The novel GDNF clinical trial offered hope for restoring brain cells in people with Parkinson’s Disease. Including 41 people with Parkinson’s Disease aged between 41 and 72, the clinical trial was designed to investigate whether GDNF could slow, stop or reverse Parkinson’s Disease. Results were recently published. Read our study summary here.
Neurotrophic factors are small proteins produced in the brain that support the growth, survival and differentiation of brain cells. These proteins are important for maintenance of brain health and consequently there has been considerable interest in whether these proteins might improve survival or indeed assist regeneration of brain cells that are affected by Parkinson’s disease.
From 2012 to 2017, UK researchers conducted a novel clinical trial looking at the effects of delivering one such Neurotrophic factor, Glial Derived Neurotrophic Factor (GDNF) directly to the brain, using a new type of surgically implanted device. Link to article here https://www.parkinsonsmovement.com/the-gdnf-trial/
Including 41 people with Parkinson’s Disease aged between 41 and 72, the clinical trial was designed to investigate whether GDNF could slow, stop or reverse Parkinson’s Disease.
The group, all of whom had been diagnosed around eight years ago, were split into two smaller groups who were allocated either a placebo or the GDNF treatment.
Using a device mounted on the skull, the researchers were able to directly infuse GDNF into the putamen – an important brain region that is affected by PD. Study participants received either placebo or GDNF treatment every four weeks for 40 weeks.
After the 40-week period, the researchers noted that there was “no overall difference between the GDNF and placebo group” in terms of their movement scores. However, nine people showed what researchers called a “clinically significant improvement”.
The researchers then conducted an extension study for an additional 40 weeks and again found no clear difference between patients who had received GDNF versus placebo.
Overall, neither the GDNF study nor the extension study helped to improve movement-related symptoms in people with Parkinson’s, ultimately leading to the conclusion that GDNF infusions at the dose and for the duration used did not improve Parkinson’s disease.
Despite these disappointing results, the study demonstrated that direct monthly brain infusions of substances that might treat Parkinson’s disease is feasible and tolerable, thus providing an important platform for future trials into possible therapies for Parkinsons’s disease.
A documentary regarding this trial has been produced by the BBC: https://www.bbc.com/news/av/stories-47483307/the-radical-drug-trial-hoping-for-a-miracle-parkinson-s-cure
