In a new research series, scientists started testing the efficacy of new treatment for Parkinson’s disease and its method of delivery. A big team of investigators from different institutes came together to conduct the trial.
The study focused on restoration of the decreasing brain cells producing dopamine in people diagnosed with Parkinson’s disease.
The findings appear in the Journal of Parkinson’s Disease and explain a novel therapy but also an innovative method of administration, through a port in the skull.
The research team set up a pilot study including 6 participants suffering from Parkinsonism. The main goal was to check if this new approach was safe or not.
The next stage of the study involved 35 participants with this disease who participated in the double-blind trial when both the researchers and the participants knew nothing about the experimental or placebo groups.
This trial continued for 9 months during which the investigators gave GDNF to half of the people and placebo to the other half.
At last, the researchers conducted an open-label trial built on the results of previous tests. In this experiment, people who had previously gotten GDNF continued having treatment for 40 weeks additionally.
At the same time, people who were previously on placebo received GDNF for 40 more weeks. At the same time, the patients involved in the parent study were screened for their participation in an extension trial.
To use the drug, the participants agreed to get a port implanted in their skulls allowing the drug infusions to get to the brains directly. After the implantation, the volunteers utilized 1000 infusions of the drug once every 4 weeks.
The scientists analyzed the results gained from first 40 weeks with no change in the brains of people receiving placebo. However, those receiving GDNF had a 100 percent improvement in the putamen area, the region that primarily controls dopamine production.
The relative and spatial magnitude of their brain improvement in the brain scans have never been seen in any of the trials conducted beforehand in reference to Parkinson’s disease, according to the experts.
This research has provided a compelling piece of evidence that it may be possible to restore the brain cells producing dopamine that are destroyed during Parkinson’s disease.
A Prominent Breakthrough
After 18 months, the participants receiving GDNF for 9 or 18 months started experiencing improvements in their motor symptoms. Their performances had been enhanced as compared to those before the trials. The investigators also concluded that prolonged use of GDNF was safe.
However, the team has also warned that by the end of the open-label trial, no proper differences were found in terms of symptom improvement between the participants receiving GDNG for 9 months or double the time period.
For this purpose, the researchers have argued that they have to perform more studies to assess how to provide this treatment to others. This will help them reap maximum benefits.