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Trophic factors for Parkinson's disease : Where are we and where do we go from here?

Paul, Gesine LU and Sullivan, Aideen M. (2019) In European Journal of Neuroscience 49(4). p.440-452
Abstract

Perhaps the most important unmet clinical need in Parkinson's disease (PD) is the development of a therapy that can slow or halt disease progression. Extensive preclinical research has provided evidence for the neurorestorative properties of several growth factors, yet only a few have been evaluated in clinical studies. Attempts to achieve neuroprotection by addressing cell-autonomous mechanisms and targeting dopaminergic neurons have been disappointing. Four different trophic factors have so far entered clinical trials in PD: glial cell line-derived growth factor, its close structural and functional analog neurturin, platelet-derived growth factor and cerebral dopaminergic neurotrophic factor. This article reviews the pre-clinical... (More)

Perhaps the most important unmet clinical need in Parkinson's disease (PD) is the development of a therapy that can slow or halt disease progression. Extensive preclinical research has provided evidence for the neurorestorative properties of several growth factors, yet only a few have been evaluated in clinical studies. Attempts to achieve neuroprotection by addressing cell-autonomous mechanisms and targeting dopaminergic neurons have been disappointing. Four different trophic factors have so far entered clinical trials in PD: glial cell line-derived growth factor, its close structural and functional analog neurturin, platelet-derived growth factor and cerebral dopaminergic neurotrophic factor. This article reviews the pre-clinical evidence for the neuroprotective and neurorestorative actions of these growth factors and discusses limitations of preclinical models, which may hamper successful translation to the clinic. We summarize the previous and ongoing clinical trials using growth factors in PD and emphasize the caveats in clinical trial design that may prevent the further development and registration of potentially neuroprotective and neurorestorative treatments for individuals suffering from PD.

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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
keywords
animal models, CDNF, clinical trials, GDNF, neurotrophic factors, neurturin, Parkinson's disease, PDGF-BB
in
European Journal of Neuroscience
volume
49
issue
4
pages
440 - 452
publisher
Wiley-Blackwell
external identifiers
  • scopus:85053315657
ISSN
0953-816X
DOI
10.1111/ejn.14102
language
English
LU publication?
yes
id
ea36a6e4-d081-4382-baa1-3f91374d130c
date added to LUP
2018-10-22 14:01:21
date last changed
2019-09-17 04:41:36
@article{ea36a6e4-d081-4382-baa1-3f91374d130c,
  abstract     = {<p>Perhaps the most important unmet clinical need in Parkinson's disease (PD) is the development of a therapy that can slow or halt disease progression. Extensive preclinical research has provided evidence for the neurorestorative properties of several growth factors, yet only a few have been evaluated in clinical studies. Attempts to achieve neuroprotection by addressing cell-autonomous mechanisms and targeting dopaminergic neurons have been disappointing. Four different trophic factors have so far entered clinical trials in PD: glial cell line-derived growth factor, its close structural and functional analog neurturin, platelet-derived growth factor and cerebral dopaminergic neurotrophic factor. This article reviews the pre-clinical evidence for the neuroprotective and neurorestorative actions of these growth factors and discusses limitations of preclinical models, which may hamper successful translation to the clinic. We summarize the previous and ongoing clinical trials using growth factors in PD and emphasize the caveats in clinical trial design that may prevent the further development and registration of potentially neuroprotective and neurorestorative treatments for individuals suffering from PD.</p>},
  author       = {Paul, Gesine and Sullivan, Aideen M.},
  issn         = {0953-816X},
  keyword      = {animal models,CDNF,clinical trials,GDNF,neurotrophic factors,neurturin,Parkinson's disease,PDGF-BB},
  language     = {eng},
  number       = {4},
  pages        = {440--452},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Neuroscience},
  title        = {Trophic factors for Parkinson's disease : Where are we and where do we go from here?},
  url          = {http://dx.doi.org/10.1111/ejn.14102},
  volume       = {49},
  year         = {2019},
}