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Common cause of ALS is protein recycling breakdown: New discovery

Kathleen Blanchard's picture

Researchers from Northwestern University say they have made breakthrough in understanding the cause of ALS, or Lou Gehrig’s disease in a new discovery. In all cases, the disease occurs as the result of a breakdown of protein recycling in the brain and spinal cord.

The disease, researchers found, causes protein breakdown, which are needed for neurons to function normally. ALS occurs because the proteins are damaged and unable to repair themselves.

For years, scientists have been mystified about the root cause of ALS, making it impossible to find treatment for the disease.

ALS breakthrough could mean new treatment

“We can now test for drugs that would regulate this protein pathway or optimize it, so it functions as it should in a normal state, said senior author Teepu Siddique, M.D., the Les Turner ALS Foundation/Herbert C. Wenske Professor of the Davee Department of Neurology and Clinical Neurosciences at Northwestern's Feinberg School.

The finding, which is published in the journal “Nature”, points to a common pathway of Lou Gehrig’s disease that occurs whether the disease is inherited, sporadic or familial.

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Siddique says the study opens up a “new field” for ALS drug therapy.

Researchers from Feinberg School of Medicine also discovered the protein ubiquilin 2 malfunctions in patients with ALS.

Ubiquilin 2 should normally act to remove damaged proteins in the motor neurons of the brain and spinal cord where they can be recycled and repaired.

In patients with Lou Gehrig’s disease, the protein lingers to cause damage; taking on the appearance of “twisted skeins of yarn”, according to the scientists.

The study authors say the ALS discovery is also applicable to other forms of neurodegenerative diseases, including Alzheimer’s disease, other types of dementia and Parkinson’s disease.

Siddique says finding a common cause for ALS has been difficult. Understanding the protein recycling breakdown can help researchers find drug therapy to remove degraded proteins from neurons that causes Lou Gehrig’s and other neurodegenerative diseases that share the same mechanism.

Image credit: Wikimedia commons



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