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

Written by Louise Stanley on . Posted in Clinical trials, Experimental medicine, Inflammation and chronic disease.

The first participant has been treated in an early-phase clinical trial investigating whether a new medical device, Carbalive, can slow disease progression and improve outcomes for patients with a rare liver disease called primary sclerosing cholangitis (PSC). The trial, known as the CATCH trial, marks the first time this device will be studied in humans with PSC-IBD.

The new clinical trial involves several BHP partners – it’s being coordinated by researchers from the University of Birmingham and the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre, and clinicians at University Hospitals Birmingham (UHB) NHS Foundation Trust. 

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.

There is currently 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.

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.”

First participant in the trial

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.

Troels Jordansen, Yaqrit’s Chief Executive Officer said: “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.

“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.”

CATCH is the latest clinical trial delivered through the NIHR Birmingham Biomedical Research Centre, a partnership between the University of Birmingham and University Hospitals Birmingham NHS Trust, which focuses on inflammatory disease research. Collaborative research from the NIHR Birmingham BRC has already seen nearly 1,000 clinical trials initiated and has informed UK clinical guidelines.

Professor Andrew Filer appointed Director of NIHR Birmingham Biomedical Research Centre

Written by Louise Stanley on . Posted in Birmingham Health Partners, Inflammation and chronic disease.

The National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC) has announced the appointment of Professor Andrew Filer as its new Director. Professor Filer takes over from Professor Paul Moss, who has served as Interim Director since June 2024.

Professor Filer is already co-lead of the BRC’s Inflammatory Arthritis research theme and brings a wealth of clinical and translational research experience to the role. His leadership will further guide the BRC in delivering cutting-edge research that transforms patient care across the region and beyond.

Professor Filer said: “I’m honoured to take on the role of Director at such an exciting time for the Birmingham BRC. Our BRC is built on a foundation of collaboration, innovation and clinical excellence. I look forward to working with our partners to ensure our research continues to make a real difference to patients’ lives.”

The NIHR Birmingham BRC is a cross-BHP collaboration, hosted by University Hospitals Birmingham NHS Foundation Trust (UHB) and delivered in partnership with the University of Birmingham and six associate academic and NHS partners across the region. It brings together clinicians, scientists and healthcare professionals to drive translational research improving outcomes for people with inflammatory diseases.

Professor Kiran Patel, Chief Medical Officer at UHB, said: “Professor Filer is passionate about integrating patient-centred approaches and high-quality discovery science to drive innovation. I am delighted he has taken on this role, and I am sure he will provide effective leadership to our incredibly successful NIHR Birmingham BRC.”

Professor Neil Hanley, Pro-Vice-Chancellor and Head of the College of Medicine and Health at the University of Birmingham, added: “I’m really excited by Andrew’s appointment. It really does reflect the strength of clinical research leadership in Birmingham. His vision and energy will be instrumental for the next phase of the BRC’s mission to deliver inclusive impact, health improvement and economic growth.”

Dr Victoria Day, Head of Infrastructure at the Birmingham BRC, commented: “Andrew brings deep knowledge of the NIHR landscape and a long-standing commitment to patient-focused research. His leadership and collaborative approach will be key to driving the Birmingham BRC forward and delivering meaningful health impact.

“I would like to take this opportunity to sincerely thank Professor Paul Moss for his support during his time as Interim Director. His leadership and commitment have contributed to the many achievements we’ve made during this period.”

Professor Andrew Filer is a Professor of Translational Rheumatology in the Department of Inflammation and Ageing at the University of Birmingham. A Fellow of the Royal College of Physicians, he holds a PhD in immunology and is an Honorary Consultant Rheumatologist. He is internationally recognised for his work in early inflammatory arthritis, particularly in developing novel imaging and biopsy techniques to study disease mechanisms and improve diagnosis.

He co-leads the Birmingham Early Arthritis Clinic and has pioneered the use of musculoskeletal ultrasound and ultrasound-guided synovial biopsy in clinical research. His research focuses on Synovial cellular biology, disease stratification, and the development of targeted therapies for rheumatoid arthritis. Professor Filer is a passionate advocate for integrating patient cohorts with high-quality basic science to accelerate translational impact.

Birmingham researchers join Nobel Prize winner to win major funding on chronic inflammation

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

Researchers at the University of Birmingham will lead a major research programme to transform the treatment of autoimmune diseases by halting chronic inflammation at its source, working in partnership with 025 Nobel Prize winner Professor Shimon Sakaguchi, from Osaka University, and Professor Calliope Dendrou from the University of Oxford.

Backed by a £3.83 million Wellcome Discovery Award, the ambitious eight-year programme brings together world-leading scientists and clinician-scientists to focus on understanding and controlling the immune system’s regulatory mechanisms, with a particular focus on the liver.

Research will focus on the role of Foxp3+ Regulatory T-cells in maintaining immune tolerance and preventing tissue damage caused by autoimmune responses. Scientists and clinicians in the UK and Japan will explore how these Regulatory T-cells discovered by Professor Sakaguchi can be used to restore immune balance and treat autoimmune diseases.

