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

点击翻译按钮获取中文摘要

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.