AANS Neurosurgeon | Volume 25, Number 4, 2016

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This Man Is Revolutionizing Our Understanding of Motor Neuron Diseases and Dementias

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It was when Xinglong Wang, PhD, received a call from a desperate father of a middle-aged son with amyotrophic lateral sclerosis (ALS) that he realized the extraordinary importance of his work. “Can you help save my son’s life?” the parent asked. On that day, Wang, assistant professor of pathology at Case Western Reserve University School of Medicine, felt the weight of human suffering on his shoulders. But this is a weight that Wang can bear. He may be in the process of entirely upending the current scientific view of ALS and other neuronal diseases. He may be a pioneer who forces scientists to discard years of medical dogma and leads clinicians to significantly improved treatments. Shortly before Wang received that call, he had published a paper in which he and his colleagues had shown that the symptoms of ALS in mice could be completely reversed by the infusion of a small-molecule peptide, PM1. Wang showed that PM1, an inhibitor of a mutated, dysfunctional protein, TAR DNA-binding protein 43 (TDP-43), could alleviate mitochondrial dysfunction and neuronal loss, and could significantly improve motor and cognitive function in previously impaired mice.

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