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Parkinson's Disease Health Center

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Gene Therapy May Improve Parkinson’s Symptoms

Study Shows Improvement in Tremors After Injection of Genetic Material Into Brain

Gene Therapy vs. Sham Treatment continued...

The other patients in the study received the gene therapy delivered via catheter in a two-hour infusion. The researchers mimicked the infusion procedure in the sham-therapy patients.

Sixteen of the patients who got the gene therapy and 21 who got the sham surgery completed the study and were included in the analysis.

Over six months of follow-up, half of the gene therapy patients achieved significant improvement in symptoms, compared to 14% of the comparison patients.

Improvements in motor control were seen at one month and continued virtually unchanged throughout the six-month study, Kaplitt says.

"Although the mean improvement was relatively modest, half the subjects improved by over 9 points on the standard Parkinson rating scale, translating to a large and moderate improvement, and 75% improved by 5 points or more. This result is similar to deep brain stimulation (DBS), but the key point here is the safety of the approach,” During says. “This approach is not just symptomatic, but has the potential of being neuroprotective and altering the natural history of the disease. As such it represents a significant advance for Parkinson's disease."

The study was published online today in the journal Lancet Neurology.

Safety of Gene Therapy

Study researcher Andrew Feigin, MD, says the gene therapy is a completely novel approach to treating Parkinson’s disease.

Feigin is an associate professor of neurology and molecular medicine at The Feinstein Institute for Medical Research in Manhasset, N.Y. He has no ties to Neurologix and is not involved with developing the gene therapy.

Feigin tells WebMD that it is not yet clear if the treatment is more effective or is as effective as deep brain stimulation (DBS), which is the most widely used surgical treatment for Parkinson’s disease.

“Even if this proves to have comparable or even near-comparable efficacy to DBS, I would think that it might still have a place in treatment,” he says. “Most importantly, this study shows this kind of therapy can be done safely.”

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