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Global study to spur new liver cirrhosis treatments

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 be the most detailed observational study of its kind, enrolling the largest number of patients and providing a detailed analysis of liver health.

This will not only enhance the understanding of NASH cirrhosis, but also help to identify translational biomarkers that will accelerate the development of future therapies.

The study will be led by researchers at Newcastle University and the University of Edinburgh, along with collaborators across Europe and it is hoped their findings will lead to earlier diagnosis and the development of a first-ever approved medicine for cirrhosis.

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, NASH causes the formation of scar tissue leading 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.

Currently there are no approved medicines for cirrhosis and so there is an urgent need for earlier diagnosis and new medicines to prevent MASH cirrhosis progression to liver failure, or to reverse the scarring of the liver once cirrhosis is established.

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 reflects the company’s commitment to improve the lives of people living with cardiovascular, renal and metabolic diseases (CRM).

Professor Neil Henderson, Professor of Tissue Repair and Regeneration at the University of Edinburgh and co-lead on the study, said: ” 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 state-of-the-art approach will allow us to accelerate the discovery of much-needed new treatments for patients with liver disease.”

The research team at the University of Edinburgh’s Institute for Regeneration and Repair will be supported by Edinburgh Innovations, the University’s commercialisation service.

Professor Quentin Anstee, Professor of Experimental Hepatology at Newcastle University and Consultant Hepatologist at Newcastle Hospital’s NHS Foundation Trust, who is co-ordinating the global study said: ” 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. ”

Lykke Hinsch Gylvin, M.D., Chief Medical Officer at Boehringer Ingelheim, added: ” Cardiovascular, renal, and metabolic diseases are the leading cause of death worldwide, accounting for up to 20 million deaths annually.

” At Boehringer Ingelheim we are focusing on understanding the whole patient and how to target specific disease mechanisms to address interconnected CRM diseases. We are very excited to work with our partners in the ADVANCE study to better understand the underlying disease processes and to bring much needed new treatments to patients with liver cirrhosis.”

The study will include 200 patients with cirrhosis. Participating patients will be recruited at specialist liver clinics at hospitals across the UK and Europe or through referral by their treating physician.

Anyone interested in taking part should discuss it with their treating physician.