Researchers at Trinity College Dublin have discovered what they term a “marvel” molecule that blocks a key driver of inflammatory diseases. The finding could meet a major unmet clinical need by inspiring new noninvasive treatments for arthritis, multiple sclerosis, and Muckle-Wells syndrome, among a myriad of other inflammatory diseases, note the scientists

In a study (“A small-molecule inhibitor of the NLRP3 inflammasome for the treatment of inflammatory diseases”) published in Nature Medicine, the international research team led by Trinity and the University of Queensland Australia showed how the molecule MCC950 can suppress the NLRP3 inflammasome, which is an activator of the key process in inflammatory diseases.

“MCC950 is thus a potential therapeutic for NLRP3-associated syndromes, including autoinflammatory and autoimmune diseases, and a tool for further study of the NLRP3 inflammasome in human health and disease,” wrote the investigators.

Inflammasomes have been identified as promising therapeutic targets by researchers over the last decade. Now, add the researchers, the discovery of MCC950's abilities represents a hugely significant development in the effort to find treatments for inflammatory diseases, for which current therapies are either highly ineffective or have major limitations.

Crucially, the finding also confirms that inflammatory diseases all share a common process, even though the part of the body becoming inflamed might differ.

“Drugs like aspirin or steroids can work in several diseases, but can have side effects or be ineffective,” explains Luke O’Neill, Ph.D., professor of biochemistry at Trinity. “What we have found is a potentially transformative medicine, which targets what appears to be the common disease-causing process in a myriad of inflammatory diseases.”

“MCC950 is blocking what was suspected to be a key process in inflammation,” says Rebecca Coll, Ph.D., lead Trinity author on the paper. There is huge interest in NLRP3 both among medical researchers and pharmaceutical companies and we feel our work makes a significant contribution to the efforts to find new medicines to limit it.”

According to Matt Cooper, Ph.D., chemist and co-senior author from the University of Queensland's Institute for Molecular Bioscience (IMB), MCC950 is able to be given orally and will be cheaper to produce than current protein-based treatments, which are given daily, weekly, or monthly by injection.

“Importantly, it will also have a shorter duration in the body, allowing clinicians to stop the anti-inflammatory action of the drug if the patient ever needed to switch their immune response back to 100% in order to clear an infection,” he points out

So far, the results have shown great promise for blocking multiple sclerosis in a model of that disease, as well as in sepsis, where in response to bacteria, potentially fatal blood poisoning occurs. However, the target for MCC950 is strongly implicated in diseases such as Alzheimer's disease, atherosclerosis, gout, Parkinson's disease and rheumatoid arthritis, which means it has the potential to treat all of these conditions.

“We are really excited about MCC950,” says Dr. O’Neill. “We believe this has real potential to benefit patients suffering from several highly debilitating diseases, where there is currently a dire need for new medicines.”

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