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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.

DaRe2THINK launches enhanced digital platform to transform clinical trials for GP practices

Written by Louise Stanley on . Posted in Clinical trials, Health inequalities.

A new digital platform has been launched to transform how NHS GP practices take part in clinical trials, making research quicker, easier and more inclusive for patients across the UK.

The DaRe2THINK clinical trial, led by BHP founder-member the University of Birmingham and the Clinical Practice Research Datalink (CPRD; part of the Medicines and Healthcare products Regulatory Agency), and run in partnership with NHS providers and the NIHR Research Delivery Network, has developed the platform to reduce paperwork for GP staff and reach communities often underrepresented in research.

The platform securely links to existing GP systems, automatically transferring relevant health data to participating NHS General Practices, significantly reducing administrative burden on clinical staff. The enhanced functionality enables practice staff to rapidly identify suitable patient cohorts for NHS clinical trials and engage participants through integrated mobile messaging systems.

DaRe2THINK already securely screens routine healthcare records from over 13 million NHS patients across more than 450 General Practices in England. This builds upon efforts to embed research into everyday NHS care.

Puja Myles, Director of CPRD at the MHRA, said: “This platform represents a paradigm shift in how we conduct primary care research. By leveraging routine health data and advanced digital tools, we’re removing traditional barriers that have prevented many communities from accessing the benefits of clinical trial participation.”

The current DaRe2THINK trial is investigating whether earlier intervention with NHS treatments can prevent blood clots and memory loss in patients with atrial fibrillation, a common heart rhythm condition associated with vascular dementia. Notably, 25% of participating practices are in England’s most health-deprived areas, addressing long-standing inequalities in clinical research participation.

Dipak Kotecha, Professor of Cardiology at the University of Birmingham, said: “DaRe2THINK is leading the way to empower patients and provide an opportunity to take part in NHS clinical trials.

“The enhanced digital platform has now been rolled out across England, making it quicker and simpler for NHS staff to reach out to their patients, and embedding prevention into routine care.”

Dr Alexandra Lee, a General Practitioner at University of Birmingham, said: “The digital trial platform used in DaRe2THINK has had a positive impact on the delivery of primary care research and has helped recruit a new cohort of general practices that haven’t previously taken part in clinical trials.”

Charles Michaleis, a DaRe2THINK Participant, said: “The DaRe2THINK study was very easy to sign up to and get involved in, with the hope that this will lead to improved treatments for NHS patients who have atrial fibrillation.”

The trial is funded by the National Institute for Health and Care Research (NIHR) Health Technology Assessment programme and supported by a dedicated public engagement team. The research was carried out at the NIHR Birmingham Biomedical Research Centre (BRC), a cross-BHP collaboration hosted by University Hospitals Birmingham.

DaRe2THINK aims to democratise access to clinical trials across all communities; the platform’s development aligns with the NHS People Plan to foster inclusive research culture and supports the broader goal of improving efficiency in community-based clinical trials.

Trial offers new hope to patients with rare skin cancer

Written by Louise Stanley on . Posted in Cancer outcomes, Clinical trials, Experimental medicine.

Researchers at BHP founder-members University Hospitals Birmingham have contributed to new findings showing that patients with Cutaneous (skin) T-cell Lymphoma (CTCL) experienced improved overall survival when treated with mogamulizumab, known by its brand name POTELIGEO.

CTCL is a rare type of cancer that begins in the white blood cells and affects the skin, causing rashes and slightly raised or scaly round patches. The most common types are mycosis fungoides and Sézary syndrome, with around 150 people diagnosed in the UK each year.

Individual’s experiences with CTCL can vary wildly – many people experience only a mild form that is not life-threatening and can be managed for years, while a smaller number develop a more serious form. Treatments either target the skin directly, using creams, light therapy, or radiotherapy, or are given as tablets or injections that circulate in the blood and work throughout the body.

POTELIGEO (mogamulizumab) is a prescription medicine given by injection into a vein and is used to treat mycosis fungoides or Sézary syndrome in adults whose disease has returned or who have not responded to at least one other treatment (oral or injectable).

Now in its tenth year, the PROCLIPI Study is the largest international study of its kind, involving 2,547 patients across 19 countries. It collects information on tests, scans, treatments, quality of life, and survival from CTCL patients, with the aim of developing a tool (known as a prognostic index) to predict outcomes for people with the disease.

The latest research confirmed that patients with advanced-stage disease treated with POTELIGEO experienced improved overall survival, with a median overall survival of 64 months compared to 54 months for patients who did not receive the treatment.

Professor Julia Scarisbrick, Consultant Dermatologist at UHB and Chief Investigator, said: “The PROCLIPI Study demonstrates the power of global collaboration in rare diseases. By bringing together data from across the world, we can generate insights that simply wouldn’t be possible in isolation.

“We are proud to coordinate this initiative here in Birmingham in partnership with Prof Kim at Stanford University, California, as we’re working to build rigorous scientific evidence while giving patients and their families a better understanding of what long-term survival looks like.”

