摘要
人间充质干细胞因具有来源广泛、无伦理限制、免疫豁免等优势和治疗潜力,成为细胞类产品开发的热点方向,但真正成药过程中存在诸多挑战。基于当下中国监管法规框架,在药品研发和审评方面,间充质干细胞治疗产品从“干细胞移植技术”向“干细胞治疗药品”转化中面临许多问题,特别是来源异质性、差异化生产工艺、质量表征等使研发和监管面临挑战。本文聚焦MSC治疗产品成药性相关研究进展,针对MSC治疗产品在国内外的注册申报现状以及成药性面临的风险与挑战进行综述,并基于技术审评角度探讨克服这些障碍和转化应用的策略,提出相关建议和对策,以期促进此类产品的研发和申报。
Mesenchymal stem cells(MSCs)have become a key focus in the development of cell-based therapeutic products due to their wide availability,lack of ethical constraints,and potential for immune privileges.However,the transition from basic MSC transplantation techniques to fully devel⁃oped therapeutic drugs presents numerous challenges given the current regulatory framework in China and from the perspective of drug development and review.This article summarizes the problems faced in this transition,particularly the challenges posed by theheterogeneity of MSC sources,the complexity of unique manufacturing processes,and the complexities of quality characterization.The article also offers suggestions and countermeasures in the hopes of advancing the research,development,and registration of MSC-based therapeutic products.
作者
韩冬梅
周菂
张毅
魏开坤
HAN Dongmei;ZHOU Di;ZHANG Yi;WEI Kaikun(Center for Drug Evaluation,National Medical Products Administration,Beijing 100076,China;State Key Laboratory of Drug Regulatory Science,Beijing 102629,China;Academy of Military Medical Sciences,Beijing 100850,China)
出处
《中国药理学与毒理学杂志》
北大核心
2025年第4期296-302,共7页
Chinese Journal of Pharmacology and Toxicology
基金
药品监管科学全国重点实验室课题(2023SKLDRS0139)。
关键词
间充质干细胞
成药性
异质性
质量表征
mesenchymal stem cell
drugability
heterogeneity
quality characterization
