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Author: Louise Stanley

Clinical trials programme kicks off with pulse survey

As part of our ‘Reducing Bureaucracy in Clinical Trials’ programme, Birmingham Health Partners is seeking the views of research-active colleagues from across the research partnership to help inform our strategy and ensure patients get access to clinical trials, quicker.

BHP’s Clinical Trials working group – established in 2018 by Professor Pam Kearns – has highlighted a number of opportunities to increase efficiency and allow us to build on the growing national momentum to improve clinical trials delivery. Now, with the ultimate aim of reducing the overall time taken to set up academic clinical trials led within BHP, we are working to improve the experience of colleagues facilitating and navigating the set-up process and welcome views from Chief and Principal Investigators, trial management teams, R&D and support staff.

Senior Programme Lead Amy Smith said: “Input from colleagues across the BHP research community will be vital for our programme. By understanding your current experiences, we’ll be able to identify areas for improvement and ensure the right support is in place at every stage of the process.”

Sir David Nicholson, BHP board member and sponsor of the project, said: “This pivotal initiative is based on BHP’s shared belief that all patients should have the opportunity to take part in research, and the knowledge that research-active healthcare organisations perform better. We are all committed to working together to reduce bureaucracy and duplication of effort in clinical trials through this project, which will offer patients access to trials sooner and ensure innovations reach the clinic more quickly. The fantastic diversity of our regional population also means that our research, and the commercial innovations which result from it, will be applicable nationally and globally.”

The survey can be accessed at the following link – Reducing Bureaucracy Programme: Experience Survey – and is open to employees of any BHP member organisation involved in research delivery. The deadline for responses is Monday 12 August 2024.

The programme has been established to respond the challenges identified in recent reviews by Professor Adam Tickell and Lord O’Shaughnessy.

Children’s brain tumours could be diagnosed with 10-minute scan

Children with the most common malignant form of brain cancer could see diagnostic wait times dramatically reduced thanks to new research that trialled a quicker and less invasive way of determining which type of tumour they have. 

The study, published in eBioMedicine, was conducted by a team of researchers led by the University of Birmingham (UoB) and Newcastle University, with Birmingham Children’s Hospital as the lead clinical centre, and funded by Children with Cancer UK and Cancer Research UK. UoB and Birmingham Women’s and Children’s NHS Foundation Trusts are both founding members of BHP.

The collaborative team identified how the four different groups of medulloblastoma, a malignant children’s brain tumour, had a specific profile based on their individual metabolism. Taking cell samples from 86 tumours, a laboratory test was used to accurately identify metabolic markers including chemicals specific to the different tumour groups.  

The study also validated previous research that found that glutamate, a metabolite present across all of the tumour cells, is linked closely with tumour prognosis. 

Significantly, the research could pave the way for using MRI scanning combined with machine learning to assess medulloblastomas for their ‘signature’ metabolic profiles without the need for invasive biopsy and could rapidly reduce the current 3-4 week wait from presentation to full diagnosis. 

Andrew Peet, Emeritus Professor of Clinical Paediatric Oncology at the University of Birmingham and an Honorary Consultant at Birmingham Women’s and Children’s NHS Foundation Trust, who is lead author of the study said: “Time is so important in cancer diagnosis so our findings on different types of medulloblastoma having a detectable signature metabolism could be game changing for quickly diagnosing, and then offering the best possible treatment for children.”

Professor Steve Clifford, Chair of Molecular Paediatric Oncology at the Newcastle University Centre for Cancer, who jointly led the study said: “Providing a rapid diagnosis using innovative scanning and AI (artificial intelligence) techniques, has the potential to revolutionise patient management, allowing early non-invasive diagnosis, tailoring of treatment decisions and reducing the period of uncertainty for patients and parents while awaiting a full diagnosis. Further, our biological findings provide critical new insights into the metabolism underpinning these tumours, and the potential to exploit these therapeutically.”

The latest findings could be game changing for children like Jack Bourne, aged six, from Birmingham who was diagnosed with medulloblastoma in March 2023.

