Kong Lulu, Xu Kailin, Chen Wei
Department of Hematology, Affiliated Hospital of Xuzhou Medical University, Xuzhou, 221002, Jiangsu, China; Blood Diseases Institute, Xuzhou Medical University, Xuzhou, 221002, Jiangsu, China.
Department of Hematology, Affiliated Hospital of Xuzhou Medical University, Xuzhou, 221002, Jiangsu, China; Blood Diseases Institute, Xuzhou Medical University, Xuzhou, 221002, Jiangsu, China.
Best Pract Res Clin Haematol. 2025 Jun;38(2):101632. doi: 10.1016/j.beha.2025.101632. Epub 2025 May 15.
Chimeric antigen receptor T cell (CAR-T) therapy targeting B-cell maturation antigen (BCMA) has emerged as a novel and effective modality for the treatment of relapsed or refractory multiple myeloma (RRMM), achieving remarkable therapeutic outcomes. However, relapse remains a major problem impeding the long-term efficacy of this therapy, with antigen-negative relapse being a particularly challenging issue. The mechanisms underlying BCMA antigen-negative relapse encompass a spectrum of phenomena, including diminished or lost tumor antigen expression, BCMA shedding, impaired antigen presentation, trogocytosis, antigen mutations, and alternative splicing. To overcome the problem of antigen-negative relapse in BCMA CAR-T therapy, a variety of strategies are being explored. These include dual/multi-specific CAR-T cell therapy, combination therapies with antibody-drug conjugates (ADCs) or bispecific T-cell engagers (BiTEs), integration with hematopoietic stem cell transplantation (HSCT), identification of novel targets, and the development of innovative cell therapies such as CAR-NK and CAR-M (CAR-Macrophage). Additionally, the optimization of CAR-T cells through gene editing technologies to enhance their durability and anti-tumor activity is a burgeoning area of research. In future, targeted BCMA CAR-T therapy is poised to place greater emphasis on individualization and precision medicine, combining multiple therapeutic approaches to reduce the incidence of relapse, thereby improving treatment efficacy and longevity.
靶向B细胞成熟抗原(BCMA)的嵌合抗原受体T细胞(CAR-T)疗法已成为治疗复发或难治性多发性骨髓瘤(RRMM)的一种新型有效方法,取得了显著的治疗效果。然而,复发仍然是阻碍该疗法长期疗效的主要问题,其中抗原阴性复发是一个特别具有挑战性的问题。BCMA抗原阴性复发的机制包括一系列现象,如肿瘤抗原表达减少或丧失、BCMA脱落、抗原呈递受损、细胞噬作用、抗原突变和可变剪接。为了克服BCMA CAR-T疗法中抗原阴性复发的问题,正在探索多种策略。这些策略包括双特异性/多特异性CAR-T细胞疗法、与抗体药物偶联物(ADC)或双特异性T细胞衔接器(BiTE)的联合疗法、与造血干细胞移植(HSCT)相结合、鉴定新靶点以及开发创新的细胞疗法,如CAR-NK和CAR-M(CAR-巨噬细胞)。此外,通过基因编辑技术优化CAR-T细胞以增强其持久性和抗肿瘤活性是一个新兴的研究领域。未来,靶向BCMA的CAR-T疗法有望更加注重个体化和精准医学,结合多种治疗方法以降低复发率,从而提高治疗效果和延长生存期。