Armed with these findings, laboratory scientists and clinicians are now preparing to test gene therapy on humans with PD. Last spring, the National Institutes of Health Recombinant DNA Advisory Committee (RAC) reviewed a PD gene therapy protocol for the first time, endorsing various recommendations by a 12-0 vote. The protocol's investigators, at Jefferson Medical College in Philadelphia and Cornell University's Weill Medical College in New York, recently submitted it to the Food and Drug Administration. Other groups also intend to treat PD patients with gene therapy, but they say they won't seek official approval for months, if not years.
Collectively, these groups have injected at least six candidate transgenes into three brain structures using two types of viruses. But until various clinical trials reveal which gene, if any, works and under what conditions, the researchers' buzzwords are safety and caution. Michael G. Kaplitt, who directs stereotactic and functional neurosurgery at ...