Project leader Ye Htun Oo, Professor of Autoimmune Liver Diseases, from BHP founder-member the University of Birmingham, has been working in research partnership for more than 12 years with Professor Sakaguchi who was announced this week as one of three co-winners of the 2025 Nobel Prize in Physiology or Medicine – alongside US scientists Mary Brunkow and Fred Ramsdell – for their work understanding how Regulatory T-cells maintain a healthy balance within the immune system, creating openings for possible new autoimmune disease and cancer treatments.

Professor Oo commented: “Congratulations to Professor Sakaguchi on his Nobel Prize success – we are looking forward to continuing the University of Birmingham’s longstanding research partnership with him as we explore together how the Regulatory T cells that he discovered 30 years ago can help to treat autoimmune liver diseases.”

He also commented that Birmingham is one of the biggest liver transplant centres in Europe and this new approach of controlling autoimmune diseases with Regulatory T cells, will give hope to prevent liver transplantations for autoimmune liver diseases in future.

Autoimmune diseases occur when the body’s immune system mistakenly attacks its own tissues. In liver diseases such as autoimmune hepatitis, primary biliary cholangitis, and primary sclerosing cholangitis, there is currently no cure, leading to chronic inflammation, organ damage, and loss of function and eventually requiring liver transplantations.

Professor Sakaguchi commented: “This exciting collaborative award will allow us to work together to understand stable functional regulatory T cells biology and to progress towards future therapy with these cells for patients with autoimmune liver diseases and multi-organ autoimmunity.”

The programme unites the expertise of Professors Sakaguchi and Professor Oo with Professor Graham Anderson, from the University of Birmingham – a leading authority on thymic T-cell development and immune tolerance – and Professor Calliope Dendrou, from the University of Oxford, an expert in immune disease single-cell and spatial multiomics.

Professor Anderson commented: “By revealing how immune cells interact with tissue during chronic inflammation, our goal is to find a way of using these Regulatory T-cells as a naturally occurring way of treating autoimmune disease of the liver – nature’s medicine. We aim to use our work as proof of concept in the liver that is transferrable to other forms of the condition which can cause significant damage to the gut, skin, and other organs.”

Professor Sakaguchi was awarded an honorary Doctor of Science degree by the University of Birmingham in 2019 in recognition of his vision and research driving the development of regulatory T cells as therapeutic in autoimmunity and organ transplantations.

Professor Shimon Sakaguchi following the award of his honorary Doctor of Science degree in 2019 – pictured with the University’s then Pro-Chancellor Ed Smith CBE (left) and former Head of College of Medicine and Health Professor David Adams. (Photo: Paul Bonning-Tyers from Ede and Ravenscroft)

New funding set to improve discharge of pancreatitis patients

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

Birmingham researchers have won funding to develop recommendations to save acute pancreatitis patients returning to hospital and find ways to support patients following discharge, including long-term conditions such as mental health and diabetes, and reduce health service use.

Acute pancreatitis is a common condition leading to approximately 40,000 people being admitted to hospitals each year in the UK. This inflammation of the pancreas causes symptoms including pain, nausea and vomiting, and severe cases of the condition can be life-threatening. Currently, recommendations for post-discharge support are limited, and around 10% of patients will be readmitted within 30 days.

After being discharged from hospital, some patients continue to have health issues because their pancreas struggles to digest food or manage blood sugar. Half of those affected by acute pancreatitis may be at risk of developing mental health issues, such as anxiety or depression.

The PANORAMA project led by surgeon and researcher Matthew Lee, expands the University of Birmingham’s extensive National Institute of Health and Social Care Research (NIHR) funded portfolio to the value of almost £1million and will run for the next three years. He said: “Patients tell us that they feel left on their own after hospital discharge following pancreatitis. This work will help us figure out we can better manage follow up, and help people to look after themselves in the community.”

Lee and team will produce high quality evidence about the accessibility, acceptability, and costs of support, care and treatments in current post-hospital pathways for people with acute pancreatitis. In a series of surveys, interviews and workshops, bringing together patients and healthcare professionals, they will determine recommendations for changes to current pathways.

The need for research in this area was highlighted by the James Lind Alliance and the study has been designed by patients, nurses, primary care and hospital doctors, as well as experts in health service research.

It is hoped the recommendations will benefit acute pancreatitis patients, families and lead to savings for the health service.

Matthew is a Clinician Scientist with the University of Birmingham, and is also an Honorary Consultant Colorectal Surgeon at University Hospitals Birmingham – both founding members of BHP.

Possible new disease targets for children with arthritis revealed by first-of-its-kind study

Written by Louise Stanley on . Posted in Experimental medicine, Inflammation and chronic disease.

Groundbreaking research by a team from BHP’s Birmingham Children’s Hospital and the University of Birmingham – working with UCL and Great Ormond Street Hospital – has revealed important clues into what is driving disease in children with arthritis.

Cutting-edge techniques have allowed scientists to uncover the unique architecture of cells and signals inside the joint as inflammation takes hold, for the first time.

