The gene therapy procedures were performed at NewYork-Presbyterian Hospital/Weill Cornell Medical Center by Michael G. Kaplitt, M.D., Ph.D., the Tara and Victor Menezes Clinical Scholar in Neurosurgery at Weill Cornell Medical College and Director of Stereotactic and Functional Neurosurgery at NewYork-Presbyterian/Weill Cornell, and Dr. Matthew J. During, Professor of Molecular Medicine at the University of Auckland. Drs. Kaplitt and During are the co-founders of Neurologix and are consultants to the company. In a joint statement, Drs. Kaplitt and During commented: "While the primary objective of any Phase I trial is to demonstrate safety, the FDA's requirement of unilateral infusion of AAV-GAD allowed us the unique opportunity to compare treated and untreated sides of the brain. In essence, the untreated side acted as a control. The combined clinical and imaging data provide powerful evidence that this treatment appears to be efficacious, as well as safe, at the current dose levels."
This Phase I trial is the culmination of more than 10 years of basic research. In 1994, Dr. Kaplitt was the first author of a paper published in Nature Genetics, along with Dr. During as senior author, which demonstrated, for the first time in a preclinical model, that AAV could be a safe and effective vehicle for gene therapy in the brain. Most importantly, AAV has never been associated with any human disease.
According to Drs. Kaplitt and During, "The goal of this research is to determine whether we can 're-set' a specific group of cells that have become overactive, causing the characteristic impaired movements associated with Parkinson's disease. The interim UPDRS scores are highly promising and, if they are borne out with additional data, would be comparable to results seen with STN Deep Brain Stimulation. Unlike deep brain stimulation, however, our gene therapy approach is much simpler, can be carried out entirely under local an