Birmingham researchers lead world-first trial for rare liver disease treatment

The first patient has been treated in an early-phase clinical trial investigating whether a new medical device, Carbalive, can slow disease progression of a rare liver disease called primary sclerosing cholangitis (PSC). 

Coordinated by clinicians at University Hospitals Birmingham (UHB) NHS Foundation Trust in collaboration with researchers from the University of Birmingham and the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, this trial, known as the CATCH trial, marks the first time will be studied in humans with PSC-IBD.

PSC affects around 3500-4000 people in the UK, including both adults and children. In PSC, the body’s immune system attacks itself, causing inflammation and scarring of the bile ducts – the small tubes that carry bile (a digestive fluid produced by the liver) to the gallbladder and small intestine. This leads to reduced or blocked bile flow, and over time, liver damage.

In around 80% of people with PSC, the body also attacks the bowel, which can lead to inflammatory bowel disease (IBD), a devastating disease that affects the lining of the gut, leading to severe gut inflammation, bleeding and diarrhoea.

Currently, there is no cure or medication to improve survival, and liver transplantation remains the only life-saving option for PSC.

The exact cause of PSC is not known, but research has shown that gut inflammation, that is also a key component of IBD, is a key trigger for the disease. Removing harmful substances and restoring the disturbed gut microbiome reduces gut inflammation, which may help slow its progression.

“PSC-IBD is a progressive condition, and with no approved treatments, it can ultimately lead to liver failure. This trial is an important step in exploring the potential of Carbalive to slow the progression of this disease and improve patient outcomes.”

Professor Palak Trivedi, University of Birmingham

Carbalive (also called YAQ001) is a medical device created by Yaqrit – a company that develops life-saving treatments for patients with advanced liver disease. This device is made up of tiny carbon beads with lots of different sized pores to absorb toxins that would otherwise return to the liver.

In pre-clinical studies, animals with cholestatic disease (a condition where bile flow from the liver is impaired) showed improvements in liver function and signs of disease activity after treatment with Carbalive. Early studies in people with cirrhosis have also shown the device to be safe and effective in restoring the gut microbiome and reducing inflammation.

Palak Trivedi, Principal Investigator of the study, Consultant Hepatologist and Clinician Scientist at UHB, and Professor of Cholestatic and Immune-mediated Liver Disease at the University of Birmingham, said: “PSC-IBD is a progressive condition, and with no approved treatments, it can ultimately lead to liver failure.

“This trial is an important step in exploring the potential of Carbalive to slow the progression of this disease and improve patient outcomes. By funding this trial, and this innovation, LifeArc offers hope to patients and their families through a promising new approach.”

Nick Haynes, from Nottingham, is the first participant to take part in the trial and began his treatment this week. Nick said: “I was diagnosed with PSC a couple of years ago, and as there aren’t many treatment options available currently, that really attracted me to taking part in this trial. I think it comes with many benefits – you hope the medicine will lead to some kind of improvement, but it’s also very reassuring to be part of enhanced screening. It’s about looking to the future too, and being part of something that could advance medicine and treatment options for people with PSC feels really positive. I really hope it becomes a viable treatment for patients in the future.”

Twelve patients with PSC-IBD will receive Carbalive orally for twelve weeks, and the data collected will assess whether the treatment improves bile flow and reduces inflammation.

The trial has been made possible through funding from LifeArc, a not-for-profit organisation supporting research into rare diseases.

“This is a great opportunity for Yaqrit to help patients with PSC-IBD while remaining highly focused on the treatment of late-stage liver disease,” said Troels Jordansen, Yaqrit’s Chief Executive Officer. “In YAQ001, the company has developed a treatment that addresses toxicities and inflammation that are common in advanced liver disease but also apparent in a broader spectrum of conditions.”

Sam Barrell, CEO of LifeArc, said: “For the 3.5 million people in the UK living with rare diseases, access to treatment is a huge challenge – 95% of conditions have none. That must change. World-first trials, like the CATCH Trial, are part of the solution, which is why LifeArc funded it through our Pathfinder Programme. We have an opportunity to make the UK the go-to place for innovative trials like this, but to realise this ambition we need to transform how we support rare disease research, making a difference for rare disease patients much faster.”