Damiani Daniela, Tiribelli Mario
Division of Hematology and Stem Cell Transplantation, Udine University Hospital, 33100 Udine, Italy.
Department of Medicine, University of Udine, 33100 Udine, Italy.
Int J Mol Sci. 2025 May 10;26(10):4571. doi: 10.3390/ijms26104571.
Monoclonal antibodies targeting specific cell surface antigens have emerged as a promising therapeutic approach for acute myeloid leukemia (AML), thus widening the treatment landscape of this heinous disease. These antibodies have been designed to selectively target and eliminate leukemic cells while limiting damage to the normal hematopoietic counterpart. Among the potential targets on AML cells, CD33, CD123, and CD47 have shown the major potential in preclinical and clinical trials. Additionally, conjugation of monoclonal antibodies with cytotoxic agents has further enhanced their therapeutic efficacy. Nonetheless, challenges such as antigen heterogeneity, resistance mechanisms, and the immunosuppressive tumor microenvironment remain significant barriers to achieving durable remission in AML patients. This review explores the mechanisms of action, current clinical developments, and ongoing trials into the role of monoclonal antibodies in AML, highlighting their potential to improve clinical outcomes when used alone or in combination with conventional therapies, making them thus able to become, in the near future, a cornerstone in the treatment of AML.
靶向特定细胞表面抗原的单克隆抗体已成为治疗急性髓系白血病(AML)的一种有前景的治疗方法,从而拓宽了这种恶性疾病的治疗前景。这些抗体被设计用于选择性地靶向和消除白血病细胞,同时限制对正常造血细胞的损害。在AML细胞的潜在靶点中,CD33、CD123和CD47在临床前和临床试验中显示出主要潜力。此外,单克隆抗体与细胞毒性药物的偶联进一步提高了它们的治疗效果。尽管如此,诸如抗原异质性、耐药机制和免疫抑制性肿瘤微环境等挑战仍然是AML患者实现持久缓解的重大障碍。本综述探讨了单克隆抗体在AML中的作用机制、当前临床进展和正在进行的试验,强调了它们单独使用或与传统疗法联合使用时改善临床结果的潜力,使其在不久的将来能够成为AML治疗的基石。