The FARGO Trial for PSC
Led by Dr Palak Trivedi
(NIHR Birmingham BRC and the University of Birmingham)
Funding research like this is only possible thanks to your generous donations and fundraising.
The FARGO Trial
The trial will explore the potential of a new treatment to slow the progression of PSC and improve quality of life for people with PSC. It is called the FARGO Trial (FAecal microbiota transplantation in primaRy sclerosinG chOlangitis) and will be led by Dr Palak Trivedi (Associate Professor and Consultant Hepatologist at the NIHR Birmingham BRC and the University of Birmingham). Dr Trivedi leads several national PSC research programmes and is experienced at running clinical trials.
Despite impressive research in the last decade, there is still no cure for PSC, nor any medication that has been shown to improve survival. Liver transplantation is the only lifesaving treatment for some PSC patients. Although a very rare disease, PSC accounts for 10-15% of all liver transplants performed in the UK and is now the leading reason for transplantation in several European countries. Moreover, PSC returns in around a third of people who have had a liver transplant.
Martine Walmsley, PSC Support Chair of Trustees, said, “Although we welcome clinical trials that test brand new drugs for PSC, progress is painfully slow. People with PSC don’t have the luxury of time and we must look at quicker medicine development routes, like repurposing therapies that are already used for other diseases.”
What is FMT?
The treatment we are investigating is called faecal microbiota transplantation (FMT). Stool will be collected from healthy donors and refined in a laboratory so that it can be given to people with PSC during their annual colonoscopy. FMT is already used to treat Clostridium difficile infection. Early research has shown that FMT is safe, is effective in treating inflammatory bowel disease, and improves liver blood test results in some individuals.
It is thought that the gut microbiome is involved in the way PSC develops and progresses. It has been shown that the makeup of gut microbiome in people with PSC is different to that found in people without liver and bowel inflammation. Our study involves taking gut microbes from healthy donors and transferring them to people with PSC using FMT. Changing the balance of the gut microbiome in people with PSC could slow or even reverse progression of the disease.
Research that matters
This clinical trial is very much patient-centred and driven. People living with PSC are particularly interested in the gut microbiome and how it affects the disease. Dr Trivedi worked regularly with a group of PSC patients to develop the research idea and to design this trial (PSC Patient Panel).
Dr Trivedi said, "I am delighted that LifeArc and PSC Support have elected to fund this study! The study design has been heavily influenced by our patients, right from choosing the mode of treatment, the duration of the study, and the quality of life elements we seek to measure. Without active patient and public involvement, this study would not have been possible, and the ongoing engagement of the groups will prove critical to the success of this trial."
LifeArc is a self-financing charity that advances promising life-science ideas into life-changing solutions for patients, particularly those living with a rare disease. Translating science is a long, expensive and unpredictable process. This means many exceptional ideas don’t make it to the patient – they don’t even make it out of the lab.
LifeArc has a wealth of experience helping patients get the best treatments that science can offer through the development of medicines, diagnostics and treatments. By working with LifeArc, we have been able to develop and co-fund an important clinical trial for PSC, which may otherwise have been out of reach.
Dr Catriona Crombie, Head of LifeArc’s philanthropic fund, said: “Our approach to funding is to work with others, to connect and uncover the potential of promising research that could potentially solve complex healthcare problems for patients. This grant, which we have jointly awarded with PSC Support, will allow Dr Trivedi’s team, to answer key questions and support the translation of this experimental treatment, from idea to direct patient benefit across the UK PSC population."
What impact will this study have?
If we get a positive result, we hope that future work on a larger scale will follow, with a view to making FMT available globally. In parallel, we will work with medicine regulators to help ensure that FMT is available as a treatment for people with PSC as soon as possible.
Where will the study be carried out?
The Birmingham team, together with teams at the Royal Free London, St Mark’s Hospital, Imperial College London, and Norfolk and Norwich University Hospitals NHS Foundation Trust, will carry out the clinical trial to test this new treatment approach.
When will the FARGO Trial begin?
Although the study has now started, the research team is currently setting everything up and FARGO is expected to begin to invite volunteers to take part in early 2023.