Viability, specificity and yields of clinical grade primary and expanded human fetal cells for neural transplantation

The MRC has sponsored a multicentre clinical trial on the safety and fesibility of fetal neural tissue transplantation in a severe progressive degenerative disease of the brain, Huntington s disease. However, this trial has been suspended after completion of the first 5 transplants because of the implementation of new EU regulations that require pharmaceutical grade handling of any cells or tissues used in patients. Although the UK trial had fully assessed the safety of the cells, we could not comply with the very stringent environmental control conditions required for accreditation. We have therefore undertaken a full rebuild of the laboratories to comply with the new regulations. In the present grant, we will undertake the detailed studies required to validate new laboratory procedures for cell preparation and expansion in cell culture and confirm the efficacy of the cells in animal models. This will allow us to complete the suspended HD trial, and to extend the multicentre UK cell transplant programme to trials in Parkinson s as well as Huntington s disease.

The MRC-sponsored multicentre UK pilot trial on the safety and fesibility of fetal neural tissue transplantation in Huntington s disease is currently suspended following introduction of the EU Cells and Tissue directive. We are just completing conversion of a small GMP laboratory in Cardiff University for handling of clinical-grade primary and short-term expanded fetal neural progenitor cells for application in neurodegenerative disease. This application is to undertake the translational studies required to validate cell preparation and expansion protocols under GMP, both to allow us to complete the suspended HD trial, and to define improved cell preparations for subsequent multicentre UK trials in both Parkinson s and Huntington s diseases.