Brains at Work Against Parkinson's Disease
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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.
Not only did the cell injections produce no average improvement in the functioning of the patients, but four patients who received the treatment developed "severe and disabling" movement disorders, according to neurologist Paul Greene, MD, one of the trial's scientists.
As Greene showed haunting videos of these patients, he said he wasn't sure what prompted the new disorders. But he said he was cheered that the fetal cells do appear to be surviving in patients' brains -- and producing dopamine. "The outcome might be different with other techniques" of fetal cell injection, he said.
Creating a reliable therapy will take lots of hard work, but some are optimistic.
"This is a very crude technology at present, but in the next five years it will be totally different," Ole Isacson, DrMedSci, a researcher at Harvard Medical School, tells WebMD.
There is also lots of talk of stem cells, the 'baby' cells in the body that are capable of turning into any specialized type of cell. Researchers have been able to encourage stem cells to multiply and specialize into various functions. Scientists say the most promising avenue is research with cells from discarded human embryos, but President George W. Bush has put federal funding for fetal stem cell research in limbo.