人源干细胞产品的药学评价考虑

Considerations on CMC evaluation of human-derived stem cell products

干细胞具有自我更新(self-renewing)和多向分化(multilineage differentiation)潜能,且不同类型的干细胞具有各自不同的优势和局限性,在基础研究和临床应用中成为热点领域。按药品进行研发的人源干细胞产品目前主要来源于成体干细胞、人胚干细胞和诱导多能干细胞三大类。人源干细胞产品复杂多样,在多种疾病治疗中展现出独特的治疗优势。本文总结了人源干细胞产品的特点和最新的研究进展,并根据药品开发和技术评价的规律,围绕生产用原材料、生产工艺、质量研究与控制、稳定性和包装容器密封系统等方面提出现阶段的药学审评考虑和讨论,供业界和监管机构探讨交流,以期能促进此类产品的临床转化和应用。

Stem cells have the potential of self-renewing and multilineage differentiation.Different types of stem cells have different advantages and limitations.It has become a hot topic of basic research and clinical application.Currently human-derived stem cell products to be developed as drugs come mainly from adult stem cells,embryonic stem cells,and induced pluripotent stem cells.Human-derived stem cell products are complex and diverse,showing unique therapeutic advantages in the treatment of a variety of diseases.This paper summarizes the characteristics and the latest research progress of human-derived stem cell products.According to the drug development and evaluation rules,this article brings up some quality assessment concerns and discussions in the major fields,such as raw materials,manufacturing process,quality research and control,stability,packaging container system,for the industry and regulators to discuss and communicate,expecting to promote the clinical transformation and application of this class of products.