NOVEL MOLECULAR TARGETS IN NASH

 

Non-alcoholic steatohepatits (NASH) is inflammation and damage to the liver due to a build-up of fat.  It is part of a group of conditions referred to as non-alcoholic fatty liver diseases.  It is similar to the kind of liver disease caused by long term, heavy drinking but people with NASH drink little or not at all.

New treatments for inflammatory diseases like NASH could be on the way thanks to a significant discovery made by a group of scientists led by Professor Luke O’Neill.  About eight years ago, they came across the compound MCC950, and began to explore its potential uses. This molecule was previously developed, then abandoned, by Pfizer two decades ago as a possible treatment for arthritis.

These researchers have found that MCC950 directly inhibits NOD-like receptor protein 3 (NLRP3).  NLRP3 is a central mechanism of liver inflammation, injury and fibrosis in various liver diseases including NASH.  The study showed that MCC950 is effective at inhibiting, or reversing inflammation, injury and fibrosis in two different models of NASH. 

This suggests that MCC950 may be used to treat a myriad of inflammatory diseases.  Furthermore, the anti-inflammatory effects of MCC950 were not associated with inhibition of other components of the inflammasome complex that are important for control of infection.

Professor O’Neill and his team hope to form a company to further develop and test this compound.  It could be 10-15 years before it could be fully approved for use in more complex diseases like Multiple Sclerosis or Alzheimer’s.

 

http://onlinelibrary.wiley.com/doi/10.1002/hep.28175/full