Irish scientists identify key molecule in fight against Alzheimers and MS

Irish scientists have identified a potent molecule that may be able to halt the progress of deadly diseases such as Alzheimers and multiple sclerosis.

Prof Luke O'Neill: Molecule can stop disease progress.

Research scientists at Trinity College in Dublin have also found that the molecule’s anti-inflammatory properties can stop a variety of other serious conditions in their tracks, among them diabetes and gout.

In the study to be published this week in the medical journal Nature Medicine, the international research, team led by Trinity and the University of Queensland in Australia, showed how the molecule MCC950 can suppress the ‘NLRP3 inflammasome’, which is an activator of the key process in inflammatory diseases.

“It is a bit like a cog in a machine that drives these diseases,” said Luke O’Neill, professor of biochemistry and director of Trinity Biomedical Sciences Institute. “I would not go as far as to say that what we have is a cure for all of them but, because they share an inflammatory component, this molecule can stop their progression.”

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

“In Alzheimers it’s the brain; in gout, it’s the big toe; in arthritis it’s in the joints,” said Prof O’Neill. “It may seem strange but the underlying process is shared in all of these conditions.”

Prof O’Neill described the discovery as one of the most exciting of his career. “I have been in the research business for 30 years and you usually end in failure, but this time we seem to have hit upon something truly transformative,” he said.

“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. Drugs like aspirin or steroids can work in several diseases, but can have side effects or be ineffective.”

Rebecca Coll, lead author on the paper, who conducted some of the work at Trinity, said: “MCC950 is blocking what was suspected to be a key process in inflammation. 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.”

Matt Cooper, chemist and co-senior author from the University of Queensland’s Institute for Molecular Bioscience (IMB), added: “MCC950 is able to be given orally and will be cheaper to produce than protein-based treatments.

“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.”

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, blood poisoning occurs.

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