Suppression of ALS-related TDP-43 aggregation by VCP inhibitor

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that causes degeneration of upper and lower motor neurons, resulting in muscle weakness and eventual death within 2-5 years after diagnosis. Motor neurons in more than 90 % of the ALS patients contain TAR DNA binding protein-43 (TDP-43) aggregation which is thought to be critical for ALS pathogenesis. However, the molecular mechanism by which this aggregate is formed remains poorly understood.