Research led by scientists at Trinity College Dublin has identified a protein called NLRP3 which plays a role in inflammatory diseases such as multiple sclerosis and cryopyrin-associated periodic syndrome (CAPS). The researchers have developed a compound that can block the action of NLRP3 and shown that it can reduce the severity of disease in a mouse showing features of human multiple sclerosis. The research team suggests that the compound could have benefits across a wide range of immune disorders, as well as against diseases like Alzheimer’s and Parkinson’s.
Inflammation is a biological process that kicks in to help the body fight infection and repair damage. Conditions such as multiple sclerosis and arthritis occur when immune cells become inappropriately activated, turning on the body’s own healthy cells. Inflammation has also been identified as an important early process involved in Alzheimer’s disease.
The research team identified a protein called NLRP3 which can trigger an inflammatory chain of events in muscular sclerosis and rarer inflammatory diseases such as cryopyrin-associated periodic syndrome (CAPS). The team searched for compounds that could block the action of NLRP3, finding one called MCC950 which could dampen the protein’s activity inside immune cells.
The team used the experimental compound MCC950 to treat mice showing features of multiple sclerosis, as well as those genetically predisposed to CAPS. Treatment with MCC950 could delay the onset and reduce the severity of features of multiple sclerosis in mice as well as expanding the life-span of those with CAPS. The researchers suggest that the compound could be developed into a potential new anti-inflammatory treatment as well as be explored for possible benefits against Alzheimer’s.
Dr Eric Karran, Director of Research at Alzheimer’s Research UK, said:
“A key step in developing any new drug is to show that you’re targeting a biological process that has an important role in driving a disease. Inflammation is a process at the heart of many serious conditions such as multiple sclerosis, as well as increasingly being implicated in diseases like Alzheimer’s. This research has identified a protein involved in inflammation and shown that blocking its action with an experimental compound could have benefits against multiple sclerosis in mice.
“This research represents an early stage in the drug discovery process, and this compound will need to be tested further to know whether it could be a safe and effective treatment for inflammatory diseases in people. Blocking inflammation is an approach being explored by researchers looking to develop new Alzheimer’s treatments but it’s unclear yet what, if any, benefits this compound may hold against the disease. We’d need to see this compound tested in animals bred to show features of Alzheimer’s to know whether the improvements seen in multiple sclerosis could also hold true for Alzheimer’s.
“Alzheimer’s Research UK is committed to driving forward early-stage drug discovery to boost the number of promising new compounds ready to be tested in people. Our Drug Discovery Alliance, launched this week, will dedicate £30m over five years to capitalise on promising new research findings and fast-track them through the drug discovery process towards the clinic.”