Rodent of the Week: Good in mice, a drug for Parkinson’s moves ahead

A drug that could halt the progression of Parkinson’s disease is successful in mice and is now being evaluated in human patients, researchers reported this week.

The drug is phenylbutyrate, which is already on the market as an orphan drug for treatment of infants with a rare genetic disorder called urea cycle disorder. Research at the University of Colorado School of Medicine shows that the medication turns on a gene that can protect dopamine neurons. It’s the destruction of nerve cells that produce dopamine that leads to the stymptoms of Parkinson’s disease, such as loss of movement. The gene that’s targeted, called DJ-1, is crucial in keeping brain cells healthy and functioning.

The gene was discovered in 2003. The Colorado researchers then began searching for a substance to turn on the gene. They identified phenylbutyrate and put it in the drinking water of mice who were genetically programmed to develop Parkinson’s disease as they aged. The mice who received the drug had no decline in physical or mental function and their brains did not show evidence of Parkinson’s disease.

“Drugs currently used to treat Parkinson’s disease just treat symptoms; they do not stop the disease from getting worse,” Dr. Curtis Freed, the lead author of the study, said in a news release. “We’ve now discovered that we can prevent the progression of the disease by turning on a protective gene in the brain.”

The medication is being given to some patients in order to test its safety. So far, studies suggest it has to be given in large doses. The drug is also being tested for the treatment of ALS (Lou Gehrig’s disease).

The study was published in the Journal of Biological Chemistry.

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