A macrophage cell therapy that is being developed by spinout Resolution Therapeutics shows promise as the first treatment for advanced liver disease, following results from a clinical trial.
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.
The findings suggest the use of macrophage therapy is a promising therapeutic option for end-stage liver disease and represents a significant step forward in regenerative medicine, experts say.
The macrophage therapy was developed following more than a decade of research by the University of Edinburgh’s Professor Stuart Forbes, of the Institute of Regeneration and Repair, alongside the Scottish National Blood Transfusion Service (SNBTS).
In 2020, Professor Forbes co-founded spinout Resolution Therapeutics to bring the team’s pioneering science to patients. The company is currently testing a new version of the macrophage therapy, known as RTX001, in patients through the EMERALD clinical trial.
The treatment involves taking immune cells from the patients’ blood and turning them into mature macrophages – a type of white blood cell that ‘eats’ infected or damaged cells – which are then re-injected back into the patient.
The macrophages 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, with 26 patients receiving the macrophage therapy, while 24 received standard care.
After four years, 70 per cent of patients who received the macrophage therapy were living without the need for a liver transplant, compared with just 40 per cent 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. No serious side-effects were reported in patients treated with the cell therapy.
The four-year follow-up period provides important insights into the long-term benefits and safety of the treatment, according to the research team.
Dr Susan Bodie, Head of Innovation Development and Licensing at Edinburgh Innovations, which supported the translation of Professor Forbes’ research in the early stages as well as company formation, said:
It is hugely rewarding to see a University of Edinburgh spinout like Resolution Therapeutics advancing a novel cell therapy, based on pioneering research, that now shows real potential to transform care for people living with advanced liver disease. ”
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 in their 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. ”
Dr Lara Campana, Scientific Co-Founder and Senior Vice President, Research & Translational Science at Resolution Therapeutics, said:
This study not only highlights the significant potential of regenerative macrophage therapy in patients with advanced cirrhosis, but it also offers us a glimpse into the mechanism whereby these cells achieve their therapeutic effect: analysis of patients’ blood revealed a strong anti-inflammatory effect of this therapy, which correlates with transplant-free survival. "
The study, funded by the Medical Research Council and Chief Scientist Office, is published in the journal Cell Stem Cell: https://www.cell.com/cell-stem-cell/fulltext/S1934-5909(26)00156-6.
The research team included scientists from the University of Dundee, the Scottish National Blood Transfusion Service, Resolution Therapeutics, Tayside Clinical Research Centre and Glasgow Royal Infirmary.
