A pioneering clinical trial was done by the researchers at Bristol, UK which brings the possibility to slow, stop or reverse the Parkinson’s Disease.
The results of this trial have been published in the Journal of Parkinson's Disease on February 27th, 2019.
Under this clinical trial program, an experimental treatment has been directly delivered to the brain. This trial has shown the possibility to restore the damaged cells in Parkinson's disease.
Parkinson's disease is a central nervous system disorder that can affect our movement. This disease causes tremors, slow movement, loss of balance and stiffness in body.
The major target behind this trial was to examine if boosting the levels of a naturally-occurring growth factor can regenerate dying dopamine brain cells in patients. These naturally occurring growth factors are also known as Glial Cell Line-Derived Neurotrophic Factor (GDNF).
Under this GDNF trial, six patients participated in the initial study. This pilot study was done to assess the safety involved in this treatment. However, 35 other individuals also participated in the nine-month double-blind trial. In this trial, a few had to receive monthly infusions of GDNF and the other had to get placebo infusions.
The principal investigator of the study, Alan L. Whone said, "The spatial and relative magnitude of the improvement in the brain scans is beyond anything seen previously in trials of surgically delivered growth-factor treatments for Parkinson's. This represents some of the most compelling evidence yet that we may have a means to possibly reawaken and restore the dopamine brain cells that are gradually destroyed in Parkinson's."
Dr. Whone also said, "It's essential to continue research exploring this treatment further. GDNF continues to hold the potential to improve the lives of people with Parkinson's."
According to Steven Gill, a lead neurosurgeon said, "This trial has shown that we can safely and repeatedly infuse drugs directly into patients’ brains over months or years."
Dr. Gill added, “This is a significant breakthrough in our ability to treat neurological conditions, such as Parkinson’s because most drugs that might work cannot cross from the bloodstream into the brain due to a natural protective barrier.”
Details of the study
After the initial nine months of GDNF study, the open-label extension study was started. The effects and safety of exposure to GDNF were studied under this study. It was done for another 40 weeks.
It has been reported that all 41 patients who were treated in the GDNF and placebo study were registered and concluded in the open-label extension study.
The researchers reported that after nine months, no change in the PET scans was found in those who were given a placebo. However, the patients who were given GDNF showed an improvement in the key area of the brain.
It is still unpredictable whether the clinical benefits delay biological changes in PET scans or those need more time to develop.
However, the combined results of these two studies have highlighted that receiving the infusions every four weeks via a skull-mounted port is feasible. The novel method of administering a drug is well tolerated.
The reports make it clear as well that further testing of GDNF in a larger scale study including the use of higher doses are required to determine the future role of GDNF in neurorestorative treatment for Parkinson's Disease.