Development and validation of functional cell therapies for Huntington's and Parkinson's diseases

Involving neural tissue transplantation in the brain, as well as stem cells, human embryos and animal experiments, this research is high profile in the general as well as scientific news media. In addition to publication in peer reviewed journals, the results of the present project will be widely disseminated to the biomedical research community, to NHS and clinical professions, and to interested charities and the lay public, through lectures and seminars in the normal way as well as in media interviews when appropriate. The PIs already have extensive experience in public communication of research results. We strive to achieve the best balance between the competing demands of proper disclosure of publicly funded science, public education, promoting enthusiasm for science and support for the MRC and biomedical research, but without raising false or premature hopes for cures that remain distant. The credibility of the team is enhanced by the close collaboration of the two PIs, one a basic scientist and the other a clinical scientist, combining resources and expertise to develop a translational programme for novel cell-based treatments for crippling human neurodegenerative diseases, spanning basic cellular, molecular and behavioural science through to clinical delivery in patients.

The UK is taking an international lead in developing stem cells for cell-based therapy applicable to a broad range of diseases. However, for these new sources of cells to come to effective application in the clinic requires that they are first evaluated and validated for specificity and functional potential in appropriate model systems, both in vitro and in vivo.

The proposed project establishes quality-controlled processing of cells in vitro, determines optimal dissection, preparation handling and transplantation protocols, develops the experimental models for functional analysis, and refines our understanding of the theoretical principles and mechanisms of function for application of cell-based therapies in two specific human neurodegenerative diseases, Huntington‘s and Parkinson‘s. The programme sustains a centre of expertise developing the quality-controlled facilities necessary to progress specific cell sources (fetal now, expanded progenitors imminently, and stem cells in the foreseeable future) to clinical application rapidly and efficiently.