"GAD treatment involves injecting a small amount of a gene-carrying virus (AAV) into the STN region of the brain to calm it down by turning the excitatory chemical glutamate into the inhibitory brain chemical GABA."  (The Guardian, Oct. 11, 2002)  The optimal location is determined with an MRI image and CT scan.  The final target is confirmed with fine electrical probes with the patient awake.  The AAV-GAD is slowly delivered through a very fine catheter for 90 minutes. (Weill Cornell Press Release, August 20, 2003)

A web cast explaining the phase II trial is available for patients. Register at http://www.neurologixpdtrial.com/

Non-dopaminergic treatment: According to the sponsor's press release in June 2010, "While dopamine clearly plays a role in Parkinson's disease, dopamine levels in the brain are inherently difficult to control, resulting in sub-optimal treatment outcomes for patients. We believe that by altering chemical targets further downstream in the brain's network that regulates movement, we have the potential to help improve outcomes and restore motor function for patients with advanced Parkinson's disease," added Matthew J. During, MD, DSc, scientific co-founder of Neurologix, "

This Phase I trial was the first FDA-approved clinical trial of gene therapy for PD.  It was an open-label, dose-escalation study involving 12 patients with severe PD of at least a five-year duration that is refractory to currently available medical therapies.  As a safety precaution, only one side of their brains was treated.  The trial consisted of three cohorts with four patients each, with the third cohort receiving 10 times the dose of the first.  (Medpage Today, Sept. 27, 2005)  Based on the UPDRS scale, average improvement of the 12 patients receiving treatment was 23%.  Improvement was dose-related with 5 of the 12 patients who received the highest dose showing improvement of 40-60%. (John Mordock, Neurologix CEO, Webcast:  Sixth Annual North American Forum for Investing and Partnering in Biotech)

John Mordock, Neurologix President and CEO stated that the company anticipates beginning a larger Phase 2 study in Parkinson's disease later in 2007.(company press release 6/21/07)

It was announced on Dec. 13, 2007 that the U.S. Food and Drug Administration has granted Fast Track Designation to Neurologix for its “experimental gene transfer procedure” to treat advanced Parkinson’s disease. Neurologix plans to launch Phase II studies of its process by early 2008.

On March 26, 2008, during a year end 2007 financial report, CEO John Mordock stated, "We are now completing steps to initiate a Phase 2 clinical trial of our Parkinson's product in the first half of 2008, subject to clearance from the FDA. " He also stated, "We completed a $15 million private placement in November 2007 for our Series D Convertible Preferred Stock, with participation from both current and new investors. This financing gives us the resources to advance our Parkinson's disease program through a Phase 2 clinical trial and our epilepsy program through a Phase 1 clinical trial." (Business Wire 3/26/08)

It was reported on March 27, 2008 that the FDA approved a Phase II trial, and expected to start recruiting 44 participants in the U.S. during the 2nd quarter of 2008. (BusinessWire 3/27/08)

In December 2009, Neurologix "announced the completion of all planned surgeries in an ongoing Phase 2 clinical trial. The controlled, double blinde blinded, 44 patient Phase 2 trial is investigating the safety and efficacy of a novel non-dopamine approach to restore motor function in Parkinson’s patients who are sub-optimally responsive to available drug therapy. The company expects to announce initial efficacy results from the trial in mid-2010. " (Businesswire 12/03/09) The investigators are assessing each of the trial participants over time for treatment effects, with the primary trial endpoint being a clinical assessment of motor function at 6 months using the Unified Parkinson’s Disease Rating Scale (UPDRS). All participants in the trial will also be monitored for safety for 12 months following their gene transfer procedure.

On June 22, 2010, Neurologix, Inc. "announced positive results in a Phase 2 trial of its investigational gene therapy for advanced Parkinson's disease (PD), NLX-P101. Study participants who received NLX-P101 experienced statistically significant and clinically meaningful improvements in off-medication motor scores compared to control subjects who received sham surgery. In the trial, this benefit was seen at one month and continued virtually unchanged throughout the six month blinded study period...." (Neurologix press release