Neurologix Completes All Surgical Procedures in Its Clinical Trial for Parkinson’s Disease
News Dec 04, 2009
Neurologix, Inc. has announced the completion of all planned surgeries in an ongoing Phase 2 clinical trial of the company’s gene transfer approach to the treatment of advanced Parkinson’s disease.
The controlled, double 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.
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.
“We are very pleased the trial sites have completed all of the surgical procedures for this important study,” said John Mordock, President and Chief Executive Officer of Neurologix. “This represents a significant milestone in the development of a potential new treatment option for the greatly underserved population of patients with advanced Parkinson’s disease.”
All surgical procedures were performed at the following institutions: Henry Ford Health System, Massachusetts General Hospital, The Ohio State University, Stanford University, University of Colorado, University of Rochester and Wake Forest University Health Sciences.
Mr. Mordock commented, “We are extremely grateful to the patients, physicians and institutions for their strong support in reaching this milestone in a timely fashion, and we look forward to getting our initial look at efficacy from the trial around mid 2010.
“In anticipation of the continued development of this new therapeutic approach,” he continued, “we are now making preparations for a Phase 3 clinical trial, which we will seek to conduct under a Special Protocol Assessment (SPA).”
Mr. Mordock noted that Neurologix’s gene therapy approach to Parkinson’s aims to provide a safer and more effective alternative to either dopamine replacement therapies or deep brain stimulation for patients with advanced stages of the disease.
Neurologix’s strategy is to deliver a gene (GAD) that reestablishes the production of GABA (gamma-aminobutyric acid), the major brain inhibitory neurotransmitter that helps “quiet” excessive neuronal firing. Scientists have determined this neurotransmitter to be deficient in the brains of patients in the advanced stages of Parkinson’s disease.
“Our Phase 1 clinical trial demonstrated an encouraging improvement in clinical symptoms of Parkinson’s disease along with a reduction in abnormal brain function as demonstrated through PET imaging,” Mr. Mordock noted.
“We are hopeful that this larger, Phase 2 treatment trial may further demonstrate our approach to be effective for improving motor symptoms and brain function in late-stage Parkinson’s disease patients while avoiding the further surgical risks and limitations of deep brain stimulation and the complications associated with over-production of dopamine often associated with currently used pharmacologics.”
“If successfully developed, this gene transfer procedure could become the first significant advance for patients with late-stage Parkinson’s disease since the introduction of deep brain stimulation.”