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

• United Kingdom

Medical Research Council

1300218.87

01-02-2011

36

• Parkinson’s disease
• Huntington’s disease

The UK is taking an international lead in developing stem cells for cell-based therapy applicable to a broad range of diseases. The purpose of this programme application is to address the translational gap to deliver clinical-grade human neuronal cells from primary, expanded and pluripotent origins for transplantation in clinical trials of human neurodegenerative disease. We complement other stem cell programmes in establishing and maintaining the infrastructure for ethically-sourced, safe, quality-assured processing of cells of clinical grade under regulatory licence, alongside establishing relevant patient cohorts, assessment programmes and clinical trial expertise, without which advances in basic cell biology cannot progress to human use or clinical trial. A major component of this programme is to develop and refine the animal models to evaluate alternative sources of cells with predictive validity of likely functional efficacy in man. Immediate applications are set to commence using primary fetal cells in 2011-2012 in Huntingtons disease and Parkinsons disease, with subsequent applications in stoke, and using expanded fetal progenitors, pending the availability of clinical grade stem cells.

• Basic research