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Blocking a key protein may help prevent or slow the debilitating symptoms of Parkinson’s disease, researchers say.
Parkinson’s disease, leukemia, c-abl, nilotinib
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Study may show new path for Parkinson’s treatments

Blocking a key protein may help prevent or slow the debilitating symptoms of Parkinson’s disease, researchers say.

A group of researchers from Johns Hopkins University studied the interactions of a protein called c-Abl with another protein called α-synuclein, and concluded that c-Abl plays a role in the development of the microscopic bodies that can kill brain cells responsible for motor control.

Parkinson’s disease is characterized by uncontrollable tremors, sleep and mood disorders, rigid muscles, loss of balance and speech impairment, among other symptoms.

"There were indications that c-Abl activity leads to Parkinson's disease, and our experiments show there is indeed a connection," said Ted Dawson, M.D., director of the Institute for Cell Engineering at Johns Hopkins. 

Study isolates effects of protein

The research served to separate the role of c-Abl from other proteins by studying its effects in genetically engineered mice. Investigators found that increased levels of c-Abl increased Parkinson’s symptoms, while diminished levels of the protein reduced symptoms.

They plan further research into the interplay of the proteins and how they affect the severity of Parkinson’s.

Could a leukemia drug be repurposed for Parkinson’s?

The U.S. Food and Drug Administration already has approved the use of nilotinib, which blocks the c-Abl protein to combat leukemia. The researchers say they hope to explore whether nilotinib could be useful in the fight against Parkinson’s.

Nilotinib, marketed as Tasigna, is aimed at patients suffering from a form of cancer called Philadelphia chromosome-positive chronic myeloid leukemia (Ph+CML).

The toll of Parkinson’s disease

Parkinson’s is a progressive, degenerative brain disorder believed to afflict about 1 million Americans and 10 million people around the world. It strikes men more often than women. It advances as the brain slowly reduces its production of dopamine, a neurotransmitter.

While it is not fatal in itself, its complications can cause death, as in the case this spring of boxer Muhammad Ali. The National Parkinson Foundation says it is the 14th leading cause of death in the United States.

In its early stages, Parkinson’s doesn’t interfere with daily activities and people can live with the disease for years. But as it progresses, Parkinson’s robs sufferers of their ability to live independently.

There is no known cure, but various medications can help keep symptoms in check.

Deeper reading

The Johns Hopkins study was published in The Journal of Clinical Investigation. You can read a simpler explanation of the research on the Johns Hopkins Medicine website.

You can read about the stages of Parkinson’s disease, the way it is treated and current thinking about how it begins at the National Parkinson Foundation site.

If you are concerned or showing symptoms of Parkinson’s disease, discuss this with your health care provider. You can find a Providence provider here.

Blocking a key protein may help prevent or slow the debilitating symptoms of Parkinson’s disease, researchers say.

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