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 TET2, ASXL1, 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.

中文翻译：

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将急性髓系白血病发病机制的最新进展转化为临床


尽管 FDA 在过去 4 年里批准了美国急性髓系白血病 (AML) 患者的九种新药，但 AML 仍然是血液系统恶性肿瘤中医疗需求未得到满足的一个主要领域。在这篇综述中，我们讨论了有前景的 AML 新型分子靶向方法的开发，包括 menin 抑制、新型 IDH1/2 抑制剂以及针对TET2 、 ASXL1和 RNA 剪接因子突变的临床前方法。此外，我们回顾了通过抗CD47、抗SIRPα和抗TIM-3抗体免疫靶向AML的进展；双特异性和三特异性抗体；以及正在开发的针对 AML 的新细胞疗法。