Designing stem-cell-based dopamine cell replacement trials for Parkinson's disease
File(s)
Author(s)
Parker, RA
TRANSEURO consortium
Piccini, Paola
Type
Journal Article
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.
Date Issued
2019-07-01
Date Acceptance
2019-06-03
Citation
Nature Medicine, 2019, 25, pp.1045-1053
ISSN
1078-8956
Publisher
Nature Research
Start Page
1045
End Page
1053
Journal / Book Title
Nature Medicine
Volume
25
Copyright Statement
© Springer Nature America, Inc. 2019
Sponsor
Commission of the European Communities
Grant Number
242003
Subjects
Science & Technology
Life Sciences & Biomedicine
Biochemistry & Molecular Biology
Cell Biology
Medicine, Research & Experimental
Research & Experimental Medicine
TRANSPLANTATION
NEURONS
TISSUE
PROGRESSION
THERAPY
GRAFTS
FUTURE
TRANSEURO consortium
Immunology
11 Medical and Health Sciences
Publication Status
Published
Date Publish Online
2019-07-01