A new type of cell therapy which could offer “real hope” to patients with advanced liver disease has been welcomed by a liver charity.
Researchers said results from a clinical trial suggest that the new type of therapy shows promise as the first treatment for advanced liver disease.
They found that patients with the condition who were treated with the cell therapy had a significantly lower risk of death or need for a liver transplant after four years compared with those who received standard medical care.
While the liver has the unique ability to regenerate itself after damage in patients with advanced liver disease, severe scarring – known as cirrhosis – can leave it damaged beyond repair, leading to liver failure.
Experts said the new treatment could offer a potential alternative to liver transplants for those in end-stage liver disease, which currently is the only curative treatment option.
Professor Stuart Forbes, from the University of Edinburgh’s Institute for Regeneration and Repair, said: “Liver disease is a major cause of death of people of working age.
“Although we can use liver transplantation as a rescue treatment for a proportion of people who have advanced liver disease, this is restricted by a lack of suitable donor organs.
“Unfortunately, many patients may die whilst on the liver transplant waiting list.
“There is therefore a desperate need for alternative treatments for patients with advanced liver disease.
“We hope this type of approach could one day add to our treatment choices for patients with advanced liver disease, reducing the need for liver transplants.”
Experts said more than three-quarters of people are diagnosed with cirrhosis when it is too late for effective treatment, contributing to more than 11,000 deaths per year in the UK.
Scientists at the University of Edinburgh have developed a “pioneering” cell therapy designed to tackle the scarred tissue and restore liver function.
The treatment involves taking immune cells from the patients’ blood and turning them into mature macrophages – a type of white blood cell that “eats” damaged or infected cells – which are then re-injected back into the patient.
The macrophages then travel to the liver, where they break down scar tissue, reduce harmful inflammation, and encourage the growth of healthy liver cells.
The treatment was tested in the Match clinical trial, which saw 26 patients receive the macrophage therapy, while 24 received standard care.
Researchers said that after four years, 70% of patients who received the macrophage therapy were living without the need for a liver transplant, compared with 40% of patients who did not receive the treatment.
There were eight deaths and no liver transplants among the patients treated with macrophages, compared with nine deaths and five liver transplants among those who received standard care.
Scientists said no serious side-effects were reported in patients treated with the cell therapy.
The research team said the four-year follow-up period provides important insights into the safety of the treatment and the long-term benefits.
Pamela Healy, chief executive of the British Liver Trust, welcomed the findings and said: “For people living with cirrhosis, these results offer something that has been in desperately short supply for far too long: real hope.
“Being told that a liver transplant may be your only option – and that one may never come – is devastating for patients and families.
“A treatment that could slow or reverse liver failure and reduce the need for transplantation would be truly life changing.”
The macrophage therapy was developed by Professor Forbes, alongside the Scottish National Blood Transfusion Service (SNBTS).
Professor Forbes co-founded spinout company Resolution Therapeutics in 2020, with the support of Edinburgh Innovations, the University’s commercialisation service.
The company is currently testing a new version of the macrophage therapy, known as RTX001, in patients through the Emerald clinical trial.
The study, funded by the Medical Research Council and Chief Scientist Office, is published in the journal Cell Stem Cell.
The research team also included scientists from the University of Dundee, SNBTS, Resolution Therapeutics, Tayside Clinical Research Centre and Glasgow Royal Infirmary.
