肿瘤细胞治疗研究
英文原题：CAR T-cell therapy to treat multiple myeloma: current state and future directions.
嵌合抗原受体（CAR）T 细胞治疗代表了在早期和晚期治疗线中治疗复发或难治性多发性骨髓瘤（MM）的变革性进展。
嵌合抗原受体（CAR）T细胞疗法是治疗早期和晚期复发或难治性多发性骨髓瘤（MM）的变革性进展。MM是一种浆细胞恶性肿瘤，传统治疗通常需要持续、复杂的药物方案，给患者带来较大负担。相比之下，CAR-T细胞疗法提供了一次性治疗选择，无需持续维持治疗。该疗法利用工程化T细胞靶向肿瘤细胞上的特定抗原，从而清除肿瘤。目前获批疗法靶向B细胞成熟抗原（BCMA）；新的靶点也在研究中，例如G蛋白偶联受体C类5组D型（GPRC5D）。尽管疗效显著，CAR-T细胞疗法仍伴随严重毒性，如细胞因子释放综合征（CRS）和免疫效应细胞相关神经毒性综合征（ICANS），需要谨慎管理。本综述概述CAR-T细胞设计与制备、当前FDA适应证，以及CAR-T治疗MM面临的挑战和未来方向。
Chimeric antigen receptor (CAR) T-cell therapy represents a transformative advancement in treating relapsed or refractory multiple myeloma (MM) in both early- and late-line settings. MM, a plasma cell malignancy, traditionally requires ongoing complex drug regimens, posing significant burdens on patients. In contrast, CAR T-cell therapy offers a one-time treatment option without the need for continuous maintenance therapy. CAR T-cell therapy leverages engineered T-cells to target specific antigens on tumor cells, leading to their elimination. Current approved therapies target B-cell maturation antigen (BCMA); new targets are under investigation, such as G-protein-coupled receptor class C group 5 member D (GPRC5D). Despite its efficacy, CAR T-cell therapy is associated with serious toxicities such as cytokine release syndrome (CRS) and immune-effector cell-associated neurotoxicity syndrome (ICANS), necessitating careful management. The review will provide an overview of the design and manufacturing of CAR T-cells and current FDA indications, as well as challenges and future directions of CAR-T therapy for MM treatment.
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