嵌合抗原受体（CAR） T细胞（CAR-T）疗法是一种创新的免疫疗法，通过基因工程技术将T细胞转化为能够识别特定肿瘤抗原的细胞。CAR-T疗法在治疗多种B细胞恶性肿瘤中表现出显著的疗效，尤其是急性淋巴细胞白血病和慢性淋巴细胞白血病。尽管其治疗潜力巨大，但也伴随有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）等严重不良反应。针对CAR-T细胞的监管法规在全球范围内逐渐完善，以确保其安全性和有效性，未来的研究方向将集中在优化CAR结构、探索新的靶点，以及降低治疗成本等方面。从CAR-T细胞疗法的机制、风险及临床监管法规3个方面进行综述，以期为CAR-T细胞治疗的安全性和有效性评估提供新的视角和策略。

Chimeric antigen receptor (CAR) T-cell therapy is an innovative immunotherapy that genetically engineers T-cells into cells capable of recognizing specific tumor antigens. CAR-T therapy has demonstrated remarkable efficacy in the treatment of a wide range of B-cell malignancies, particularly acute lymphoblastic leukemia and chronic lymphocytic leukemia. Despite its great therapeutic potential, it is accompanied by serious adverse effects such as cytokine release syndrome (CRS) and immune effector cellassociated neurotoxicity syndrome (ICANS). Regulatory regulations for CAR-T cells are gradually being improved globally to ensure their safety and efficacy. Future research will focus on optimizing the structure of CAR, exploring new targets, and reducing the cost of treatment. This paper reviews the mechanisms, risks and clinical regulations of CAR-T cell therapy, with the aim of providing new perspectives and strategies for evaluating the safety and efficacy of CAR-T cell therapy.

R979.1

中国食品药品检定研究院学科带头人培养基金（2023X3）