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 . https://doi.org/10.3233/JPD-202004

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.
Other contributor: University of Helsinki, Mart Saarma / Principal Investigator
University of Helsinki, Neuroscience Center
University of Helsinki, Divisions of Faculty of Pharmacy








Date: 2020
Language: eng
Number of pages: 17
Belongs to series: Journal of Parkinson's disease
ISSN: 1877-7171
DOI: https://doi.org/10.3233/JPD-202004
URI: http://hdl.handle.net/10138/319218
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
NRTN
Parkinson's disease
clinical trials
NIGRAL DOPAMINERGIC-NEURONS
NEUROTROPHIC FACTOR GDNF
CONVECTION-ENHANCED DELIVERY
INTRAPUTAMENAL INFUSION
DOUBLE-BLIND
SUBSTANTIA-NIGRA
GENE DELIVERY
LESION MODEL
RAT STRIATUM
NEURTURIN
3112 Neurosciences
3124 Neurology and psychiatry
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