Gene-Therapy Tried For Parkinson’s

Mike Castle lay motionless as surgeons drilled two holes into his skull and injected a virus deep into his brain. The virus carries a gene and a tantalizing hope: that just maybe it could stall the Parkinson’s disease slowly crippling him. The Illinois man is among a few dozen patients enrolling in the first attempts at gene therapy for Parkinson’s, a milestone in the quest to better treat the degenerative brain disease.It’s a time of mixed excitement and caution: These first three studies are to see if gene therapy is safe to try, not to prove whether it works. Yet studies in monkeys suggest at least one of the approaches has the potential to finally target the underlying disease, not merely tame its symptoms."It’s this delicate balance between giving (patients) hope but making it clear to them, and to the world, that this is still highly experimental,” says Dr. William J. Marks Jr. of the University of California, San Francisco, who is leading the most closely watched approach — using a nerve growth factor to rescue dying brain cells."It’s a gamble,” agrees Dr. Leo Verhagen of Chicago’s Rush University Medical Center, a co-researcher in the project who treated Castle."This is the first trial that, if it works, could slow down the disease’s progression,” he explains.Yet to stress the experiment’s unknowns, he bluntly told Castle, “We are happy if we don’t make you worse.”

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