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Review
. 2024 Nov;14(8):1659-1676.
doi: 10.1177/1877718X241301041. Epub 2024 Dec 27.

Neurotrophic factors for Parkinson's disease: Current status, progress, and remaining questions. Conclusions from a 2023 workshop

Roger A Barker  1 , Mart Saarma  2 , Clive N Svendsen  3 , Catherine Morgan  4   5 , Alan Whone  4   5 , Massimo S Fiandaca  6 , Matthias Luz  6 , Krystof S Bankiewicz  6   7 , Brian Fiske  8 , Lyndsey Isaacs  9 , Arthur Roach  10 , Thomas Phipps  11 , Jeffrey H Kordower  12 , Emma L Lane  13 , Henri J Huttunen  14 , Aideen Sullivan  15 , Gerard O'Keeffe  16 , Valeria Yartseva  17 , Howard Federoff  17   18
Affiliations
Review

Neurotrophic factors for Parkinson's disease: Current status, progress, and remaining questions. Conclusions from a 2023 workshop

Roger A Barker et al. J Parkinsons Dis. 2024 Nov.

Abstract

In 2023, a workshop was organized by the UK charity Cure Parkinson's with The Michael J Fox Foundation for Parkinson's Research and Parkinson's UK to review the field of growth factors (GFs) for Parkinson's disease (PD). This was a follow up to a previous meeting held in 2019.1 This 2023 workshop reviewed new relevant data that has emerged in the intervening 4 years around the development of new GFs and better models for studying them including the merit of combining treatments as well as therapies that can be modulated. We also discussed new insights into GF delivery and trial design that have emerged from the analyses of completed GDNF trials, including the patient voice, as well as the recently completed CDNF trial.2 We then concluded with our recommendations on how GF studies in PD should develop going forward.

Keywords: GDNF; Parkinson's; animal models; end points; gene therapy; patient voice; trials; workshop.

Plain language summary

For many years, scientists have explored the idea of administering growth factors to the brain, thereby repairing the damage associated with neurodegenerative diseases like Parkinson's disease (PD). Growth factors like glial cell line-derived neurotrophic factor (GDNF) have shown promise in animal models of PD, and initial clinical trials suggested that this approach may be beneficial for patients. However, there has been some uncertainty in the field after a number of clinical trials did not reach their primary endpoints. Nevertheless, the development of growth factor therapies for PD has continued, with new trials underway. Recent developments in this scientific area were discussed at a workshop organized by a number of PD charities in 2023. The discussion and conclusions from that workshop are presented in this new paper.

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