Exciting Yet Cautious Hope For Parkinson's Sufferers

We are beginning to see the very first attempts at gene manipulation therapies for curing dieases. It this therapy for Parkinson's a retro virus, sort of an intelligent nanoscopic pair of scissors, find's it's way into the exact correct spot to alter a gene. Let's hope it works. Parkinson's is an age related disease but can affect the young. Michael Jay Fox is widely known to suffer from the disease.

Gene-Therapy Tried for Parkinson's

BY LAURAN NEERGAARD, AP Medical Writer

4 hours ago

WASHINGTON - 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."

Parkinson's disease gradually destroys brain cells that produce dopamine, a chemical crucial for the cellular communication that controls muscle movement. As dopamine levels drop, symptoms increase: tremors in the arms, legs and face; periodically stiff or frozen limbs; slow movement; impaired balance and coordination. It afflicts about 1.5 million Americans.

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Gene therapies remain controversial and may for quite some time. It's partly from misunderstanding the science and partly from personal differences held regarding what is ethical to do in changing genes that could possibly affect future generations. This is not that kind of therapy as I understand it. Hopefully people will reserve judgment until some positive results may be witnessed spread large and wide. Perhaps then people will calm down some and consider the fact that there but for the grace of chance or whatever, they too could need such a radical intervention. It's always easy to pontificate while one is young or healthy and the bad things only happen to others.