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Parkinson's Disease Health Center

Parkinson's Gene Therapy Seems to Work

Patient: 'If You Didn't Know I Had Parkinson's Disease, You Couldn't Tell'
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A Pacemaker for the Brain continued...

It doesn't work right away. It takes time for GAD levels to build up.

"Nothing happened for the first week. And then nothing happened for the second week. And then a month, and two months, and then at three months I thought I was a little better, nothing much," Klein says. "It was like watching grass grow. But about six months later, I started feeling a lot better."

During says that Klein's movement problems got about 40% better after treatment. Not all patients have done that well. But those who got the highest dose tended to get the most improvement.

One year after treatment, nine of the 12 patients showed an average improvement of 37% on a measure of Parkinson's disease severity. Five patients had between 40% and 65% improvement.

"We are encouraged the results seem to be maintained over time," During says. "When we look at the other side of the brain, the side we did not treat, we always see worsening of disease. This is a progressive disease. If we hadn't done the treatment, we would expect these patients to be doing significantly worse."

Does It Really Work?

These very encouraging results don't prove anything, During notes. That will depend on a large clinical trial in which participants aren't aware of what treatments they receive and some participants receive no active treatment (placebo). A trial like this is in the planning stages.

Until then, it's just another promising treatment, says Parkinson's researcher Curt R. Freed, MD, head of the division of clinical pharmacology and toxicology at the University of Colorado Health Science Center.

"It is promising at this stage, but like everything else, the proof will be in the pudding," Freed tells WebMD. "Everything will depend on their clinical trial."

Freed should know. Twenty years ago, he and others started getting intriguing results when they implanted fetal brain cells into the brains of people with Parkinson's diseaseParkinson's disease. These transplanted cells are intended to replace the dopamine-producing cells that die off in Parkinson's disease.

Only about 3% of those transplanted cells survive. But those that survive, Freed reported at the Neuroscience conference, take root and grow -- and keep on growing. If at least 40,000 of the cells survive, it means patients no longer have to take L-dopa, the most important Parkinson's treatment.

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