Expected outcome

Repair-HD will yield a major impact on the creation of new knowledge and the development of new techniques controlling differentiation and proliferation of human stem cells for therapeutic purposes. It will advance clinical translation of this research by leading to 'first in man' trials of stem cell-based therapy in HD.

Improved understanding of mechanisms of neuronal differentiation - Repair-HD will reveal fundamental principles of fate determination and lineage selection for effective differentiation of hPSCs from different sources to authentic MSNs, with a high degree of reliability, stability and reproducibility, as required for clinical application.

Innovative tools for quality control (QC)  - Repair-HD will deliver a specific set of tools for quality assessment of differentiated cells intended for use in HD and HD animal models. This will act as a template for the development of QC measures for cell replacement therapy in other neurodegenerative conditions.

New objective clinical assessment tools - A well established assessment protocol (CAPIT-HD) already exists for neural transplant patients. Repair-HD will validate new assessment tools such as language and social cognition, objective assessment of behavioural deficits or performance-based measures.

Improved health for HD patients - HD has devastating effects on the patients' health and quality of life as it affects many modalities such as cognitive deterioration, behavioural abnormalities and motor dysfunction. Even a modest improvement in function arising from Repair-HD could be significant to the individual in terms of improved health. Moreover, developing a treatment for HD would be the first example of a dementia being treated by regenerative medicine.

Reduced health care costs - HD and neurodegenerative diseases in general are associated with a particularly high economic burden. If transplantation of hPS-MSNs is feasible and effective, this technology could be transferable to other neurodegenerative conditions.The availability of an abundant supply of effective cells for transplantation would constitute a readily available and more economically viable treatment, thus reducing significantly the burden of health care costs.