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Parkinson's Disease
Working to create the first therapy to stop disease progression

Parkinson's disease is a progressive neurological disorder that affects approximately one million Americans each year. Characterized by muscular tremors, stiffness and slowness, and eventually poor balance control—the disease is caused by loss of nerve cells in the brain that contain dopamine.

While Parkinson's is most often diagnosed in its early stages when its effect on a patient's quality of life is not too significant, available treatments only address its symptoms and nothing is available to stop progression of the disease—frustrating both patients and doctors.

Researchers at Columbia, led by Dr. Lloyd Greene in the Department of Pathology and Dr. Robert Burke in the Department of Neurology, investigated a class of compounds (mixed lineage kinase inhibitors) that have shown promise in animal studies to block a signal pathway that appears to be involved in the programmed death of brain cells.

After one of the new compounds was tested in animals to ensure that it would be safe to give to humans, the FDA approved a large clinical trial, called the PRECEPT trial, to test its safety and efficacy in stopping the progression of disease in 800 Parkinson's disease patients.

"Animal models make it possible for us to study how these neurons slowly die in Parkinson's, in ways that are technically impossible in living human brains," said Dr. Burke. "Animals are necessary because while tissue cultures are important to the research, ultimately these mechanisms are simply not represented by cultures. Additionally, neural systems are completely beyond what our best computers are capable of doing today - each neuron has interactions with billions of other neurons and computers do not yet provide adequate modeling of these complexities."

Based on the results of the PRECEPT trial, Dr. Burke and his team at the Morris Udall Center for Parkinson Disease Research at Columbia University plan to design new studies to confirm their findings or develop new compounds for testing. Funded by the National Institute of Neurological Disorders and Stroke, the Udall Center is continuing its mission to better understand the mechanisms of dopamine neuron degeneration in Parkinson's, aiming to offer new hope to patients and their families by developing therapies to prevent progression of this devastating disease.
© 2005 Columbia University