AANS Neurosurgeon | Volume 26, Number 1, 2017

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Study Offers Potential New ALS Treatments

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In a study recently published in the journal Proceedings of the National Academy of Sciences, researchers from the University of North Carolina School of Medicine announced their evidence-based description of the neuronal protein clumps thought to be important in Amyotrophic Lateral Sclerosis (ALS). This research development could be a crucial step toward developing drugs to stop the creation of the clumps and the progression of the disease. Cures for ALS and other neurodegenerative diseases have long eluded researchers, largely because their causes have remained mysterious. “One of the biggest puzzles in health care is how to address neurodegenerative diseases; unlike many cancers and other conditions, we currently have no leverage against these neurodegenerative diseases,” said the study’s lead author. The study focused on a subset of ALS cases – an estimated one to two percent – that are associated with variations in a protein known as SOD1. However, even in patients without mutations in their SOD1 gene, this protein has been shown to form potentially toxic clumps. During the study, the researchers discovered that the protein formed temporary clumps of three, known as a “trimer,” and that these clumps are capable of killing motor neuron-like cells grown in the laboratory. According to researchers, this is a major step because the exact toxic interactions responsible for the death of motor neurons in patients with ALS have been unknown. To read more about this study, click here.

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