New ‘Culprit’ Found in Lou Gehrig’s Disease

Following a major Northwestern Medicine breakthrough that identified a common converging point for all forms of amyotrophic lateral sclerosis (ALS and Lou Gehrig’s disease), a new finding from the same scientists further broadens the understanding of why cells in the brain and spinal cord degenerate in the fatal disease. Less than three months ago, Northwestern research found that the crucial recycling system for cells in the brain and spinal cord was broken in people with ALS. And one mutated gene had a key role. Like a loafing worker, it wasn’t doing its job to recycle damaged cells. Now, scientists have discovered a second faulty gene – a new loafing worker – in the same recycling pathway. The finding is reported in the Archives of Neurology.

“Now that we have two bad players, it shines more light on this broken pathway,” said senior author Teepu Siddique, MD, the Les Turner ALS Foundation/Herbert C. Wenske Professor of the Davee Department of Neurology and Clinical Neurosciences at Northwestern University Feinberg School of Medicine and a neurologist at Northwestern Memorial Hospital. “This gives us a clear target to develop drug therapies to try to fix this problem. It strengthens our belief that this broken system is at the heart of ALS.”

New ‘Culprit’ Found in Lou Gehrig’s Disease – News Center (northwestern.edu)

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