Potential Parkinson'S Disease Cure

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Kirk Monroe, 202/872-4445 I
MPORTANT NOTE: For information of the clinical trials please call Guilford Pharmaceuticals at 410/631-6449

POTENTIAL PARKINSON'S DISEASE CURE

Drug repairs brain's nerve cells, banishes most disease symptoms in animal trials

Washington, DC -- Parkinson's disease may be reversible if a drug that has worked in animal tests is as successful in humans. Results with the compound will be presented at the American Chemical Society national meeting in San Francisco on April 15, and were recently published in a scientific journal.* The drug, developed by Guilford Pharmaceuticals of Baltimore, represents a "tremendously exciting" advance, says Dr. Jonathan Pincus, M.D., a Georgetown University physician who is a Parkinson's disease expert. Pincus says this is "the first time there has been a compound that could get into the brain and stimulate nerve growth."

Parkinson's patients, who include boxer Muhammad Ali, develop tremors, muscle weakness and a shuffling gait as the nerve cells (neurons) in the brain involved in motor control are gradually destroyed. More than 1.5 million Americans suffer from the disease, which ultimately renders patients entirely rigid. The drug works by regenerating damaged neurons, resulting in more than 90 percent recovery of normal behavior in animal trials, says Dr. Gregory Hamilton, principal scientist in Guilford's research department. "What we're really excited about is that these compounds don't just slow down progression of the disease." The animal trials suggest "we may be able to take patients who have begun to show the outward manifestations of Parkinson's disease -- the tremor, the motor-control deficits -- and push them back over the threshold into normal behavioral function. We hope to reverse the disease and not simply slow it down." Hamilton believes this reversibility is "completely unpreceden

So far the compound, along with a number of similar analogs, has been successfully tested in rats and mice, without turning up any problems with toxicity or side effects. They are now being examined in rhesus monkeys, and Hamilton says human clinical trials might begin at the end of this year or early next year. The compounds, which can be administered orally, are small molecules termed neuroimmunophilin ligands. They bind to cellular proteins known as immunophilins.

Intriguingly, the animal tests show these compounds can regenerate other types of damaged nerves, without affecting normal, healthy neurons. Hamilton says that means these drugs could potentially treat nerve damage in conditions such as diabetes, carpal tunnel syndrome or Bell's palsy. Further, the compounds "protect other types of neurons in the brain against toxic damage," Hamilton says. So Guilford is looking into their impact on Alzheimer's disease, with "encouraging initial results." Other potential targets include multiple sclerosis, traumatic head and spinal cord injuries and stroke. Hamilton adds that "it is our feeling at this point that almost any disease that involves chronic degeneration of nerves may be affected in a positive manner by these compounds. They appear to have an extremely general effect to promote regeneration of damaged nerves."

*Proceedings of the National Academy of Sciences, Vol. 94, No. 5, pp. 2019-24

# # # # MEDI 172 will be presented at 3:10 p.m., Tues., April 15, in Room 114, Exhibit Level, Moscone Center. The national meeting of the American Chemical Society will be held in San Francisco, April 13-17. This paper is among 7,700 presentations to be made.

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The American Chemical Society, founded in 1876, is the world's largest scientific society, with more than 151,000 members.