Washington, Mar 9 (ANI): Researchers at the University of Colorado School of Medicine have discovered a drug that stops the progression of Parkinson's disease in mice and is currently being tested on humans.
Senior author Curt Freed, who heads the division of Clinical Pharmacology and Toxicology at the CU School of Medicine, said that the drugs currently used to treat Parkinson's disease just treat the symptoms, but do not stop the disease from getting worse.
"We've now discovered that we can prevent the progression of the disease by turning on a protective gene in the brain," he said.
Lead author Wenbo Zhou, assistant professor of Medicine, and Freed, a national pioneer in Parkinson's research, 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. Dying midbrain dopamine neurons causes parkinson's disease.
Zhou and 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.
After testing many drugs, the team found that phenylbutyrate could activate DJ-1 and keep dopamine neurons from dying.
Next, they put the drug in the drinking water of mice genetically programmed to get Parkinson's disease as they aged.
Aging mice receiving the drug were able to move normally, had no decline in mental function, and their brains did not accumulate the protein that causes Parkinson's.
By contrast, older animals that did not get the drug saw a steady decline in their ability to move as abnormal proteins damaged their brains.
The researchers began giving phenylbutyrate to people in 2009, to test the safety of the drug in Parkinson patients.
Zhou and Freed believe the discovery offers new hope for those suffering from Parkinson's disease.
The results have been published online in the Journal of Biological Chemistry. (ANI)
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