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Parkinson's Patient Gets Gene Therapy

Daring Treatment Would Improve Symptoms, Slow Disease

Parkinson's Gene Therapy vs. Deep Brain Stimulation

The STN isn't a new target for Parkinson's treatments. Because it goes haywire, one idea is to surgically sever its connections to the rest of the brain.

Another idea is deep brain stimulation. This is a thin electrode implanted into the STN. Electric current sent through the electrode quiets the STN.

Kaplitt says the gene therapy should work even better than deep brain stimulation. It should not only calm the STN, but it should also make the STN send out calming signals to other brain regions involved in Parkinson's disease. This is likely to protect brain cells from getting worn out -- thereby slowing the relentless progress of Parkinson's.

These possibilities are why Hauser thinks the gene therapy is a good idea.

"A very realistic outcome to hope for is that this treatment would be similar to the deep brain stimulator," he says. "If so, it is worth the risk. The best to hope for is that this would work better than the deep brain stimulator, so that people won't fluctuate up and down. That is possible."

Assuming no safety issues arise, a second patient will get the Parkinson's gene therapy in September. As long as things go well, there will be one new patient each month for a year.

"We've been working on this 15 years," Kaplitt says. "We've tried very hard to bring this to the public in a rational way. We're not claiming to have a cure. We are just trying to make things better for our patients."

The gene therapy is owned by Neurologix Inc. of Newark, Del. Kaplitt serves as a consultant to Neurologix.


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