Barker Roger A, Studer Lorenz, Cattaneo Elena, Takahashi Jun
John van Geest Centre for Brain Repair, Forvie site, University of Cambridge, Cambridge, UK.
Memorial Sloan Kettering Cancer Center, NY, USA.
NPJ Parkinsons Dis. 2015 Sep 24;1:15017. doi: 10.1038/npjparkd.2015.17. eCollection 2015.
Translating new cell-based therapies to the clinic for patients with neurodegenerative disorders is complex. It involves pre-clinical testing of the cellular product and discussions with several regulatory agencies, as well as ethical debates. In an attempt to support efforts around the world, we set up a global consortium that brings together the major funded teams working on developing a stem cell-derived neural transplantation therapy for Parkinson's disease (PD). This consortium, G-Force PD, involves teams from Europe, USA, and Japan, and has already met on two occasions to discuss common problems, solutions, and the roadmap to the clinic. In this short review, we lay out the brief history and rationale for this initiative and discuss some of the issues that arose in our most recent meeting (May 2015) as we consider undertaking first-in-human clinical trials with stem cell-derived neurons for PD.
将基于细胞的新疗法应用于神经退行性疾病患者的临床治疗是一项复杂的工作。这涉及到对细胞产品进行临床前测试,与多个监管机构进行讨论,以及伦理辩论。为了支持全球范围内的相关努力,我们成立了一个全球联盟,将致力于开发用于帕金森病(PD)的干细胞衍生神经移植疗法的主要受资助团队聚集在一起。这个联盟,即G-Force PD,成员来自欧洲、美国和日本,已经召开了两次会议,讨论共同问题、解决方案以及临床应用路线图。在这篇简短的综述中,我们阐述了这项倡议的简要历史和基本原理,并讨论了在我们最近一次会议(2015年5月)上出现的一些问题,当时我们正在考虑开展使用干细胞衍生神经元治疗PD的首次人体临床试验。