TUCSON, Ariz. (13 News) – Dr. Xinglong Wang, Professor at R. Ken Coit College of Pharmacy for University of Arizona, came to the Tucson Now newsdesk to discuss with Makalya Goos to discuss his, and other researchers, breakthrough research in the fight against ALS.
University of Arizona researchers say a new experimental drug may open a promising path toward slowing ALS, a fatal neurodegenerative disease that still has no cure. In a Nature Aging study, the team focused on TDP-43, a protein that abnormally clumps inside nerve cells in nearly all ALS cases. Instead of trying to clear those clumps, researchers identified a small, highly conserved region of TDP-43 that appears to drive harm, and showed that disabling that region reduced nerve cell death without disrupting the protein’s normal function.
They then tested an experimental compound called XL20, designed to bind that target region. Importantly, XL20 crossed the blood-brain barrier. In mice, the drug extended median survival by about a week and reduced muscle weakness; in lab tests using human motor neurons, it also reversed some disease-like damage…