New Protein Discovery Could Mean New Treatment For Parkinson's Disease

First Posted: Jun 29, 2016 08:59 AM EDT

Researchers have revealed that inhibiting the activity of a particular protein in the brain could stop or slow down the development of Parkinson's disease. So, it may be safe to say that new treatments for the disease are underway. What's more, there is already a drug approved for the treatment of leukemia that can block the protein in question.

According to, researchers at Johns Hopkins said they have picked out two very important new clues in the fight against the degenerative disease. The two new clues were: blocking an enzyme called c-Abl stops the disease in specially bred mice, and that a chemical tag on a second protein can signal the disorder's presence and progression.

"There were indications that c-Abl activity leads to Parkinson's disease, and our experiments show there is indeed a connection," says Ted Dawson, M.D., Ph.D., professor of neurology and director of the Institute for Cell Engineering at the Johns Hopkins University School of Medicine. "There is already a Food and Drug Administration-approved c-Abl inhibiting drug in use for leukemia," he adds, "so we're interested in whether it could be used safely against Parkinson's disease or as a starting point to develop other treatments."

Autopsies showed that c-Abl is especially active in the brains of people with Parkinson's disease, which is a progressive disorder of the nervous system that affects voluntary movement. However, Eurekalert reported that Han Seok Ko, Ph.D., assistant professor of neurology at Johns Hopkins noticed that the drugs that were used in those studies were also blocking similar proteins, so it is hard to determine if the beneficial results were only c-Abl inhibition.

To find out more about the case, the team conducted experiments on mice that were specially made to develop Parkinson's disease and eliminate the gene for c-Abl, which resulted to a reduce in the symptoms of their disease. However, researchers found that increasing the amount of the said enzyme worsened and hastened the progress of the disease, Medical News Today reported.

Then, researchers identified the basic mechanism which c-Abl appears to cause the degenerative disease. They discovered that c-Abl functions with another protein known as α-synuclein which accumulates in the brains of people with Parkinson's disease, forming clumps known as Lewy bodies.

Previous research has claimed that a buildup of α-synuclein usually results in the death of brain cells related to motor control. The authors said that these findings indicate that blocking c-Abl activity could prevent or slow the development of PD.

Dr. Dawson also observed that there is already a c-Abl-inhibiting drug that had been approved by the U.S. Food and Drug Administration (FDA) for the treatment of leukemia. The team now plans to investigate whether this drug might show promise to prevent Parkinson's disease, or whether it could lead them to other possible treatments.

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