Alexandra Mars, a 51-year-old patient at UHB’s Queen Elizabeth Hospital took part in the study, and said: “I have had tumour mycosis fungoides for over 10 years. I was most recently treated with mogamulizumab for two years and had no significant side effects. It appears to have put my disease to sleep, and I have not been on any medication since February. It is amazing to finally be able to have a break from treatment, which is so tiring, and not have to think about this disease.”

Due to the rarity of CTCL and its wide variation in presentation and progression, enrolling patients in clinical trials can be challenging. Despite these difficulties, growing evidence is helping doctors understand which treatments may be most beneficial.

NIHR Clinical Research Facility celebrates year of growth and success

Written by Louise Stanley on . Posted in Birmingham Health Partners, Clinical trials, Experimental medicine.

Birmingham’s NIHR/Wellcome Trust Clinical Research Facility (CRF) has celebrated yet another successful year, with thousands of patients and volunteers taking part in pioneering studies that are shaping the future of medicine.

Established in 2001, the CRF provides a high-quality environment for experimental and early-phase research across all age groups.

Spanning two of Birmingham Health Partners’ NHS member Trusts, the CRF runs its adult unit from Queen Elizabeth Hospital Birmingham (QEHB), part of University Hospitals Birmingham NHS Foundation Trust, and its paediatric unit from Birmingham Children’s Hospital, part of Birmingham Women’s and Children’s NHS Foundation Trust (BWC).

Between 31 March 2024 and 1 April 2025, the CRF supported 409 active studies – 334 at UHB and 75 at BWC – involving 13,503 patient visits and the recruitment of 2,514 participants.

The facility has seen a particular growth in Advanced Therapy Investigational Medicinal Product (ATIMP) studies, which explore innovative treatments based on genes, cells, or tissues. It has also expanded work in maternal health, mental health and neonatal research, as well as opening studies in new disease areas.

Collaboration is central to the CRF’s approach, with almost half of its studies (48%) co-delivered in partnership with other NIHR infrastructure, including the Birmingham Biomedical Research Centre. This way of working ensures expertise and resources are shared to maximise patient benefit and research impact.

The year also saw significant investment, with over £4 million secured by the facility to open a new clinical research unit in the MIDRU building at Heartlands Hospital, as well as new laboratory and endoscopy equipment purchased for both QEHB and Good Hope Hospital.

Patients themselves have spoken warmly of their experience at the CRF, with recent feedback including: “Every week has been a pleasure visiting the Wellcome Research Centre”, “All staff were extremely friendly, supportive and knowledgeable” and “They are kind and thoughtful, giving you all the information you need to feel comfortable”.

Jo Gray, Head of Research and Development Operations, said: “I’m incredibly proud of all our colleagues in the Clinical Research Facility for their dedication and hard work in delivering patient-centred research across a wide range of studies and disease areas. Their impact on patients is evident in the feedback we receive, and we look forward to building on last year’s successes through continued collaboration with our industry partners.”

Members of the CRF team gathered to celebrate another successful year

New vaccine trial for head and neck cancer patients

Written by Louise Stanley on . Posted in Cancer outcomes, Clinical trials, Experimental medicine.

Patients in Birmingham who have advanced head and neck cancers may be eligible to take part in a new clinical trial of a potential cancer vaccine, supported by the NHS Cancer Vaccine Launch Pad (CVLP).

Queen Elizabeth Hospital Birmingham, part of BHP founder-members University Hospitals Birmingham NHS Foundation Trust (UHB), is one of 15 sites across the country aiming to recruit more than 100 patients over the next year.

The investigational cancer vaccine in this latest trial on the platform uses mRNA technology to help the immune system recognise and kill cancer cells which express human papillomavirus (HPV) proteins.

The first head and neck cancer patients in England have received the investigational mRNA cancer vaccine in the clinical trial, known as AHEAD-MERIT (BNT113-01), with more patients to soon be enrolled at their nearest NHS hospital. 

More than 11,000 new head and neck cancer cases are diagnosed in England every year, with cancers typically developing in the mouth, throat or voice box.  

Despite advances in care for patients with head and neck cancer, the advanced form of the disease is difficult to treat and has high rates of recurrence, with two-year survival rates at under 50%.

The investigational cancer vaccine is designed to encode two proteins that are frequently found in head and neck squamous cell cancers associated with human papillomavirus (HPV-16). This is the most common type of head and neck cancer, accounting for 95% of these types of cancers, and the vaccine aims to train the immune system to fight the cancer.

NHS England is partnering with life sciences company BioNTech to help identify potentially eligible patients to refer to NHS hospitals running the clinical trial.

Dr Paul Sanghera, Consultant Oncologist and Principal Investigator of the trial at Queen Elizabeth Hospital Birmingham, said: “This clinical trial marks an important step forward in the search for better treatments for advanced head and neck cancers, which remain a significant challenge in oncology.

