Wojcicki Anna V, Kadapakkam Meena, Frymoyer Adam, Lacayo Norman, Chae Hee-Don, Sakamoto Kathleen M
Division of Hematology/Oncology, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.
Division of Neonatal and Developmental Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA 94305, USA.
Cancers (Basel). 2020 Feb 13;12(2):441. doi: 10.3390/cancers12020441.
Acute myeloid leukemia (AML) is a clinically and genetically heterogenous malignancy of myeloid progenitor cells that affects patients of all ages. Despite decades of research and improvement in overall outcomes, standard therapy remains ineffective for certain subtypes of AML. Current treatment is intensive and leads to a number of secondary effects with varying results by patient population. Due to the high cost of discovery and an unmet need for new targeted therapies that are well tolerated, alternative drug development strategies have become increasingly attractive. Repurposing existing drugs is one approach to identify new therapies with fewer financial and regulatory hurdles. In this review, we provide an overview of previously U.S. Food and Drug Administration (FDA) approved non-chemotherapy drugs under investigation for the treatment of AML.
急性髓系白血病（AML）是一种髓系祖细胞的临床和基因异质性恶性肿瘤，可影响各年龄段的患者。尽管经过数十年的研究以及总体疗效有所改善，但标准疗法对某些AML亚型仍然无效。目前的治疗强度大，会导致多种副作用，不同患者群体的反应各异。由于新药研发成本高昂，且对耐受性良好的新型靶向疗法存在未满足的需求，替代药物研发策略变得越来越有吸引力。重新利用现有药物是一种识别新疗法的方法，其面临的财务和监管障碍较少。在本综述中，我们概述了美国食品药品监督管理局（FDA）此前批准的正在研究用于治疗AML的非化疗药物。
