“This is the first phase 2 study conducted under a rigorous randomized, double-blind, sham-controlled surgical design to conclusively demonstrate that gene therapy can be effective for neurological diseases.”
People with advanced Parkinson’s disease taking an experimental gene therapy saw a significant easing of their motor skill problems in a mid-stage clinical study. The results hold promise that gene therapy may become a new treatment for a debilitating disorder with no known cure.
The mid-stage study of 45 patients, funded by Neurologix to test its novel gene therapy, met its primary endpoints for efficacy, safety, and tolerability. Results were published in an online-first edition of The Lancet Neurology.
Parkinson’s disease patients treated with the gene therapy saw a statistically significant improvement, in their motor abilities at one month compared to patients treated with sham surgery, which continued throughout the blinded six-month study period. Study results also showed that half of the subjects treated with the gene therapy achieved previously defined moderate-to-large clinically-meaningful symptom improvements, compared to just 14 percent of subjects receiving sham surgical treatment. No serious adverse events related to the gene therapy were reported.
Limb tremors, rigid limbs, trouble with balance, and slowness of movement are some of the manifestations of Parkinson’s disease, a neurodegenerative disorder caused by the gradual deterioration of nerve cells in the brain. Effective treatments are few.
For the 1 million Americans who currently live with Parkinson’s, treatment often involves the use of levodopa, a drug that stimulates the production of dopamine. It is not always effective and many patients develop complications from taking it. The alternative is electrical deep brain stimulation, which requires the implantation of permanent medical devices in the brain.
“This is the first phase 2 study conducted under a rigorous randomized, double-blind, sham-controlled surgical design to conclusively demonstrate that gene therapy can be effective for neurological diseases. This confirms our phase 1 results and indicates that NLX-P101 may provide a safe, effective and minimally invasive treatment option for patients with Parkinson’s disease,” says senior author and co-principal investigator Andrew Feigin, of The Feinstein Institute for Medical Research at North Shore - LIJ Health System in Manhasset, New York.
Neurologix’ scientific co-founders, Matthew During and Michael Kaplitt, have been at the forefront of gene therapy research since 1989. This mid-stage trial was the culmination of nearly 20 years of work in adeno-associated virus gene transfer technology. The therapy they have developed, NLX-P101, aims to reset the overactive brain cells in the subthalamic nucleus to inhibit electrical activity and return brain network activity to more normal levels.
Based on the results of this study, which Neurologix has discussed with the U.S. Food and Drug Administration, the company plans to submit a protocol for a phase 3 trial later this year.
March 18, 2011
http://www.burrillreport.com/article-gene_therapy_shows_promise_in_treating_parkinson%e2%80%99s_disease.html