NEUROLOGIX COMPLETES TRIAL OF PARKINSON'S GENE THERAPY
Neurologix has successfully completed its Phase I trial of gene therapy for Parkinson's disease with statistically significant results. The data was presented at the 36th Annual Meeting of the Society of Neuroscience in Atlanta.
The open-label, dose-escalating, unilateral trial enrolled four patients in each of three cohorts. All procedures were performed under local anesthesia, and all patients were discharged from the hospital within 48 hours of the procedure and followed for 12 months. Primary outcomes of the study design, safety and tolerability, were successfully met. There were no adverse events reported relating to the treatment. Though efficacy was only designated as a secondary outcome, the trial also yielded statistically significant clinical efficacy and neuro-imaging results.
The gene transfer procedure utilized the adeno-associated virus (AAV) vector, a virus that has been used safely in a variety of clinical gene therapy trials. In its Parkinson's disease trial, Neurologix used its proprietary AAV-GAD gene transfer technology.
At one year, all 12 patients as a group demonstrated a clinical improvement of 25 percent in the Unified Parkinson's Disease Rating Scale (UPDRS) compared with baseline. Nine of the 12 patients showed an average improvement of 37 percent, and five of these patients had substantial improvement of between 40 percent and 65 percent.
Clinical improvement also correlated well to metabolic brain changes as measured by Positron Emission Tomography (PET) scan. PET is an imaging method that measures brain metabolism following the injection of a radioactive analog of glucose (fluorodeoxyglucose). The PET scan data revealed a significant improvement in brain metabolism on the treated side of the brain as compared with the untreated side. In its next trial, the company plans to infuse its treatment into both sides of the brain.
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