South Asian-American Doctor Headed Research Leads To ALS Breakthrough

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Northwestern Medicine study headed by Dr. Teepu Siddique for the first time has identified a common cause of all forms of ALS.

CHICAGO — The underlying disease process of amyotrophic lateral sclerosis (ALS and Lou Gehrig’s disease), a fatal neurodegenerative disease that paralyzes its victims, has long eluded scientists and prevented development of effective therapies.

Now, a new Northwestern Medicine study headed by Dr. Teepu Siddique for the first time has identified a common cause of all forms of ALS.

The basis of the disorder appears to be a broken down protein recycling system in the neurons of the spinal cord and the brain. Optimal functioning of the neurons relies on efficient recycling of the protein building blocks in the cells. In ALS, that recycling system is broken. The cell can’t repair or maintain itself and becomes severely damaged.

The discovery by Northwestern University Feinberg School of Medicine researchers, published in the journal Nature, provides a common target for drug therapy and shows that all types of ALS are, indeed, tributaries, pouring into a common river of cellular incompetence.

“This opens up a whole new field for finding an effective treatment for ALS,” senior author Siddique, the Les Turner ALS Foundation/Herbert C. Wenske Professor of the Davee Department of Neurology and Clinical Neurosciences at Northwestern’s Feinberg School and a neurologist at Northwestern Memorial Hospital, said in a press release.

”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.”

The discovery of the breakdown in protein recycling may also have a wider role in other neurodegenerative diseases, specifically the dementias. These include Alzheimer’s disease and frontotemporal dementia as well as Parkinson’s disease, all of which are characterized by aggregations of proteins, Siddique said. The removal of damaged or misfolded proteins is critical for optimal cell functioning, he noted.

In related research, Feinberg School researchers also discovered a new gene mutation present in familial ALS and ALS/dementia, linking these two forms of the disease.

Siddique has been searching for the causes and underlying mechanism of ALS for more than a quarter century. He said he was initially drawn to it because “it was one of the most difficult problems in neurology and the most devastating, a disease without any treatment or known cause.”

“These people in the prime of their lives and the peak of their productivity get this devastating illness that kills them,” Siddique said. “The people who get ALS/dementia, an even more vicious disease, have a double whammy.”

The study was supported by the National Institute of Neurological Disorders and Stroke, the Les Turner ALS Foundation, the Herbert and Florence C. Wenske Foundation, the Blazeman Foundation for ALS and other sources.