干细胞来源细胞治疗产品非临床安全性评价概述

Overviews of non-clinical safety evaluation of stem cell-derived cellular therapy products

随着细胞治疗产品研发的快速发展,新的干细胞治疗产品不断被开发,但帮助设计非临床安全性评价研究的相关指导性文件相对不足。干细胞治疗产品不仅来源不同,产品安全性因素也不同,存在异位组织形成、不可控生物分布、免疫原性和成瘤性等潜在风险。不同的干细胞治疗产品给非临床安全性研究带来极大挑战。本文简要概述干细胞的种类及干细胞来源细胞治疗产品非临床安全性评价一般原则,重点关注干细胞来源细胞治疗产品非临床安全性评价中动物选择、试验设计和成瘤性研究等方面,以期为我国干细胞来源细胞治疗产品的非临床安全性评价提供参考。

With the rapid research and development of cellular therapy products,new stem cell-derived cellular therapy products are constantly being developed,but only few relevant guidance documents to assist the design of studies for non-clinical safety evaluation have been published.Stem cell-derived cellular therapy products not only come from different sources,but also have different safety factors and potential risks of ectopic tissue formation,uncontrolled biological distribution,immunogenicity and tumorigenicity,etc.Different stem cell-derived cellular therapy products bring great challenges to non-clinical studies.The paper gives a brief overview of the types of stem cells and general principles for the non-clinical safety evaluation of stem cell-derived cellular therapy products,focusing on the animal selection,experimental design and tumorigenicity study,in order to provide some references for non-clinical safety evaluation of stem cell-derived cellular therapy products in China.