A small gene therapy study involving a handful of Parkinson's patients has produced impressive data indicating that they may be on to a potential breakthrough. Scientists at New York University used a genetically modified virus to introduce a gene that produces the chemical GABA, which inhibits nerve cells over-stimulated by Parkinson's. And they tracked the results with a PET scan. Six months after beginning therapy, patients demonstrated an average 30 percent improvement in symptoms with one volunteer showing an impressive 65 percent improvement. Only one side of their brains was treated in order to better track results and protect volunteers from any unexpected side affects. The technique was developed by Neurologix, which is now beginning to map out a mid-stage study.
Because all of the patients received the therapy in the open-label study, the researchers wanted to be able to verify the impact of the therapy with PET scans, eliminating any potential placebo effect. "This study demonstrates that PET scanning can be a valuable marker in testing novel therapies for Parkinson's disease," said lead researcher Dr. David Eidelberg.
- see this release
- read this report on the study from the BBC