Tucson Now: A groundbreaking advancement in ALS coming from UofA

University of Arizona researchers have identified a possible new way to slow amyotrophic lateral sclerosis, or ALS, a fatal neurodegenerative disease with no cure.

The team focused on TDP-43, a protein that forms abnormal clumps inside nerve cells in nearly all ALS cases. Rather than removing the clumps, researchers targeted a small section of the protein linked to nerve cell damage. Disabling that section reduced cell death while preserving TDP-43’s normal function.

Researchers tested an experimental drug called XL20, which is designed to attach to that target area and can cross the blood-brain barrier. In mice, XL20 extended median survival by about a week and reduced muscle weakness. Tests using human motor neurons also showed that the drug reversed some damage associated with the disease.

Dr. Xinglong Wang, a University of Arizona pharmacy professor, discussed the research on Tucson Now. The researchers said earlier detection and treatment could improve the drug’s potential benefits. They also said the approach may eventually apply to other dementias linked to TDP-43, including LATE and some cases of Alzheimer’s disease.