Gene therapy for Parkinson’s advances
Brain surgery to insert genetic cargo improves movement for some
By Nathan Seppa
Using brain surgery to insert replacement genes, doctors can alleviate some movement problems in people with Parkinson’s disease. While not all of the gene therapy recipients in a new study improved, the group on average registered tangible gains after getting a gene that revs up production of a much-needed neurotransmitter, researchers report in an upcoming issue of Lancet Neurology.
Notably, none of the patients had significant side effects attributable to the therapy.
“The pendulum on gene therapy has really swung back and forth,” says study coauthor Matthew During, a physician and neuroscientist at Ohio State University in Columbus. “It was enormously hyped at first.” But the death of a patient in Philadelphia in 1999 and the appearance of leukemia in children in France getting gene therapy for an immune disorder — leading to a temporary suspension of trials in 2003 — stalled the research. “The field languished for a while,” During says.
But he and his colleagues have continued to pursue the technology, using a disabled, nonpathogenic virus as the delivery vehicle for potentially useful genes. To treat Parkinson’s disease, the team has targeted a troublesome part of the brain where signaling gets obstructed in patients with the neurological disorder.