fatal to highly curable, making it the most successful paradigm for targeted therapy in leukemia. The standard therapy for AML remained nearly unchanged over the past four decades. Recently, with the advent of novel targeted drugs, the treatment of AML has been increasingly promising. Here, this review briefly introduced the etiology and pathogenesis of leukemia, focusing on molecular mechanism and targeted therapy for APL, and finally put forward major advances in promoting the precision therapy for AML.

CSTR: 32200.14.cjcb.2022.01.0023