Published in Science Translational Medicine, the study investigated juvenile idiopathic arthritis in children – caused by the immune system mistakenly attacking joints – which affects more than 10,000 children in the UK. It causes swelling, stiffness and pain in the joints over years or decades, leading to damage of the joints and long-term disability. While there are pain management treatments available, which in some cases achieve remission, there is no cure – and it can take time to find which treatment works for each person. Treatments don’t work in the same way for every child, suggesting there are hidden differences between individuals that we are yet to fully understand.

Deepening the scientific and clinical community’s understanding of the condition is vital if more effective treatments are to be found, and undertaking biopsies in young children provides a new way forward. The study’s potential has been advocated for by families of children with arthritis, who agreed that the procedure would be acceptable to families, especially compared to living with a chronic inflammatory disease.

In a world first, tiny tissue samples were collected from the joint lining when children were having medicine injected into the joint, which were then analysed with advanced imaging and gene-profiling technologies. The fine resolution maps of the joints revealed differences between children of different ages and cell changes in those with more severe disease – creating unique cellular ‘fingerprints’ which may help researchers understand why some drugs work better for some children, and not others. The joints of children with arthritis also looked significantly different to those with adults, demonstrating the need to understand arthritis in children better.

Mapping out the networks of cells in the joint revealed a barrier layer (pink), with immune cells (navy) flooding in through blood vessels (light blue), which increase in number as the disease continues

Professor Adam Croft, Versus Arthritis Professor of Rheumatology at the University of Birmingham and chief investigator of the study, said: “We know how frustrating it can be for families and young people to find a drug that best works for their arthritis. Finding ways to better predict which medicines will be beneficial for a particular child would mean we were able to treat the disease more rapidly and effectively. To achieve this goal, we first needed to understand what cells make-up the lining of the joint where the inflammation occurs. Equipped with that knowledge, we can now start to tackle the next challenge, determining how these cellular fingerprints within the joint tissue can help us predict which drug will work best, ensuring we give the right drug, to the right child, at the right stage of their disease.”

Professor Lucy Wedderburn, University College London Great Ormond Street Institute of Child Health and Consultant of Paediatric Rheumatology at Great Ormond Street Hospital said: “This study represents a real step change in our work with children and young people who live with arthritis, and has been a huge team effort. Rather than having to rely on blood tests which often do not tell us accurately what is happening in the joint, we can now directly analyse the joint lining, across different types of childhood arthritis and different ages. Our findings show that younger children have different types of immune cells invading their joints compared to older children. Samples from children with arthritis looked different to adult samples, with a different make up of immune cells, blood vessels and distinct connective tissue cells. This suggests that treatments may need to vary depending on age and shows why we can’t just extend studies from adult studies to understand arthritis in children.”

The study was funded by the Medical Research Council, Versus Arthritis, National Institute of Health and Care Research, Great Ormond Street Hospital Charity, amongst others, and delivered through the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC).

Instrumental to driving this research forward was Dr Eslam Al-Abadi, a study investigator from the Birmingham Women’s and Children’s Hospital NHS Foundation Trust, who sadly passed away before publication. His incredible efforts in seeking to improve the care of children with this disease are gratefully acknowledged.

Promising new steroid drug could offer safer arthritis treatment

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

Early research has found that a new steroid drug, used for treating Duchenne Muscular dystrophy, has shown significant promise in treating inflammatory diseases such as rheumatoid arthritis.

Research published in Rheumatology revealed that Vamorolone, a glucocorticoid, showed to be just as effective as standard glucocorticoids to treat inflammation but with minimised negative side effects.

Glucocorticoids are some of the most widely used drugs to treat patients with a diverse range of inflammatory diseases. Unfortunately, whilst they effectively reduce inflammation and pain, they can also cause severe side effects. These include muscle and bone loss that can increase the risks of falls and fractures.

Vamorolone is a unique metabolism-resistant steroid approved by the FDA which appears to provide significant anti-inflammatory benefits while causing fewer harmful effects on muscle and bone.

The study, funded by the Foundation to Eradicate Duchenne and utilising mouse models with chronic rheumatoid arthritis, shows Vamorolone could be a promising alternative treatment for patients living with the disease.

Dr Rowan Hardy, Associate Professor in Steroid Metabolism and Signalling at BHP founder-member the University of Birmingham, said: “If Vamorolone is effective in patients with rheumatoid arthritis, it would allow us to better control disease activity, whilst preserving muscle and bone to reduce the risks of fractures and falls.”

The study team have now secured further funding with the Foundation to Eradicate Duchenne that will allow the research team to better understand the processes whereby Vamorolone is able to protect muscle and bone in patients with inflammatory disease. 

Through their involvement with the Birmingham Rheumatology group under Professor Adam Croft, and the NIHR Birmingham Biomedical Research Centre’s Inflammatory Arthritis Theme, the team will now work with clinicians to examine the possibility for new clinical trials to examine Vamorolone in rheumatoid arthritis patients. 

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