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Global study to spur new liver cirrhosis medicines
A new £30 million study is aiming to speed up the development of new treatments for advanced liver cirrhosis.
The ADVANCE (Accelerating Discovery: Actionable NASH Cirrhosis Endpoints) study will follow 200 patients with cirrhosis – scarring caused by long-term damage of the liver – to uncover why some patients experience liver failure or develop liver cancer.
Researchers from the Universities of Edinburgh and Newcastle hope their findings will lead to earlier diagnosis and the development of a first-ever approved medicine for cirrhosis.
Urgent need
It is estimated that more than 440 million people worldwide live with a condition referred to as non-alcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH) – an inflammatory liver disease that is caused by accumulation of fat in the liver.
Over time, this causes scar tissue to form, which leads in many cases to liver cirrhosis. This can result in serious complications, including liver failure or liver cancer, and may result in the patient needing a liver transplant.
Ongoing assessment
The global study will enroll patients who have been diagnosed with or are thought to be at risk of advanced cirrhosis due to fatty liver disease.
Participants will initially undergo a biopsy to collect a small sample of liver tissue so that detailed changes in how genes are activated in the liver can be assessed.
Blood tests and MRI scans will be performed at regular intervals over the next two years. The data generated will allow researchers to see how disease-related changes evolve in the body as cirrhosis progresses.
The study is funded by pharmaceutical company Boehringer Ingelheim, and researchers will be supported by the University of Edinburgh’s commercialisation service, Edinburgh Innovations.
Liver disease has reached epidemic proportions worldwide. Therefore, there is a huge need to develop potent, new treatments for liver scarring. To help address this, over the last several years we have harnessed a new technology in Edinburgh called single cell RNA sequencing. Using this new technology has allowed us to study human liver scarring in high definition for the first time, and we hope that this cutting-edge approach will allow us to accelerate the discovery of much-needed new treatments for patients with liver disease. Professor Neil Henderson Co-lead of the study and Professor of Tissue Repair and Regeneration at the University of Edinburgh
We aim to work out why, even at the most advanced stages of liver disease, there is substantial variation in how the disease progresses with some people remaining well for many years whilst others rapidly experience liver failure or develop liver cancer. Working internationally with our collaborators, we will then use this knowledge to improve how patients are diagnosed, and to help develop new medicines. Professor Quentin Anstee Coordinator of the study and Professor of Experimental Hepatology at Newcastle University and Consultant Hepatologist at Newcastle Hospitals NHS Foundation Trust
Related links
The University of Edinburgh's Institute for Regeneration and Repair
Edinburgh Medical School
Edinburgh Innovations
[Image credit: Mohammed Haneefa Nizamudeen via Getty Images]
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