Jack’s dad Tom said: “We’ve been through 13 months of treatment but six weeks of that was just waiting to find out what type of tumour he had. We were so scared.”

Within weeks of starting school, Jack had started experiencing sickness and headaches which doctors put down to possible separation anxiety or vertigo. But when parents Tom and Tom and Suzanna noticed that he was struggling to walk, they sought a second opinion and Jack was referred to Birmingham Children’s Hospital the same day.

“When they told me the results of the MRI scan, I didn’t know what to feel,” said Tom. “As we were trying to digest everything, they were asking us to sign consent forms because they wanted to operate first thing the next morning. You’re reading these forms and all you see is – he might not make it out alive. It’s heartbreaking, it really is.”

Jack pulled through the ten-hour operation to remove the tumour, but it would take more than four weeks for doctors to figure out what specific type of medulloblastoma he had in order to effectively treat it.

“The research that’s going into diagnosing tumours is really important,” said Tom. “In Jack’s case there was quite a delay while they sent his tumour to Great Ormond Street to be analysed. During that time Jack was given some chemo just to start things off because they just wanted to do something rather than just wait. But all you want is for your child to be given the best possible treatment right from the start.” 

Christiana Ogunbote, Head of Research at Children with Cancer UK said: “We are incredibly proud to help fund this innovative medulloblastoma research and are excited to see how it could change the experiences of children diagnosed with this disease and their families. Discovering new ways to improve outcomes for children with cancer is at the heart of what we are trying to achieve. Through continued and sustained investments in research we look forward to a day where every child can survive their cancer diagnosis.” 

Dr Laura Danielson, Children’s and Young People’s Research Lead at Cancer Research UK, said:  “Developing quicker, less invasive ways to accurately diagnose the different types of medulloblastoma, the most common malignant brain tumour in children, is a crucial step in improving outcomes for young patients. 

“This important study has identified a new way to distinguish between the four subgroups of medulloblastoma. This discovery paves the way for the development of simple imaging tests that could quickly and accurately diagnose the different types of medulloblastoma. 

“This kind of discovery research is important to drive new and improved ways to better detect and treat cancers affecting children and young people.”

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Statisticians call for rigour and transparency in the evaluation of diagnostic tests

Recommendations – developed by a working group of statisticians – on reframing the evaluation of in vitro diagnostic tests have been published today by the Royal Statistical Society in its Series A journal. The group was co-chaired by Professor Jon Deeks, at BHP founder-member the University of Birmingham and former RSS President, Professor Deborah Ashby, at Imperial College London.

The report, which will be submitted to the UK Covid-19 Inquiry, is intended to help prevent future scenarios in which IVDs are marketed widely, but later attract serious concerns about the standards applied to their evaluation. Its co-authors include statisticians from the Universities of Oxford, Cambridge, Edinburgh, Birmingham and the London School of Hygiene and Tropical Medicine.

The research was prompted by concerns about the standards applied to the evaluation of diagnostic tests during the Covid-19 pandemic – particularly lateral flow tests – however the recommendations cover all new tests, especially those designed to detect infectious diseases.

It is published today in the RSS’s Series A journal and also presented at the Evidence Based Early Diagnosis conference at St Andrews.

The RSS Working Group on Diagnostic Tests set out 22 recommendations, designed to ensure that in vitro diagnostic (IVD) tests – which typically test samples of fluids such as blood, urine or saliva – are statistically robust and fit for purpose. The RSS Working Group identified Study-Design matters (10 recommendations); Regulation matters (6 recommendations); Transparency matters (6 recommendations).

Jon Deeks, Professor of Biostatistics at the University of Birmingham, said: “The Covid-19 pandemic provided a microcosmic insight into inadequacies in current processes to evaluate and regulate diagnostic tests. It’s important that we learn from these failures and establish robust processes that can be applied broadly across diagnostic tests.”

The report covers three areas of diagnostic testing: study-design of evaluations; regulation of tests; and transparency of test evaluations.

