Brains at Work Against Parkinson's Disease
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Schneider's research involves giving patients doses of GM1 ganglioside, a molecule that naturally occurs in the lining of brain cells. Animal and human trials, he says, already suggest that the molecule blocks brain cell death and promotes dopamine production.
"The trick is if you can identify patients early on and keep them in a mild state of the disease," he tells WebMD. "Everyone wants to say, 'I've found the cure for Parkinson's,' but 'cure' is maybe not the word. If we can significantly slow the progression of the disease, to me, that's tantamount to a cure." There are three substances now in human clinical trials for brain protection against Parkinson's, he says.
According to Schneider, Parkinson's patients have shown improved movement even four years after starting to take GM1, which seems protective especially against slowed movement and rigidity. Moreover, the medication appears to have few side effects. But lots of questions remain, including how to get GM1 into the brain easily, and how the molecule actually works.
For those patients suffering the disease's full ravages, with most of their dopamine already gone, scientists are looking to restore brain functions. Unfortunately, there have been recent disappointments with several cutting-edge approaches.
The injection of specially altered viruses carrying genes that stimulate growth of nerve cells -- or gene therapy -- may help stimulate the brain to repair itself. But the whole field of gene therapy is struggling in the wake of tightened scrutiny after a teenager died while participating in a University of Pennsylvania clinical trial for another disease.
Still, gene therapy research on monkeys and rats has shown results against Parkinson's.
"This is very exciting," says Martha Bohn, PhD, director of the neurobiology program at Northwestern University Medical School in Chicago.
But she and other researchers worry about the long-term effects of injecting lots of new genes into the brain without the ability to turn them off, if necessary. Questions also remain on where in the brain the genes should go and what kinds of viruses are effective in delivering the genes effectively.
Another hope-filled field to help restore the brain has been stem cell transplants. Long-term improvements have been observed in several dozen patients around the world. But troubling published results came out this March, when American scientists injected dopamine-producing nerve cells from aborted fetuses into Parkinson's patients.