Scripps Florida breakthrough blocks Parkinson's
Palm Beach Post
February 11, 2011
By Jeff Ostrowski
Scientists at Scripps Florida have discovered a way to stop the progress of Parkinson's disease, the brain disorder that afflicts about 1 million Americans and has defied the search for a cure.
Scripps researchers gave mice and rats a pill that successfully stalled the disease's march. In two articles published this week in the journal ACS Chemical Neuroscience, the scientists say they've found a molecule that blocks the enzyme JNK from killing neurons in rodents with Parkinson's.
The drug doesn't reverse the damage of Parkinson's, said Philip LoGrasso, leader of the Scripps Florida team that conducted the research, but it could be "a major breakthrough" for Parkinson's patients.
"It won't cure them," LoGrasso said. "But they would begin taking it and hopefully the disease wouldn't get any worse."
The drug might not be given to humans for years, if ever. But if this invented-in-Jupiter pill finds its way to pharmacies, its success would provide both financial and psychic payback for the $579 million that state and local taxpayers spent to set up the Scripps Florida labs here.
The treatment would be the first to protect the brain from the ravages of Parkinson's. Commonly prescribed drugs - including levodopa and so-called MAO-B inhibitors - counteract Parkinson's without slowing it.
"Everything available treats symptoms but doesn't prevent neurodegeneration," LoGrasso said.
Parkinson's is a degenerative neurological disorder that hampers the brain's ability to produce dopamine. Symptoms include tremors, slow movement and balance problems.
Parkinson's strikes 1 percent of people over 60, according to the Michael J. Fox Foundation. Boxing great Muhammad Ali and actor Michael J. Fox are two prominent victims of the disease.
LoGrasso's team conducted its research with a $7.6 million grant from the National Institutes of Health.
In mice, Scripps' drug - known as SR-3306 - protected 72 percent of dopamine-producing neurons. In rats, the compound saved only 30 percent of neurons, but even that level of protection reduced rats' motor dysfunction by 90 percent, Scripps scientists said.
Now that they've determined SR-3306 works, scientists must test it for side effects. Scripps researchers will begin a year of toxicology testing in animals after they receive their next round of federal funding in July, LoGrasso said.
If those studies go well, the next step would be tests in humans. Parkinson's experts caution that what works for rodents won't necessarily succeed in humans.
"The animal models for Parkinson's disease don't always translate into humans - different species, different brains," said Sami Fam, an analyst at Decision Resources, a drug research firm in Burlington, Mass.
But, he said, Scripps Florida's findings are promising.
"A true neuroprotectant would be a blockbuster," Fam said. "If you could give a drug that protects the neurons in the brain from dying, then that would be great."
Americans spend about $1 billion a year on Parkinson's drugs. Because levodopa is available in generic form, it's inexpensive, Fam said. However, levodopa tends to work for only a few years, so doctors try to delay using it.
LoGrasso said he has talked to private companies about commercializing the Parkinson's treatment, but he wouldn't offer details.
If the drug works in humans, it would be a boon both for Parkinson's patients and for Palm Beach County's burgeoning biotech hub. State and local taxpayers spent $579 million on the Scripps Florida labs in Jupiter, plus $187 million for Max Planck Florida, also in Jupiter.
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