Key recommendations included:

  • Evaluation needs to take into account each specific intended use of the test, including the person being tested, the target condition and even the facilities where the testing will be done. Field or clinical evaluation studies should be carried out for each intended use.
  • Direct comparison of alternative IVDs and testing strategies should be available to inform clinical and public health decision-making.
  • The Medicines and Healthcare products Regulatory Agency (MHRA) should collaborate with independent experts to revise the national licensing process for IVDs. This will ensure public safety is protected. Protocols and reports for test evaluations should be publicly available to ensure transparency in all planning and decision-making.

The publication of the report is relevant for the opening of the ‘Test, Trace and Isolate’ module of the UK Covid-19 Inquiry. It also coincides with the MHRA’s recently-launched consultation on improved safety for high-risk diagnostic devices.

Professor Sheila Bird at the MRC Biostatistics Unit at the University of Cambridge, said: “Past Royal Statistical Society Working Party reports on matters which affect the public health have had enduring impact. Official Statistics – Counting with Confidence led to the UK Statistic Act of 2007; Statistics and Statisticians in Drug Regulation led to the appointment of professional statisticians by the UK, and later, European drug regulator; Statistical Issues in First-in-Man Studies led to safety-enhanced study-designs with open protocols. I hope that this month’s consultation by MHRA is indicative that Diagnostic Tests is making its mark already.”

Dr Andrew Garrett, President of the Royal Statistical Society, said: “The report provides a thorough evaluation of both diagnostic tests and diagnostic testing. It addresses how to develop, regulate, and use diagnostic tests in the future – a subject that is of increasing importance to individual and public health.”

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World-first colorectal cancer vaccine trial treats first UK patient in Birmingham

The Queen Elizabeth Hospital Birmingham (QEHB), operated by BHP founder-member University Hospitals Birmingham NHS Foundation Trust, has treated its first patient in England with a personalised vaccine against their bowel cancer, in a clinical trial which is part of NHS England’s new Cancer Vaccine Launch Pad (CVLP).

In a national first, father-of-four Elliot Pfebve received the developmental jab within the Clinical Research Facility at QEHB, one of several sites taking part in the colorectal cancer vaccine trial sponsored by BioNTech SE.

The trial is one of several that will be taking place in NHS trusts across the country to treat different types of cancer. Thousands more patients are expected to benefit from NHS England’s new CVLP, which will enable those wanting to participate in clinical trials to be fast-tracked to one of the nearest participating hospitals.

Patients who agree to take part have a sample of their cancer tissue and a blood test taken. If they meet a clinical trial’s eligibility criteria, they can be referred to their nearest participating NHS site, meaning patients from hospitals across the country will find it easier than ever to take part in groundbreaking research.

The investigational cancer vaccines evaluated in the colorectal cancer trial are based on mRNA – the same technology used for the Pfizer-BioNTech COVID-19 vaccine – and are created by analysing a patient’s tumour to identify mutations specific to their own cancer. Using this information, medics then create an experimental individualised cancer vaccine.

The developmental vaccines are designed to induce an immune response that may prevent cancer from returning after surgery on the primary tumour, by stimulating the patient’s immune system to specifically recognise and potentially destroy any remaining cancer cells.

The investigational cancer vaccines being jointly developed by biopharmaceutical companies BioNTech and Genentech, a member of the Roche Group, are still undergoing trials and have not yet been approved by regulators.

Higher-education lecturer Elliot, 55, had no cancer symptoms and was diagnosed through a routine health check with his GP.

A CT scan and a colonoscopy confirmed he had colon cancer and Eliott had surgery to remove the tumour and 30cm of his large intestine. He was then referred to the QEHB for initial rounds of chemotherapy and to take part in a clinical trial.

Eliott said: “Taking part in this trial tallies with my profession as a lecturer, and as a community-centred person. I want to impact other people’s lives positively and help them realise their potential.

“Through the potential of this trial, if it is successful, it may help thousands, if not millions of people, so they can have hope, and may not experience all I have gone through. I hope this will help other people.”

