将急性髓细胞白血病发病机制的最新进展转化为临床实践。
Translating recent advances in the pathogenesis of acute myeloid leukemia to the clinic.
机构信息
Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA.
Human Oncology and Pathogenesis Program, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York 10065, USA.
出版信息
Genes Dev. 2022 Mar 1;36(5-6):259-277. doi: 10.1101/gad.349368.122.
Despite FDA approval of nine new drugs for patients with acute myeloid leukemia (AML) in the United States over the last 4 years, AML remains a major area of unmet medical need among hematologic malignancies. In this review, we discuss the development of promising new molecular targeted approaches for AML, including menin inhibition, novel IDH1/2 inhibitors, and preclinical means to target , , and RNA splicing factor mutations. In addition, we review progress in immune targeting of AML through anti-CD47, anti-SIRPα, and anti-TIM-3 antibodies; bispecific and trispecific antibodies; and new cellular therapies in development for AML.
尽管在过去 4 年里，美国食品药品监督管理局（FDA）批准了 9 种治疗急性髓细胞白血病（AML）的新药，但 AML 仍是血液系统恶性肿瘤领域未满足医疗需求的主要领域。在这篇综述中，我们讨论了 AML 有前途的新型分子靶向方法的发展，包括门冬酰胺酶抑制剂、新型 IDH1/2 抑制剂，以及靶向 、 和 RNA 剪接因子突变的临床前方法。此外，我们还综述了通过抗 CD47、抗 SIRPα 和抗 TIM-3 抗体、双特异性和三特异性抗体，以及 AML 开发中的新型细胞疗法进行 AML 免疫靶向治疗的进展。
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