NEUROLOGIX ANNOUNCES INTERIM RESULTS OF GENE THERAPY CLINICAL TRIAL

Neurologix has announced positive interim results of its landmark gene therapy clinical trial for patients with Parkinson's disease. Neurologix's 12-patient, dose-escalating Phase I trial is the world's first study to use a viral vector (the nonpathogenic adeno-associated virus, or AAV) for the treatment of an adult neurological disease.

In the trial, the vector was injected into a specific target site in the brain in order to transfer a gene to treat Parkinson's disease. The gene encodes glutamic acid decarboxylase (GAD), an enzyme that synthesizes the major inhibitory neurotransmitter in the brain, (gamma)-aminobutyric acid (GABA).

Under the FDA-sanctioned trial protocol, patients with advanced Parkinson's disease received unilateral (one side of the brain) infusion of AAV-GAD via a hair-thin catheter into the subthalamic nucleus (STN), a deep brain structure known to function abnormally in Parkinson's patients. According to the interim findings, Neurologix's STN AAV-GAD treatment appears to be safe and well-tolerated in advanced Parkinson's disease, with no evidence of adverse effects or immunologic reaction related to the study treatment.