Thirty hospitals in England are already signed up to the pioneering Cancer Vaccine Launch Pad – one of the biggest projects of its kind in the world – with more sites joining the platform over the coming months.

The scheme aims to expand and work with a range of partners in the pharmaceutical industry to include patients across many cancer types who could potentially join a vaccine trial, such as those with pancreatic and lung cancer.

Principal Investigator for the trial at QEHB, Consultant Clinical Oncologist, Dr Victoria Kunene, said: “The investigational cancer vaccines are based on mRNA and are created by analysing a patient’s tumour to identify mutations specific to their own cancer. Using this information, we can create an individualised investigational cancer vaccine, but it is too early yet to say if these will be successful, though we are extremely hopeful.

“Based on the limited data we currently have of the in-body response to the vaccine, this could prove to be a significant and positive development for patients, but more data is yet needed and we continue to recruit suitable patients to the trial to establish this further.”

Amanda Pritchard, NHS chief executive, said: “Seeing Elliot receive his first treatment as part of the Cancer Vaccine Launch Pad is a landmark moment for patients and the health service as we seek to develop better and more effective ways to stop this disease. 

“Thanks to advances in care and treatment, cancer survival is at an all-time high in this country, but these vaccine trials could one day offer us a way of vaccinating people against their own cancer to help save more lives.

“The NHS is in a unique position to deliver this kind of world-leading research at size and scale, and as more of these trials get up and running at hospitals across the country, our national match-making service will ensure as many eligible patients as possible get the opportunity to access them.”

Trials have already enlisted dozens of patients, although the majority of participants are expected to be enrolled from 2026 onwards.

Professor Peter Johnson, NHS national clinical director for cancer at the NHS said: “We know that even after a successful operation, cancers can sometimes return because a few cancer cells are left in the body, but using a vaccine to target those remaining cells may be a way to stop this happening.

“Access to clinical trials could provide another option for patients and their families, and I’m delighted that through our national launch pad we will be widening the opportunities to be part of these trials for many more people, with thousands of patients expected to be recruited in the next year.”

Executive Director of Research and Innovation at Cancer Research UK, Iain Foulkes, said: “It’s incredibly exciting that patients in England are beginning to access personalised cancer vaccines for bowel cancer.

“This technology pioneers the use of mRNA-based vaccines to sensitise people’s immune system and in turn detect and target cancer at its earliest stages. Clinical trials like this are vital in helping more people live longer, better lives, free from the fear of cancer. If successful, the vaccine will be a game changer in preventing the onset or return of bowel cancer.”

Last year, the Government signed an agreement with BioNTech to provide up to 10,000 patients with precision cancer immunotherapies by 2030.

BioNTech has already begun conducting clinical trials in the UK, and the NHS launch pad is helping to accelerate the identification of eligible patients for those trials in England.

The vaccines being tested as part of the trials aim to help patients with different types of cancer and, if successfully developed, researched and approved, cancer vaccines could become part of standard care.

The NHS is working in partnership with Genomics England on the launch pad, with work already helping patients access the latest testing technologies and ensures they are given more targeted precision treatments for their cancer.

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E-MOTIVE wins prestigious Trial of the Year Award

The landmark E-MOTIVE study, led by University of Birmingham researchers and coordinated by the Birmingham Clinical Trials Unit, as been awarded ‘David Sackett Trial of the Year Award’ by the Society of Clinical Trials, recognising the importance of the findings and the potential impact as the simple, low-cost approach is rolled out around the world, dramatically improving maternal health across the globe. The trial tested a package of low-cost interventions that resulted in a 60% reduction in heavy bleeding following childbirth.

Each year the award goes to a randomized, controlled trial published in the previous calendar year that is considered to improve the lot of humankind and provide the basis for substantial, beneficial change in healthcare, amongst other criteria.

“This has been the largest set of nominations for the Trial of the Year Award in all my time on the committee. We received numerous nominations for worthy trials, from around the world and across a large number of clinical disciplines – including obstetrics, emergency medicine, infectious disease, and cancer. We had a challenging time as a committee to choose a winner” said Andrew Cook, Chair of the SCT David Sackett Trial of the Year Committee.

