干细胞制剂临床前安全评价方法

Nonclinical safety assessment for stem cell-based medicinal

近年来干细胞技术的迅速发展使得干细胞制剂成为了新的研究热点,干细胞制剂在治疗退化性疾病、癌症以及损伤组织修复上得到了广泛的应用。由于干细胞具有多向分化和增殖能力,因此其临床前安全性需结合临床应用方法及干细胞特点进行综合评价。目前美国及欧洲对于干细胞制剂的临床前安全性评价已建立了较为完善的体系,而国内的评价体系刚刚起步,对干细胞临床使用的安全性还缺乏系统性的评价和规范性的要求。本文就干细胞制剂的特点、国际上对于其临床前安全性评价的要求以及干细胞制剂临床前安全性评价要点做简要介绍和阐述。

Recent breakthroughs in stem cell technology have generated tremendous enthusiasm to explore the stem cell-based medicinal. Stem cell-based medicinal has been considered for the treatment of degenerative dis- ease, cancer and repair of damaged tissue. As the stem cell has the capacity of multi-lineage differentiation and proliferation, the nonclinical safety assessment of stem cell-based medicinal should consider the characteristics of the applied stem cell as well as its proposed clinical application. The evaluation system and criteria of nonclinical safety assessment for stem cell-based medicinal has been developed well in the USA and EU ; however the safety e- valuation system in China has just been set up. The systematic assessment and standard criteria has not been devel- oped and applied for the safety assessment of stem cell-based medicinal in China yet. This article briefly introduced the characteristics and the safety evaluation requirements for sterrL cell-based medicinal as well as the factors that should be considered during the nonclinical safety assessment.