嵌合抗原受體（CAR） T細胞（CAR-T）療法是一種創(chuàng)新的免疫療法，通過基因工程技術將T細胞轉化為能夠識別特定腫瘤抗原的細胞。CAR-T療法在治療多種B細胞惡性腫瘤中表現出顯著的療效，尤其是急性淋巴細胞白血病和慢性淋巴細胞白血病。盡管其治療潛力巨大，但也伴隨有細胞因子釋放綜合征（CRS）和免疫效應細胞相關神經毒性綜合征（ICANS）等嚴重不良反應。針對CAR-T細胞的監(jiān)管法規(guī)在全球范圍內逐漸完善，以確保其安全性和有效性，未來的研究方向將集中在優(yōu)化CAR結構、探索新的靶點，以及降低治療成本等方面。從CAR-T細胞療法的機制、風險及臨床監(jiān)管法規(guī)3個方面進行綜述，以期為CAR-T細胞治療的安全性和有效性評估提供新的視角和策略。

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

中國食品藥品檢定研究院學科帶頭人培養(yǎng)基金（2023X3）