Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by a CAG triplet expansion in the Huntington gene, which causes progressive neuropsychiatric and motor dysfunction and leads to death. There is currently no cure for HD - treatment is strictly palliative.
The mutation resulting in HD causes selective loss of striatal neurons, leading to more widespread brain degeneration. However, a proposed mechanism of engineered mesenchymal stem/stromal cells (MSCs) as a potential treatment for HD will be discussed in a webinar from RegMedNet on Wednesday 13 May. Vicki Wheelock, Jan Nolta and Kyle Fink from University of California, Davis (CA, USA) will discuss this potential treatment, progress made to date in developing and manufacturing MSC/brain-derived neurotrophic factor (BDNF) in readiness to apply for regulatory approval to conduct a first-in-human Phase I trial, results of preclinical studies in transgenic HD mouse models in support of the application for regulatory approval, design of the lead-in observational study, and the future planned Phase I clinical trial and potential future applications of this approach in other neurological disorders.
An important mechanism postulated to lead to neuronal death in HD is impaired expression of BDNF, and multiple studies in animal models support increased BDNF as a potential disease-modifying therapy for HD. MSCs have innate neurorestorative properties that promote endogenous neuronal growth, so could provide an exciting new treatment option for the disease.
'This webinar is an excellent way to exchange knowledge in regenerative medicine applications, so that groups working to make a difference in a particular rare disease, like Huntington's, might be able to impact other teams working on related conditions,' said Jan Nolta, Director of the Institute for Regenerative Cures and Stem Cell Program, UC Davis. 'For patients suffering from rare neurodegenerative diseases, every day is important, so the more we can share knowledge the faster we can all get to meaningful treatments.'
'Huntington's disease is a devastating neurodegenerative disease that, at present, has no treatment, let alone a cure,' added Alexandra Thompson, Community Manager for RegMedNet. 'This webinar will provide an update on the work Vicki Wheelock, Jan Nolta and Kyle Fink from University of California, Davis, have carried out to plan and prepare for a Phase I trial of their gene-modified mesenchymal stem cell therapy. If undertaken, this trial could be a great step towards finding a potential therapy for HD, as well as other neurological disorders.'