Cytokine Signalling Forum

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A small-molecule inhibitor of the NLRP3 inflammasome for the treatment of inflammatory diseases

Coll RC, Robertson AA, Chae JJ, et al. - Nat Med. 2015 Feb 16. doi: 10.1038/nm.3806. [Epub ahead of print]

A team of scientists at Trinity College Dublin and the University of Queensland Australia, led by Professor Luke O'Neill, have identified a key molecule that may result in the development of new anti-inflammatory therapies for diseases such as: cryopyrin-associated periodic syndrome (CAPS), multiple sclerosis, type 2 diabetes, Alzheimer’s disease and atherosclerosis.

Professor O'Neill and his team have identified MCC950 as a potent, selective, small-molecule inhibitor of the NLRP3 inflammasome (a key component of the inflammatory process). MCC950 inhibition of NLRP3 in vivo has been shown to attenuate the severity of a model of multiple sclerosis. In addition, ex vivo activity has been seen in Muckle–Wells syndrome.

The findings suggest they have found a potentially transformative medicine, which targets what appears to be the common disease-causing process in a myriad of inflammatory diseases. This discovery potentially represents a major development in the search for new treatments for inflammatory diseases. Further research is ongoing to define the precise mechanism of action.