Scientists have developed a new inhibitor for the treatment of inflammatory diseases. They have come up with a potent, selective, small-molecule inhibitor called MCC950. MCC950 is the inhibitor of NLRP3, a NOD-like receptor (NLR) family, pyrin domain–containing protein 3 inflammasome. NLRP3 inflammasome s a component of the inflammatory process, and its activation is pathogenic in diseases like multiple sclerosis, type 2 diabetes, Alzheimer’s disease and atherosclerosis.
The results were published in Nature Medicine and could prove to be a therapy for various diseases in the future. MCC950 specifically inhibits only NLRP3 and not other inflammasomes. MCC950 reduced interleukin-1β (IL-1β) production in vivo and attenuated the severity of experimental autoimmune encephalomyelitis (EAE), a disease model of multiple sclerosis.
Dr. Rebecca Coll, from UQ’s Institute for Molecular Bioscience, said “Inflammatory diseases result when our immune system is unable to switch off and so causes chronic inflammation in the body. Current therapies for inflammatory diseases, such as asprin, ibuprofen and steroids, don’t work well in severe cases and are not targeted, which can limit their effectiveness and cause side-effects. We now know that MCC950 can block an important component of the immune response — an inflammasome called NLRP3 that ‘switches on’ inflammation in our immune cells.”
Researchers hope that this development would help in treating patients diagnosed with Cryopyrin-Associated Periodic Syndromes (CAPS), a family of rare and severe autoinflammatory diseases caused by a genetic mutation to NLRP3. Professor Luke O’Neill, Co-author and from Trinity College Dublin said, “We are excited about MCC950, which we believe has real potential to benefit patients suffering from several highly debilitating diseases, where there is a dire need for new medicines.”