Following treatment, patients were evaluated at 1, 3, 6 and 12 months. These evaluations included scoring via the UPDRS, neuropsychological testing, videotaped examinations and timed motor tasks. PET scans were also taken at baseline, 6 and 12 months.
The Gene Therapy Procedure
The surgery entailed a stereotactic neurosurgical procedure performed under local anesthesia with the patient awake. First, MRI was used to image the target subthalamic nucleus (STN) region of the brain. The STN was mapped using microelectrodes by recording from single neurons as the electrode was slowly moved towards the STN. Once a signature firing pattern was obtained confirming that the electrode was in the STN, the fine-wire electrode was removed, leaving only the microelectrode sheath through which a hair-thin (165 micron) hollow tube was inserted. Thirty-five microliters containing 3.5, 10 or 35 billion particles (depending upon dose cohort) of the AAV (adeno-associated virus) viral vector with a human GAD gene (cDNA) were then infused at 0.5 microliters/minute, together with 15 microliters of 25% mannitol. After the 100-minute infusion period, the delivery catheter was withdrawn and the incision closed. No hardware was left behind following this procedure, and all patients were discharged within 48 hours of the procedure.
Neurologix, Inc. is a development-stage company, which through its subsidiary, Neurologix Research, Inc., is engaged in the research and development of proprietary treatments for disorders of the brain and central nervous system, primarily uti