人间充质干细胞生物学有效性的质量评价

Quality evaluation for biological effectiveness of human mesenchymal stem cells

人间充质干细胞(human mesenchymal stem cells,h MSCs)是一类存在于不同组织间质或血管周围、形态类似于成纤维细胞的成体干细胞。hMSCs具有独特的、与治疗疾病密切相关的生物学有效性(biological effectiveness),因此已成为临床研究中发展最为迅速的一类干细胞。其生物学有效性可归为三大类:多向分化潜能、免疫调控功能和组织再生功能,而每一类别中又含有细化的内容(或质量属性)。生物学有效性是临床前研究阶段hMSCs产品综合质量评价的重要内容,而相关质量评价的目的是用于预测或确保相关产品临床治疗有效性的重要依据。由于如何有效预测干细胞临床治疗的有效性是影响其产品研发的重要瓶颈因素,因此对hMSCs"生物学有效性"的评价在其产品研发中变得非常重要。本文首先总结了目前国际上有关hMSCs生物学有效性相关质量属性的共识,进而提出了一些有效评价hMSCs生物学有效性不同方面的评价技术和评价策略,以帮助建立综合性hMSCs生物学有效性质量评价技术体系。

Human mesenchymal stem cells （hMSCs） is a type of fibroblast-like adult stem cells residing in mesenchymal/stromal or perivascular areas of almost all types of tissues, hMSCs possess unique biological effectiveness contributing to therapeutic effects, thus emerging as the most prevalent cell type among all stem cells undergoing clinical studies. The biological effectiveness of hMSCs can be categorized into three groups, which are the differentiation potential, immunomodulation and tissue regeneration, with each group containing further detailed quality attributes or requirements. As a major part of the quality evaluation and control, the biological effectiveness needs to be thoroughly assessed during preclinical stages of product development to maximally predict and assure clinical efficacy of hMSCs products. Since the assuring of clinical efficacy of stem cell products during preclinical stages has become a bottleneck hindering the product development, it thus makes the effective assessment for biological effectiveness of hMSCs extremely important. In this review, we first summarized the current understanding from the international societies regarding the biological effectiveness of hMSCs, and then proposed the relevant assessment technologies as well as assessment strategies with a goal of establishing a comprehensive quality evaluation system for assessing the biological effectiveness of hMSCs products.