Phase I Trial demonstrates safety and improvement in motor function and brain metabolism.
Neurologix reported successful completion of its Phase I trial of the first gene therapy for Parkinson’s disease with statistically significant results, which were presented at the “36th Annual Meeting of the Society of Neuroscience” in Atlanta.
“This gene therapy trial is particularly unique and the clinical data unusually promising because the treatment was confined to just one side of the brain,” points out Matthew J. During, M.D., a company founder. In its next trial, the company plans to infuse its treatment into both sides of the brain.
Findings of the open label, dose-escalating, unilateral trial confirmed the safety and tolerability in all 12 patients studied for a year, according to the company. Though efficacy was only designated as a secondary outcome, the trial also yielded statistically significant clinical efficacy and neuro-imaging results.
At one year, all 12 patients as a group demonstrated a clinical improvement of 25% in the Unified Parkinson’s Disease Rating Scale, compared to baseline (p < 0.005). Of the 12 patients, nine showed an average improvement of 37% and five had substantial improvement between 40% and 65%.
Clinical improvement also correlated well to metabolic brain changes. The PET scan data revealed a significant improvement (p < 0.001) in brain metabolism on the treated side of the brain as compared to the untreated side.