嵌合抗原受体T细胞产品非临床安全性评价内容和主要关注的问题探讨

doi:10.19803/j.1672-8629.2022.08.01

中国药物警戒 ›› 2022, Vol. 19 ›› Issue (8): 813-816.
DOI: 10.19803/j.1672-8629.2022.08.01

• CAR-T 细胞非临床安全性研究与评价专栏 • 上一篇下一篇

嵌合抗原受体T细胞产品非临床安全性评价内容和主要关注的问题探讨

黄瑛, 侯田田^Δ, 秦超, 霍艳, 王三龙, 文海若^#, 耿兴超^*

中国食品药品检定研究院国家药物安全评价监测中心,药物非临床安全评价研究北京市重点实验室,北京 100176

收稿日期:2022-04-22 出版日期:2022-08-15 发布日期:2022-08-15
通讯作者: ^*耿兴超,男,博士,研究员,药理毒理。E-mail：gengxch@nifdc.org.cn;^#为共同通信作者。
作者简介:黄瑛,女,博士,副研究员,药理毒理。^Δ为并列第一作者。
基金资助:
国家重点研发计划（2021YFA1101602）; 中国科学院战略先导科技专项（XDA1604050202）

Nonclinical research on CAR-T cell products: main concerns and key issues

HUANG Ying, HOU Tiantian^Δ, QIN Chao, HUO Yan, WANG Sanlong, WEN Hairuo^#, GENG Xingchao^*

National Center for Safety evaluation of Drugs, National Institutes for Food and Drug Control, Key Laboratory of Beijing for Nonclinical Safety Evaluation Research of Drugs, Beijing 100176, China

Received:2022-04-22 Online:2022-08-15 Published:2022-08-15

摘要/Abstract

摘要： 目的探讨嵌合抗原受体T（chimeric antigen receptor T, CAR-T）细胞产品非临床研究的主要内容和关键问题,为CAR-T产品研究和开发提供参考。方法针对CAR-T产品特点,基于对CAR-T细胞产品的评价实践,结合国内外CAR-T细胞非临床研究现状和相关文献报告,对其非临床安全性评价研究内容和主要关注的问题进行综述。结果对CAR-T产品非临床评价中动物模型的选择、药效学评价、毒性研究以及生物分布研究等方面的最新进展和研究进行阐述并提出建议。结论细胞作为一种复杂的新型生物治疗产品,体内使用可能存在安全性风险。在CAR-T细胞非临床研究中,应根据产品特点和具体情况,结合国内外相关标准和技术要求以及发展趋势进行设计和开展试验。

关键词: 嵌合抗原受体T细胞, 非临床研究, 动物模型, 毒性, 安全性, 有效性

Abstract: Objective To explore the main contents and key issues of non-clinical research on chimeric antigen receptor T (CAR-T) cell products, and to provide useful references for the research and development of CAR-T products. Methods According to the characteristics of CAR-T products, based on the evaluation practice of CAR-T cell products in our center, combined with the non-clinical research status of CAR-T cells at home and abroad and relevant literature reports, the research contents and main concerns of non-clinical safety evaluation of CAR-T cells were summarized. Results The latest progress and academic opinions on the selection of animal models, pharmacodynamic evaluation, toxicity study and biological distribution study in the non-clinical evaluation of CAR-T products were expounded and suggestions were put forward. Conclusion CAR-T cells, as a complex new biotherapy product, may have a variety of safety risks when used in vivo. In the non-clinical study of CAR-T cells, the design and test shall be carried out according to the product characteristics and specific conditions, in combination with relevant domestic and foreign standards, technical requirements and development trends.

Key words: chimeric antigen receptor T(CAR-T) cells, nonclinical research, animal models, toxicity, safety, efficacy

中图分类号:

R979.1

黄瑛, 侯田田, 秦超, 霍艳, 王三龙, 文海若, 耿兴超. 嵌合抗原受体T细胞产品非临床安全性评价内容和主要关注的问题探讨[J]. 中国药物警戒, 2022, 19(8): 813-816.

HUANG Ying, HOU Tiantian, QIN Chao, HUO Yan, WANG Sanlong, WEN Hairuo, GENG Xingchao. Nonclinical research on CAR-T cell products: main concerns and key issues[J]. Chinese Journal of Pharmacovigilance, 2022, 19(8): 813-816.

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嵌合抗原受体T细胞产品非临床安全性评价内容和主要关注的问题探讨

Nonclinical research on CAR-T cell products: main concerns and key issues

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