Novel Small-molecule Inhibitor of NLRP3 Inflammasome as Possible Treatment for Amyotrophic Lateral Sclerosis

Abstract

Objectives: The nucleotide-binding domain leucine-rich repeat and pyrin domain containing receptor protein 3 (NLRP3) inflammasome is over activated in neurodegenerative disorders such as Amyotrophic Lateral Sclerosis (ALS), leading to chronic inflammation by activating several cytokines. A novel small-molecule inhibitor C77 was described to block NLRP3 inflammasome, therefore our study is focused in optimizing and understanding the capabilities of the small-molecule inhibitor for possible treatment of ALS. We hypothesize that C77 can cross the BBB and the muscle, therefore blocking NLRP3 inflammasome and slow disease progression and mortality.