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Designing stem-cell-based dopamine cell replacement trials for Parkinson's disease

Barker, Roger A., Farrell, K., Guzman, N., He, X., Lazic, S., Moore, S., Morris, R., Tyers, P., Wijeyekoon, R., Daft, D., Hewitt, S., Dayal, V., Foltynie, T., Kefalopoulou, Z., Mahlknecht, P., Lao-Kaim, N., Piccini, P., Bjartmarz, H., Björklund, A., Lindvall, O., Nelander-Wahlestedt, J., Parmar, M., Paul, G., Widner, H., Church, A., Dunnett, S., Peall, K., Rosser, A., Gurruchaga, J., Palfi, S., Piroth, T. and Winkler, C. 2019. Designing stem-cell-based dopamine cell replacement trials for Parkinson's disease. Nature Medicine 25 (7) , pp. 1045-1053. 10.1038/s41591-019-0507-2
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Abstract

Clinical studies of Parkinson’s disease (PD) using a dopamine cell replacment strategy have been tried for more than 30 years. The outcomes following transplantation of human fetal ventral mesencephalic tissue (hfVM) have been variable, with some patients coming off their anti-PD treatment for many years and others not responding and/or developing significant side effects, including graft-induced dyskinesia. This led to a re-appraisal of the best way to do such trials, which resulted in a new European-Union-funded allograft trial with fetal dopamine cells across several centers in Europe. This new trial, TRANSEURO (NCT01898390), is an open-label study in which some individuals in a large observational cohort of patients with mild PD who were undergoing identical assessments were randomly selected to receive transplants of hfVM. The TRANSEURO trial is currently ongoing as researchers have completed both recruitment into a large multicenter observational study of younger onset early-stage PD and transplantation of hfVM in 11 patients. While completion of TRANSEURO is not expected until 2021, we feel that sharing the rationale for the design of TRANSEURO, along with the lessons we have learned along the way, can help inform researchers and facilitate planning of transplants of dopamine-producing cells derived from human pluripotent stem cells for future clinical trials.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Biosciences
MRC Centre for Neuropsychiatric Genetics and Genomics (CNGG)
Medicine
Neuroscience and Mental Health Research Institute (NMHRI)
Publisher: Nature Publishing Group
ISSN: 1078-8956
Date of First Compliant Deposit: 10 July 2019
Date of Acceptance: 3 June 2019
Last Modified: 19 Oct 2019 22:09
URI: http://orca-mwe.cf.ac.uk/id/eprint/124136

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