US clinical-stage biotechnology company Neurologix Inc (OTC: NRGX) said today it has completed all planned surgeries in an ongoing Phase II study of its gene transfer approach to the treatment of advanced Parkinson's disease.
The controlled, double-blind, 44-patient Phase II 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.
CEO John Mordock said that the company'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, added Mordock and continued that scientists have determined this neurotransmitter to be deficient in the brains of patients in the advanced stages of Parkinson's disease.
Parkinson's disease is a progressive disorder caused by degeneration of nerve cells in the part of the brain that controls movement. The diagnosis of Parkinson's disease depends on the presence of one or more of the four most common motor symptoms of the disease, namely: resting tremor, slow movement, rigidity and postural instability. Secondary and non motor symptoms are increasingly recognized by doctors as important to treating Parkinson’s disease.
Showing posts with label gene transfer. Show all posts
Showing posts with label gene transfer. Show all posts
Friday, December 11, 2009
Subscribe to:
Posts (Atom)