The trial is based on the research of University of Auckland's (Auckland, New Zealand) Matthew During and Weill Cornell Medical College's (New York, NY) Michael Kaplitt, who have published work showing that the adeno-associated virus can deliver theGAD gene to cells in the subthalamic nucleus (STN), an area of the brain that is overactive in PD (BN 10/11/02, p.1). Twelve patients with severe PD of at least five years' duration that is not responding to current therapies will be recruited and followed by doctors at North Shore University Hospital Movement Disorders Center (Manhasset, NY). The GAD genes are delivered via catheter in a 90-minute infusion of 3.5 billion virus particles. Once inside the nerve cells, GAD begins producing gamma-aminobutyric acid (GABA), which inhibits cell activity and may lessen the impaired movements and other symptoms of PD. Preclinical studies also suggested silencing the STN may arrest or delay disease progression.
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