Researchers are actively investigating the potential of neurotrophic or nerve growth factors as a treatment for people with Parkinson’s. Additionally, alternative methods of delivering these promising molecules into the brain are currently being explored.

Neurotrophic factors are proteins that are naturally produced in the brain to help nurture, grow and protect nerve cells (neurons). Two neurotrophic factors, Glial cell line-derived neurotrophic factor (GDNF) and Cerebral Dopamine Neurotrophic Factor (CDNF), have been tested both in the laboratory and in clinical trials for people with Parkinson’s, though the results so far have been inconclusive.

What is Cure Parkinson’s doing to further neurotrophic factor research?

Cure Parkinson’s continues to support research exploring neurotrophic factors for people with Parkinson’s and has been funding projects in this area as well as coordinating conversations and building collaborations around the future of this field.


Professors Aideen Sullivan and Gerard O’Keeffe from University College Cork are studying how a neurotrophic growth factor called Growth/Differentiation Factor 5 (GDF5) affects neurons in Parkinson’s using laboratory models; GDF5 helps neurons in a different way to GDNF, and the hope is that this approach could be more helpful for people with Parkinson’s. They are also looking at the combined delivery of GDF5 and GDNF, to enhance their therapeutic potential.


Like most neurotrophic factors, CDNF is too large to pass through the blood-brain barrier – a semi-permeable layer of cells that protects and controls what passes in and out of the brain. This means that therapies involving neurotrophic factors often require brain surgery to directly administer the molecules to the brain.

However, Professor Mart Saarma from the University of Helsinki has identified and been working to develop an active fragment of CDNF, known as C-CDNF. In this Cure Parkinson’s funded project, the investigators are testing C-CDNF in laboratory models of Parkinson’s to determine the optimal size for passage into the brain whilst retaining its potentially neuroprotective qualities.

Neurotrophic Factor Workshop

Last summer, Cure Parkinson’s hosted a neurotrophic factor workshop at Cumberland Lodge in Windsor alongside its annual International Linked Clinical Trials (iLCT) meeting. At this meeting, a group of international key opinion leaders with direct knowledge and practical experience in the field of neurotrophic factors gathered to share their findings and offer insights into the field as it currently stands. A commentary paper summarizing the key outcomes of this meeting will be published later this year.

In Summary

Researchers are studying ways to incorporate neurotrophic factors into viable treatment options for people with Parkinson’s and they’re testing these in both the lab and in clinical trials. Some methods are invasive so it’s important to involve people with Parkinson’s in deciding what is best – where hurdles exist for some methods of delivery, alternative options need to be explored. Cure Parkinson’s is optimistic about this research and is keen to see how it develops as a potential treatment.

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