GDNF and Parkinson's Disease : Where Next? A Summary from a Recent Workshop

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Barker , R A , Björklund , A , Gash , D M , Whone , A , Laar , A V , Kordower , J H , Bankiewicz , K , Kieburtz , K , Saarma , M , Booms , S , Huttunen , H J , Kells , A P , Fiandaca , M S , Stoessl , A J , Eidelberg , D , Federoff , H , Voutilainen , M H , Dexter , D T , Eberling , J , Brundin , P , Isaacs , L , Mursaleen , L , Bresolin , E , Carroll , C , Coles , A , Fiske , B , Matthews , H , Lungu , C , Wyse , R K , Stott , S & Lang , A E 2020 , ' GDNF and Parkinson's Disease : Where Next? A Summary from a Recent Workshop ' , Journal of Parkinson's disease , vol. 10 , no. 3 , pp. 875-891 .

Title: GDNF and Parkinson's Disease : Where Next? A Summary from a Recent Workshop
Author: Barker, Roger A.; Björklund, Anders; Gash, Don M.; Whone, Alan; Laar, Amber Van; Kordower, Jeffrey H.; Bankiewicz, Krystof; Kieburtz, Karl; Saarma, Mart; Booms, Sigrid; Huttunen, Henri J.; Kells, Adrian P.; Fiandaca, Massimo S.; Stoessl, A. Jon; Eidelberg, David; Federoff, Howard; Voutilainen, Merja H.; Dexter, David T.; Eberling, Jamie; Brundin, Patrik; Isaacs, Lyndsey; Mursaleen, Leah; Bresolin, Eros; Carroll, Camille; Coles, Alasdair; Fiske, Brian; Matthews, Helen; Lungu, Codrin; Wyse, Richard K.; Stott, Simon; Lang, Anthony E.
Contributor organization: Mart Saarma / Principal Investigator
Institute of Biotechnology
Helsinki Institute of Life Science HiLIFE
University of Helsinki
Neuroscience Center
Henri Juhani Huttunen / Principal Investigator
Divisions of Faculty of Pharmacy
Regenerative Neuroscience
Date: 2020
Language: eng
Number of pages: 17
Belongs to series: Journal of Parkinson's disease
ISSN: 1877-7171
Abstract: The concept of repairing the brain with growth factors has been pursued for many years in a variety of neurodegenerative diseases including primarily Parkinson's disease (PD) using glial cell line-derived neurotrophic factor (GDNF). This neurotrophic factor was discovered in 1993 and shown to have selective effects on promoting survival and regeneration of certain populations of neurons including the dopaminergic nigrostriatal pathway. These observations led to a series of clinical trials in PD patients including using infusions or gene delivery of GDNF or the related growth factor, neurturin (NRTN). Initial studies, some of which were open label, suggested that this approach could be of value in PD when the agent was injected into the putamen rather than the cerebral ventricles. In subsequent double-blind, placebo-controlled trials, the most recent reporting in 2019, treatment with GDNF did not achieve its primary end point. As a result, there has been uncertainty as to whether GDNF (and by extrapolation, related GDNF family neurotrophic factors) has merit in the future treatment of PD. To critically appraise the existing work and its future, a special workshop was held to discuss and debate this issue. This paper is a summary of that meeting with recommendations on whether there is a future for this therapeutic approach and also what any future PD trial involving GDNF and other GDNF family neurotrophic factors should consider in its design.
Subject: GDNF
dopaminergic neurons
Parkinson's disease
clinical trials
3112 Neurosciences
3124 Neurology and psychiatry
Peer reviewed: Yes
Rights: cc_by_nc
Usage restriction: openAccess
Self-archived version: publishedVersion

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