Basic science and preclinical development of cell transplantation for basal ganglia disorders

细胞移植治疗基底神经节疾病的基础科学和临床前进展

• 批准号: G1001257-E01/1
• 负责人: Stephen Dunnett
• 金额: $ 142.6万
• 依托单位: CARDIFF UNIVERSITY
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2011
• 资助国家: 英国
• 起止时间: 2011 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=G1001257-E01%2F1
• 关键词: Basic; science; preclinical; development; cell

This programme provides the infrastructure, models and expertise to deliver human neurons (primary fetal, expanded fetal and stem cells) for use in clinical trials of cell transplantation in neurodegenerative disease. Specifically, in the Cardiff Brain Repair Group, we have established an ethically sourced supply and quality-controlled production of human embryonic brain cells for transplantation in both Huntington?s and Parkinson?s disease. We have also established the cell culture and animal model test systems to determine the precise preparation and implantation protocols that are most likely to lead to functional benefit (i.e. recovery in both motor and cognitive symptoms) in trials set to commence/resume in both PD and HD in 2011-12. Moreover, the UK is a world leader in fundamental stem cell biology which are likely to yield the next generation of pluripotential cells for therapy in a wide range of diseases; however, if we are to reap the benefit from the signficant government support and public investment in this area, we need to have in place the infrastructure to test the functional efficacy of new stem cell-based preparations, to prepare cells to clinical-grade quality standards, and to undertake clinical trials with skilled surgery and fully validated assessment protocols in well characterised patients. Thus, alongside the present establishment of proof-of concept with primary fetal cells, this programme grant underpins the expertise and infrastructure within the Cardiff Brain Repair Group that is required to take new stem cell therapies rapidly into clinical trial as soon as they become available. Without such proactive preparation and support, the UKs intellectual lead in fundamental stem cell biology is likely to be squandered to implementation and commercialisation elsewhere.

该计划提供基础设施，模型和专业知识，以提供人类神经元（原代胎儿，扩增的胎儿和干细胞）用于神经退行性疾病细胞移植的临床试验。具体来说，在卡迪夫脑修复小组，我们已经建立了一个道德来源的供应和质量控制的生产人类胚胎脑细胞移植在亨廷顿？s和帕金森？的疾病。我们还建立了细胞培养和动物模型试验系统，以确定在2011-12年开始/恢复的PD和HD试验中最有可能产生功能获益（即运动和认知症状恢复）的精确制备和植入方案。此外，英国是基础干细胞生物学领域的世界领导者，这些干细胞可能会产生下一代多能细胞，用于治疗多种疾病;然而，如果我们要从这一领域的重大政府支持和公共投资中获益，我们需要建立基础设施来测试新的干细胞制剂的功能功效，制备符合临床级质量标准的细胞，并在特征明确的患者中进行熟练手术和充分验证的评估方案的临床试验。因此，除了目前建立的原代胎儿细胞概念验证外，该计划拨款还支持了卡迪夫脑修复小组的专业知识和基础设施，需要将新的干细胞疗法在可用后迅速进入临床试验。如果没有这种积极的准备和支持，英国在基础干细胞生物学方面的知识领先地位很可能被浪费在其他地方的实施和商业化上。