In a major breakthrough in the battle against Parkinson's disease, a research team at the University of Colorado School of Medicine has discovered a drug (phenylbutyrate) that stops the progression of the degenerative illness in mice and is now being tested in humans. "Drugs currently used to treat Parkinson's disease just treat symptoms; they do not stop the disease from getting worse," said senior author Dr. Curt Freed, who heads the division of Clinical Pharmacology and Toxicology at the CU School of Medicine. "We've now discovered that we can prevent the progression of the disease by turning on a protective gene in the brain." The results were published online on March 3, 2011, in the Journal of Biological Chemistry. Lead author Dr. Wenbo Zhou, Assistant Professor of Medicine, and Dr. Freed, a national pioneer in Parkinson's research, and colleagues have found that the drug phenylbutyrate turns on a gene that can protect dopamine neurons in Parkinson's disease. The gene, called DJ-1, can increase production of antioxidants like glutathione to reduce the debilitating effects of excess oxygen in brain cells. In addition, activating DJ-1 helps cells eliminate abnormal proteins that otherwise accumulate and kill brain cells. Dopamine neurons are particularly susceptible to too much oxygen and abnormal protein deposits. Parkinson's disease is caused by dying midbrain dopamine neurons. Dr. Zhou and Dr. Freed have studied the DJ-1 gene since 2003 when a European group discovered that mutations in DJ-1 could cause Parkinson's disease. The Colorado scientists immediately started work to see why the gene was so important and have published a series of papers on the subject since 2005. But to convert their findings into a practical treatment for Parkinson's disease, they needed to find a drug to turn on the DJ-1 gene.
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