Mah Nancy, Seltmann Stefanie, Aran Begoña, Steeg Rachel, Dewender Johannes, Bultjer Nils, Veiga Anna, Stacey Glyn N, Kurtz Andreas
Berlin-Brandenburger Centrum für Regenerative Therapien (BCRT), Charité - Universitätsmedizin Berlin, Berlin, Germany.
Berlin-Brandenburger Centrum für Regenerative Therapien (BCRT), Charité - Universitätsmedizin Berlin, Berlin, Germany.
Stem Cell Res. 2020 Jun 27;47:101887. doi: 10.1016/j.scr.2020.101887.
The value of human pluripotent stem cells (hPSC) in regenerative medicine has yet to reach its full potential. The road from basic research tool to clinically validated PSC-derived cell therapy products is a long and winding one, leading researchers, clinicians, industry and regulators alike into undiscovered territory. All stakeholders must work together to ensure the development of safe and effective cell therapies. Similarly, utilization of hPSC in meaningful and controlled disease modeling and drug screening applications requires information on the quality and suitability of the applied cell lines. Central to these common goals is the complete documentation of hPSC data, including the ethical provenance of the source material, the hPSC line derivation, culture conditions and genetic constitution of the lines. Data surrounding hPSC is scattered amongst diverse sources, including publications, supplemental data, researcher lab books, accredited lab reports, certificates of analyses and public data repositories. Not all of these data sources are publicly accessible nor associated with metadata nor stored in a standard manner, such that data can be easily found and retrieved. The Human Pluripotent Stem Cell Registry (hPSCreg; https://hpscreg.eu/) was started in 2007 to impart provenance and transparency towards hPSC research by registering and collecting standard properties of hPSC lines. In this chapter, we present a short primer on the history of stem cell-based products, summarize the ethical and regulatory issues introduced in the course of working with hPSC-derived products and their associated data, and finally present the Human Pluripotent Stem Cell Registry as a valuable resource for all stakeholders in therapies and disease modeling based on hPSC-derived cells.
人类多能干细胞（hPSC）在再生医学中的价值尚未完全发挥出来。从基础研究工具发展到经过临床验证的PSC衍生细胞治疗产品，这条路漫长而曲折，引领着研究人员、临床医生、行业人士和监管机构进入未知领域。所有利益相关者必须共同努力，以确保安全有效的细胞疗法的开发。同样，在有意义且可控的疾病建模和药物筛选应用中使用hPSC需要有关所应用细胞系的质量和适用性的信息。这些共同目标的核心是hPSC数据的完整记录，包括源材料的伦理来源、hPSC系的衍生、培养条件和细胞系的基因构成。围绕hPSC的数据分散在各种来源中，包括出版物、补充数据、研究人员实验室记录、认可的实验室报告、分析证书和公共数据存储库。并非所有这些数据源都是公开可用的，也没有与元数据相关联，也不是以标准方式存储的，因此数据不容易被找到和检索。人类多能干细胞登记处（hPSCreg；https://hpscreg.eu/）于2007年成立，通过登记和收集hPSC系的标准特性，为hPSC研究提供来源和透明度。在本章中，我们简要介绍基于干细胞的产品的历史，总结在使用hPSC衍生产品及其相关数据过程中出现的伦理和监管问题，最后介绍人类多能干细胞登记处，将其作为基于hPSC衍生细胞的治疗和疾病建模中所有利益相关者的宝贵资源。