Postpartum haemorrhage (PPH), or severe bleeding after birth, is the leading cause of maternal deaths worldwide. It affects an estimated 14 million women each year and results in around 70 000 deaths – mostly in low and middle-income countries – equivalent to 1 death every 6 minutes. The E-MOTIVE study found that objectively measuring blood loss using a simple, low-cost collection device called a ‘drape’ and bundling together WHO-recommended treatments – rather than offering them sequentially – reduced severe bleeding by 60%, and women were less likely to lose their life.

Dr Adam Devall collected the award, on behalf of the E-MOTIVE team, from the Society of Clinical Trials 45th Annual Meeting, in Boston, USA, and said: “I’m honoured to accept the Trial of the Year Award on behalf of the E-MOTIVE project. E-MOTIVE was a huge international team effort, and this award speaks to the dedication of teams at each of our 80+ sites. More high-quality clinical trial evidence is desperately needed for pregnancy and maternal health so we’re delighted to receive this recognition of our work and the impact it will have on deaths from PPH.”

Professor Arri Coomarasamy, who led the E-MOTIVE trial and is the Co-Director of the WHO Collaborating Centre on Global Women’s Health at the University of Birmingham said: “This new approach to treating postpartum haemorrhage could radically improve women’s chances of surviving childbirth globally, helping them get the treatment they need when they need it”.

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Birmingham Health Partners announces theme leads to drive strategy

The second city’s clinical-academic alliance, Birmingham Health Partners, has appointed three strategic theme leads to support the implementation of its new five-year mission: to work together, transforming Birmingham’s healthcare through high impact innovation.

Taking the new role of Health Inequalities Lead, Dr Joht Singh Chandan is a Clinical Associate Professor in Public Health at the University of Birmingham where his research focuses on identifying and addressing health inequalities – with a particular interest in abuse and violence prevention inspired by many years of working as a voluntary police officer.

As the UK’s third-poorest city, with a diverse ethnic profile and socioeconomic demographics, Birmingham experiences significant health disparities. Joht will develop a detailed action plan for improving population health in the city, underpinned by his experience of issues that impact widely on health and wellbeing; factors that prevent early detection; and barriers to accessing healthcare.

Joht said: “We shouldn’t see reducing health inequality as just the responsibility of public health bodies. The determinants of inequality are so interlinked that not only can we not tackle issues in isolation, we can’t tackle them as one institution. Working across the partnership and linking health data platforms, we’ll be able to work in a much more representative and inclusive way to improve physical and mental health outcomes for our local communities.”

Tasked with optimising data integration across the partnership, Professor Simon Ball has been appointed Academic Lead for Data. A Consultant Nephrologist, Simon has had various roles in developing electronic health care records and using data to improve patient care and support research. His other roles currently include Associate Director for the Midlands Health Data Research UK and Senior Responsible Officer for the West Midlands Secure Data Environment (WMSDE).

Simon said: “NHS Trusts in Birmingham were among the first to adopt electronic health records systems, meaning we have access to a wealth of data – including blood tests, scans and biopsies – spanning several decades. This can provide valuable insight into an array of diseases, health conditions and care pathways – but only if it is integrated. Working together, BHP and the WMSDE can ensure our data is analysed, learnt from and used to optimise healthcare across our region.”

Leading the ‘Reducing Bureaucracy in Clinical Trials’ programme is Amy Smith, an experienced Senior Programme Lead with considerable experience in clinical trials across multiple BHP NHS Trusts and NIHR infrastructure. The programme responds to the challenges identified by Professor Adam Tickell and Lord O’Shaughnessy, ensuring patients in Birmingham get access to clinical trials more quickly. 

Amy said: “I am very proud to be leading this exciting project which showcases BHP as a leader in clinical trials.  Through trust, transparency, and collaboration we will harness the extensive knowledge and expertise within BHP, delivering improved setup times. Ultimately our aim is to make trials accessible to a diverse range of patients, quicker – increasing our attractiveness to funders and industry partners.”