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

Autor:	Brian K. Fiske, Simon R.W. Stott, Mart Saarma, Camille Carroll, David Eidelberg, Roger A. Barker, Richard K. Wyse, Patrik Brundin, Amber D. Van Laar, Jamie L. Eberling, David T Dexter, Henri J. Huttunen, Merja H. Voutilainen, Adrian P. Kells, Jeffrey H. Kordower, Eros Bresolin, Alasdair Coles, Leah Mursaleen, Don M. Gash, Karl Kieburtz, Alan L Whone, Howard J. Federoff, A. Jon Stoessl, Sigrid Booms, Lyndsey Isaacs, Massimo S. Fiandaca, Anthony E. Lang, Codrin Lungu, Krystof S. Bankiewicz, Helen Matthews, Anders Björklund
Přispěvatelé:	Barker, Roger [0000-0001-8843-7730], Coles, Alasdair [0000-0003-4738-0760], Apollo - University of Cambridge Repository, Mart Saarma / Principal Investigator, Institute of Biotechnology, Helsinki Institute of Life Science HiLIFE, University of Helsinki, Neuroscience Center, Henri Juhani Huttunen / Principal Investigator, Biosciences, Divisions of Faculty of Pharmacy, Regenerative Neuroscience
Rok vydání:	2020
Předmět:	0301 basic medicine Parkinson's disease Neurturin Nigrostriatal pathway Review 3124 Neurology and psychiatry GENE DELIVERY DOUBLE-BLIND 0302 clinical medicine Neurotrophic factors NEUROTROPHIC FACTOR GDNF Glial cell line-derived neurotrophic factor CONVECTION-ENHANCED DELIVERY NIGRAL DOPAMINERGIC-NEURONS SUBSTANTIA-NIGRA biology Dopaminergic RAT STRIATUM Parkinson Disease GDNF 3. Good health NRTN Neuroprotective Agents medicine.anatomical_structure LESION MODEL Substantia nigra 03 medical and health sciences Cellular and Molecular Neuroscience Therapeutic approach INTRAPUTAMENAL INFUSION medicine Animals Humans Glial Cell Line-Derived Neurotrophic Factor clinical trials dopaminergic neurons urogenital system business.industry 3112 Neurosciences Neurosciences Genetic Therapy medicine.disease 030104 developmental biology nervous system Parkinson’s disease biology.protein Biochemistry and Cell Biology Neurology (clinical) NEURTURIN business Neuroscience 030217 neurology & neurosurgery
Zdroj:	Journal of Parkinson's disease, vol 10, iss 3 Barker, R A, Bjorklund, A, Gash, D M, Whone, A, Van Laar, A, Kordower, J H, Bankiewicz, K, Kieburtz, K, Saarma, M, Booms, S, Huttunen, H J, Kells, A, Fiandaca, M S, Stoessl, A J, Eidelberg, D, Federoff, H, Voutilainen, M, 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 Journal of Parkinson's Disease
ISSN:	1877-718X 1877-7171
DOI:	10.3233/jpd-202004
Popis:	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.
Databáze:	OpenAIRE
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