Experimental medicine – Birmingham Health Partners

Trial offers new hope to patients with rare skin cancer

Written by Louise Stanley on 29/10/2025. 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 24/09/2025. 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 02/09/2025. 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

Adding dendritic cell vaccine to liver cancer therapy slows disease progression

Written by Louise Stanley on 29/08/2025. Posted in Clinical trials, Experimental medicine.

Patients with intermediate-stage primary liver cancer who received a vaccine of dendritic cells (DC) alongside their cancer therapy saw a longer time without tumour progression in response to standard treatment – found a study by BHP founding-member the University of Birmingham, funded by the National Institute for Health and Care Research (NIHR).

The results of the ImmunoTACE trial, the first clinical trial of its kind, published in Clinical Cancer Research, found improved progression-free survival (PFS) for patients with hepatocellular carcinoma (HCC) who received the cell-based vaccine expanded from their own white blood cells. The vaccine was administered alongside usual treatment with tumour chemoembolisation, a treatment for blood vessels that feed the tumour, plus chemotherapy.

The collaborative trial between the University of Birmingham, fellow BHP members University Hospitals Birmingham, Nottingham University Hospitals NHS Trust and Aintree University Hospital and Clatterbridge saw 48 patients recruited to receive either standard treatment alone or standard treatment plus a cellular vaccine using dendritic cells loaded with cancer antigens to stimulate immune responses against the cancer.

In the experimental arm of the trial the average time to progression of the tumour was 18 months compared with only 10 months in the group who only received standard treatment.

Professor David Adams, Chief Investigator of the study, Emeritus Professor of Hepatology at the University of Birmingham and past Director of BHP, said: “The results from this phase 2 trial are very promising and offer a potential new treatment option for patients with primary liver cancer, one of the highest causes of cancer-related death worldwide.

“As far as we know, ImmunoTACE is the first controlled clinical trial to show that a cell-based vaccine using lab-grown dendritic cells can improve patient outcomes with liver cancer. The results warrant further investigation and could in future offer much needed hope and a better treatment option for patients.”

The vaccine is made with dendritic cells (DC) which help orchestrate the immune system’s response to diseases including cancer by activating immune killer cells to recognise and destroy cancer cells.

The dendritic cells used in the study were expanded from the patients’ own white blood cells by growing them in a purpose-built laboratory for eight days with proteins taken from cancer cells. The cells allow the immune system to see these proteins and then to mount an immune attack on the cancer cells that bear them.

Patients received the DC vaccine at the same time as standard treatment with chemo-embolisation and then monthly for a further three months.

While dendritic cells are produced naturally in the body, studies have shown that in patients with cancer they can become “exhausted” and get stuck within the tumour rather than carrying cellular information back to the lymph nodes where they can activate immune killer cells. The idea of DC vaccine is to restore and uncover immune responses to the cancer. The current trial design reports that this therapy can be both affordable and effective.

Dr Yuk Ting Ma, lead author of the study and Associate Clinical Professor at the University of Birmingham, and an Honorary Consultant in Hepatobiliary Oncology at the University Hospitals Birmingham NHS Foundation Trust said: “These are very promising findings that demonstrate the potential use of dendritic cell vaccines in a widely prevalent and hard to treat cancer. With our approach to developing the vaccine, focusing on stimulation with multiple tumour antigens, we have shown a strong signal that we believe warrants testing in larger trials in patients with liver cancer.

“Dendritic cell vaccines also represent a potential additional immune therapy to add to current checkpoint inhibitors. Future studies will look at whether adding DC vaccination to standard immunotherapy can derive better outcomes for patient with HCC who show only modest responses to current checkpoint inhibitor drugs.”

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

Written by Louise Stanley on 03/07/2025. 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.

New UK network to strengthen regulatory environment for advanced therapies

Written by Louise Stanley on 12/02/2025. Posted in Birmingham Health Partners, Experimental medicine.

A new network has been established to foster collaboration among regulators, industry, and researchers to ensure there is a world-leading regulatory environment in the UK for advanced therapies that supports companies in the development, trial, and launch of these innovative treatments.

The network has been established by the Cell and Gene Therapy Catapult (CGT Catapult), an independent innovation and technology organisation specialising in the advancement of the cell and gene therapy industry, and the Birmingham Health Partners Centre for Regulatory Science and Innovation (CRSI), funded by Innovate UK and support from the MHRA.

The effective regulation of advanced therapies is vital to ensure that patients can access these transformative treatments promptly, to prepare the healthcare sector for the large-scale deployment of these therapies, and to ensure patient safety.

To help achieve this, the network will work with the UK regulators, predominately the MHRA, to identify effective and efficient regulatory strategies that address the unique challenges of these therapies. By sharing its recommendations with therapy developers and regulatory and healthcare stakeholders, it will support the UK to build a first-in-class regulatory ecosystem that welcomes and encourages healthcare innovation. This aims to enable the timely development of, and improved access to, safe and effective advanced therapies.

The network is one of seven new Centres of Excellence in Regulatory Science and Innovation established by Innovate UK to help shape the development and approval of medical innovations in the UK.

Matthew Durdy, Chief Executive of the CGT Catapult, said: “Advanced therapies are both an opportunity for patients and an opportunity for the UK. This initiative is part of a drive to keep the UK as a leader in this field.”

Melanie Calvert, Deputy Director of the Birmingham Health Partners Centre for Regulatory Science and Innovation, Director of the Centre for Patient Reported Outcomes Research, Professor of Outcomes Methodology at the University of Birmingham, NIHR Senior Investigator and co-lead of the NIHR Birmingham Biomedical Research Centre’s Patient Reported Outcomes research theme, said: “As the number of advanced therapies requiring regulatory approval increases, it is essential that regulatory frameworks are developed that are fit-for-purpose. Our focus at Birmingham is understanding how the patient voice can help shape regulatory decision making and ensuring that we understand the risks and benefits of treatment from the patient perspective. I am excited to leverage our extensive expertise in the field and work with our partners to provide patients with timely access to transformative treatments.”