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

Roger A. Barker, Anders Björklund, Don M. Gash, Alan Whone, Amber Van Laar, Jeffrey H. Kordower, Krystof Bankiewicz, Karl Kieburtz, Mart Saarma, Sigrid Booms, Henri J. Huttunen, Adrian P. Kells, Massimo S. Fiandaca, A. Jon Stoessl, David Eidelberg, Howard Federoff, Merja H. Voutilainen, David T. Dexter, Jamie Eberling, Patrik BrundinLyndsey Isaacs, Leah Mursaleen, Eros Bresolin, Camille Carroll, Alasdair Coles, Brian Fiske, Helen Matthews, Codrin Lungu, Richard K. Wyse, Simon Stott, Anthony E. Lang

Research output: Contribution to journalArticlepeer-review

39 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)875-891
Number of pages17
JournalJournal of Parkinson's Disease
Issue number3
StatePublished - 2020
Externally publishedYes


  • GDNF
  • NRTN
  • Parkinson's disease
  • clinical trials
  • dopaminergic neurons

ASJC Scopus subject areas

  • Clinical Neurology
  • Cellular and Molecular Neuroscience


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