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Researchers find promising therapy for Parkinson's
February 6, 1997
Web posted at: 10:30 p.m. ESTIn this story:
- Therapy may also work on other diseases
- Years before it can be tried on humans
- Related stories and sites
(CNN) -- The trembling hands and shuffling feet of people with Parkinson's disease are the result of a communications breakdown in the brain.
The problem is that brain cells die, depriving the body of a chemical called dopamine that is critical for delivering messages from one nerve cell to the next.
Why they die is a mystery, but researchers say they have used gene therapy in rats to protect nerve cells that make the crucial chemical.
A University of Rochester team used a modified version of the common cold virus to introduce into rats' brains a gene that produces a protein messenger that protects cells that make dopamine.
The experiments, which are reported in Friday's edition of the journal Science, is not a cure for the disease. But, as Rochester neurobiologist Martha Bohn puts it, "It's an excellent first step.
Therapy may also work on other diseases
"Ultimately," she said, "we may be able to develop this approach for human disease. Not only Parkinson's disease, but other neuro-degenerative diseases."
Bohn is now working on other ways to deliver the gene, and will also test the approach on monkeys, who provide a better model of Parkinson's.
Parkinson's targets a tiny area deep in the brain stem, where nerve cells produce dopamine.
Using a gene that produces a potent chemical called GDNF, researchers injected it into the damaged brains cells of rats. The damaged cells began producing GDNF, which nerve cells need to stay healthy and to produce dopamine.
Given this treatment, rats with a condition like Parkinson's lived significantly longer than those that did not.
"We are putting in less than a billionth of an ounce of GDNF protein into the injection site," Bohn said, "so it's a fairly non-invasive procedure. Very low levels of this protein in a very focused area of the brain, and we still get very remarkable effects."
Years before it can be tried on humans
Bohn said it will be years before researchers will be able to try such therapy on humans, but that in time it could offer a major advance. Current treatments stave off the symptoms of Parkinson's, but don't prevent the destruction of dopamine cells.
"The exciting part of this finding," she said, "is that we know you can put the gene into the right part of the brain and get GDNF secreted continuously at low levels," she said.
"So if we can develop this approach to slow down the death of these nerve cells, then we will have made a milestone step."
Correspondent Andrew Holtz and Reuters contributed to this report.
Related story:
- Gene therapy no quick fix - September 13, 1996
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