A study on ensuring the quality and safety of pharmaceuticals and medical devices derived from processing of autologous human induced pluripotent stem(-like) cells
A study on ensuring the quality and safety of pharmaceuticals and medical devices derived from processing of autologous human induced pluripotent stem(-like) cells
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DOI：
10.1016/j.reth.2015.06.002
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发表时间：
2015-12-01
影响因子：
4.3
通讯作者：
Yamato, Masayuki
中科院分区：
文献类型：
--
作者：
Hayakawa, Takao;Aoi, Takashi;Yamato, Masayuki
作为一系列努力，为使用人类干细胞产品的再生医学的健康发展制定合适的措施，我们研究了科学原理，概念和基本技术要素，以确保来自自体人类iPS细胞或iPS细胞样细胞的治疗产品的质量和安全性，考虑到科学和技术进步，伦理学，监管原理，和人类干细胞衍生产品的国际趋势。这导致日本厚生劳动省药品和食品安全局于2012年9月7日发布的第0907-4号日本官方通知“关于确保来自自体诱导多能干（样）细胞加工的药品和医疗器械的质量和安全性的指导原则”的制定。本文件解决了来自自体人iPS细胞（或iPS细胞样细胞）的产品的各个方面，除了之前描述的基于自体人干细胞的产品需要考虑的类似点。主要的附加点包括（1）可能存在在特定生物学特征方面不同于iPS细胞的自体人iPS细胞样细胞;（2）在必要和重要的情况下，自体人iPS（样）细胞用作再生医学的适当起始材料;（3）自体人iPS（样）细胞系的建立及其表征;（4）细胞建库和/或在生产过程的适当阶段（如有必要）可能建立源自自体人iPS（样）细胞的中间细胞系;以及（5）对最终产品中存在未分化细胞的担忧;此类细胞可能导致异位组织形成和/或肿瘤发生。本指南的最终目的是尽快为患有常规方式难以治疗的严重疾病的患者提供合适的医疗机会。(C)2015年，日本再生医学会。制作和主办：Elsevier B. V.
As a series of endeavors to establish suitable measures for the sound development of regenerative medicine using human stem cell-based products, we studied scientific principles, concepts, and basic technical elements to ensure the quality and safety of therapeutic products derived from autologous human iPS cells or iPS cell-like cells, taking into consideration scientific and technological advances, ethics, regulatory rationale, and international trends in human stem cell-derived products. This led to the development of the Japanese official Notification No. 0907-4, "Guideline on Ensuring the Quality and Safety of Pharmaceuticals and Medical Devices Derived from the Processing of Autologous Human Induced Pluripotent Stem(-Like) Cells," issued by Pharmaceuticals and Food Safety Bureau, Ministry of Health, Labour and Welfare of Japan, on September 7, 2012. The present paper addresses various aspects of products derived from autologous human iPS cells (or iPS cell-like cells), in addition to similar points to consider that are described previously for autologous human stem cell-based products. Major additional points include (1) possible existence of autologous human iPS cell-like cells that are different from iPS cells in terms of specific biological features; (2) the use of autologous human iPS(-like) cells as appropriate starting materials for regenerative medicine, where necessary and significant; (3) establishment of autologous human iPS(-like) cell lines and their characterization; (4) cell banking and/or possible establishment of intermediate cell lines derived from autologous human iPS(-like) cells at appropriate stage(s) of a manufacturing process, if necessary; and (5) concerns about the presence of undifferentiated cells in the final product; such cells may cause ectopic tissue formation and/or tumorigenesis. The ultimate goal of this guidance is to provide suitable medical opportunities as soon as possible to the patients with severe diseases that are difficult to treat with conventional modalities. (C) 2015, The Japanese Society for Regenerative Medicine. Production and hosting by Elsevier B.V.