“These cancers are notoriously difficult to treat, and access to this investigational vaccine could offer patients a potential new option in their treatment journey. While we are still in the early stages, the hope is that this trial will pave the way for improved outcomes for those living with these challenging conditions.”

Matthew Metcalfe, Hospital Executive Director at Queen Elizabeth Hospital Birmingham, said: “We are incredibly proud to be one of the 15 sites across the country taking part in this important clinical trial. It reflects our ongoing commitment to driving forward research aimed at improving outcomes for patients in Birmingham and beyond, offering new hope to those facing these challenging diagnoses.”

Dr Iain Foulkes, Executive Director of Research and Innovation at Cancer Research UK, said: “It’s great to see more clinical trials of vaccines for head and neck cancer supported by the Cancer Research UK-funded Southampton Clinical Trials Unit.

“Research into personalised cancer treatments is vital. There are over 200 different types of cancer and it’s unlikely there will ever be a single cure that works for everyone. That’s why it’s vital that we support a wide range of research, so that more people can live longer, better lives, free from the fear of cancer.”

More cancer vaccines news from across BHP

Birmingham academic appointed Clinical Lead of new NIHR network

Written by Louise Stanley on . Posted in Birmingham Health Partners, Clinical trials.

The National Institute for Health and Care Research (NIHR) has awarded £6.5 million, funded jointly through a public-private partnership with the pharmaceutical industry, to establish a UK-wide Commercial Research Delivery Centre (CRDC) Network. The Network is hosted by University Hospitals of Leicester NHS Trust (UHL), with Birmingham Health Partners’ Managing Director Professor Lorraine Harper as its Clinical Lead, and will commence formally on 1 September 2025.

The new Network will provide strategic coordination of all 21 CRDCs across the the UK. It will play a key role in building research capacity, streamlining the interface between industry and the UK clinical trials delivery infrastructure, and enhancing efficiency to deliver commercial clinical research through harmonised processes, in line with the Government’s call to turbocharge medical research earlier this year.

The Network will:

  • Provide strategic leadership and national coordination across the 21 CRDCs across the UK
  • Offer a central point of contact for industry sponsors
  • Facilitate study feasibility, placement and setup across the UK
  • Foster collaboration with regulators, wider NIHR and UK-wide delivery infrastructure, and other key stakeholders to advance UK Clinical Research Delivery
  • Support workforce development, inclusion, and public involvement
  • Facilitate the integration of the Primary Care CRDCs (PC-CRDCs) in England in autumn 2025, adopting them into the Network

By aligning CRDC efforts and offering a cohesive offer to industry, the Network will ensure that the CRDCs deliver against industry expectations for faster, more efficient set up and delivery of commercial research in the UK.

Lorraine Harper, Professor of Nephrology at the University of Birmingham and Managing Director of Birmingham Health Partners, is the Director of the Central and North West Midlands CRDC and has been appointed Clinical Lead of the new Network. She said: “The vision of the Network is to ensure equitable access, diverse recruitment and a much more efficient model of trial delivery, improving NHS and patient access to trials. With Birmingham Health Partners already leading a regional programme to reduce bureaucracy in clinical trials, and BHP member Birmingham Women’s and Children’s Hospitals hosting the Central and North West Midlands CRDC, this is an exciting opportunity to align all our work and deliver a ‘gold standard’ for commercial clinical trials.”

BHP founding member Birmingham Women’s and Children’s Hospitals NHS Foundation Trust hosts the £7m Central and West North Midlands (C&NWM) CRDC, working closely with regional partners Midlands Partnership University NHS Foundation Trust – host of Staffordshire and Shropshire, Telford and Wrekin Health Research Partnership (SSHERPA) – and the Black Country Provider Collaborative.

The C&NWM region, home to 4.2 million people, includes many of the UK’s most economically deprived communities who face significant health inequalities and higher rates of serious illness. The CRDC will focus on addressing these inequities by increasing access to clinical trials for patients who have the greatest need – dovetailing with BHP’s strategic focus on addressing health inequalities and giving greater opportunities for residents to participate in research.

Professor Melanie Davies, Professor of Diabetes Medicine at the University of Leicester and Honorary Consultant Diabetologist for University Hospitals of Leicester NHS Trust, and Director of the CRDC Network, said: “We are really proud that University Hospitals of Leicester NHS Trust has been awarded this funding to host this federated CRDC Network across the four nations of the UK. We are fully committed to working with our partners across the UK to deliver faster, more efficient set up and delivery of commercial research. We want to drive increased investment from industry and enable even more people to take part in studies that can lead to the future approval of new medicines and devices for the benefit of patients in the UK.”

Dr Maria Koufali, NIHR Life Sciences Industry Director, said: “The UK CRDC Network is a critical part of our national effort to transform UK clinical research delivery. By streamlining trial set-up and expanding access into community and underserved settings, it will help make the UK one of the most attractive destinations globally for commercial research. This means faster access to innovative treatments for patients, greater investment in the NHS and a stronger life sciences sector that boosts the health and wealth of the nation.”