Glial cell line-derived neurotrophic factor (GDNF) is a protein that has been shown to be neuroprotective for dopamine neurons, the cells in the brain that are badly affected by Parkinson’s.
Cure Parkinson’s has been involved in and has championed the neurotrophic factor called Glial cell-derived neurotrophic factor (or GDNF) since 2003 through the commitment and determination of the charity’s late co-founder and president Tom Isaacs. In February 2019, the results of the Phase II Bristol study, supported by Cure Parkinson’s, investigating the use of GDNF in people with Parkinson’s were published in the research journals Brain and the Journal of Parkinson’s Disease (JoPD)
Nerve growth or neurotrophic factors are small proteins that support neurons and encourage their growth and survival during development. They have also been shown to rescue neurons in laboratory models of neurodegenerative conditions, exhibiting powerful neuroprotective properties. There has been considerable preclinical research exploring their potential use in Parkinson’s. Unfortunately, the translation of that research into humans has not been easy.
This ground-breaking trial involved the mechanical delivery of this experimental treatment directly into the brains of people with Parkinson’s in an attempt to rescue and restore damaged neurons.
While this clinical trial of GDNF did not meet its primary endpoint (this is a pre-determined measure of efficacy), there were some very interesting findings. The brain imaging data, for example, suggested that GDNF was having a biological effect in the brain. But importantly the clinical findings of the study did not mirror the participant’s experience in terms of benefit. Examples of this were demonstrated in the award winning BBC2 documentary: The Parkinson’s Drug Trial: A Miracle Cure?
As a result, the interpretation of all the results of this trial has been challenging.
Following publication of the trial results, Cure Parkinson’s has been working with research partners and the Parkinson’s community to address some of the uncertainties and issues raised in this trial and to explore the appropriate pathway forward for GDNF.