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£12m research centre will improve efficiency of rare disease trials to unlock tests and treatments

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

Researchers from BHP founder member the University of Birmingham are part of a new £12m research centre to improve clinical trials for rare diseases.

The LifeArc Centre for Acceleration of Rare Disease Trials brings together a consortium of three universities from across the UK. Newcastle University, Queen’s University Belfast, and University of Birmingham are pooling their expertise in a partnership coordinated by Professor David Jones, Professor of Liver Immunology at Newcastle University.

The £12m centre will focus on improving the efficiency of rare disease trials and increasing the number of opportunities for patients to take part, through a new UK ‘4 nations’ approach to deliver trials of new treatments using ‘one stop’, patient friendly models.

The team will do this by creating a rare disease trial recruitment portal and will design and deliver trials in partnership with patients. This will speed up the delivery of clinical trials for people with rare diseases and enable more rapid approval of new therapies for use in the NHS.

Professor Timothy Barrett, Director of the Centre for Rare Disease Studies at the University of Birmingham commented: “Birmingham is justly proud of its hospital services and scientific research for people living with rare conditions, which build on our partnership between hospitals and University and reflects the cosmopolitan nature of our region. 

“This award will represent a stepping stone in our ambition for patients in Birmingham to get more treatments to more people with rare diseases, faster. It also allows us to expand capacity for rare disease clinical trials for the whole of the UK.”

Kerry Leeson-Beevers is the parent of a child with the rare genetic condition, Alström Syndrome, which often causes loss of vision and hearing, and can lead to serious life-threatening problems with the heart, liver and kidneys.

Kerry, who is also CEO of Alström Syndrome UK, explained: “We have no specific treatment for Alström Syndrome and when my son, Kion, was a baby, I was told it could take around 10 years for any treatment to be developed. 20 years later, we are still waiting. People living with rare conditions don’t have the luxury of time and the mainstream way of delivering healthcare and drug development rarely works for people with rare conditions.

“As a mum and the Chief Executive of Alström Syndrome UK, having a centre that will deliver a coordinated, inclusive and supportive approach to accelerate clinical trials gives me great hope.”

The LifeArc Centre for Acceleration of Rare Disease Trials, along with the the LifeArc Centre for Rare Respiratory Diseases, LifeArc Centre for Rare Kidney Diseases, and LifeArc Centre for Rare Mitochondrial Diseases, has been awarded a share of nearly £40M over five years from the not-for-profit medical research charity, LifeArc.

Each centre will tackle an area of unmet need, to unlock science, accelerate medical progress and have the greatest impact for patients.

Dr Catriona Crombie, Head of Rare Disease at LifeArc, said: “We’re extremely proud to be launching four new LifeArc Translational Centres for Rare Diseases. Each centre has been awarded funding because it holds real promise for delivering change for people living with rare diseases. These centres also have the potential to create a blueprint for accelerating improvements across other disease areas, including common diseases.”

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Midlands-Wales Advanced Therapy Treatment Centre part of £17.9m network renewal

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

The Advanced Therapy Treatment Centre network includes the Midlands-Wales Advanced Therapy Treatment Centre, jointly delivered by BHP founding members the University of Birmingham and University Hospitals Birmingham.

The National Institute for Health and Care Research (NIHR), Innovate UK, the Advanced Therapy Treatment Centre Network and the Cell and Gene Therapy Catapult (CGT Catapult) have announced a £17.9 million strategic initiative to keep the UK as a location of choice for advanced therapy research and advanced therapy medicinal product (ATMP) clinical trials.

The initiative will provide a further four years of funding for the Advanced Therapy Treatment Centre Network (ATTC Network) which is currently composed of three centres: Innovate Manchester Advanced Therapy Centre Hub; Midlands-Wales Advanced Therapy Treatment Centre; and the Northern Alliance Advanced Therapies Treatment Centre.

The Midlands-Wales centre has multiple sites across England and Wales, with the Birmingham hub being jointly delivered by BHP founding members the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust, directed by Professor Philip Newsome from the University’s Institute of Immunology and Immunotherapy. Its aim has been to enable UK advanced therapy companies to reach the clinical market, whilst simultaneously building clinical capacity and capability regionally to deliver these breakthrough therapies to patients. It brings together a wide range of specialists in advanced therapy manufacturing including academic and commercial partners, logistics companies, specialists in clinical trial delivery and teams focussed on IT solutions and health economics.

Professor Philip Newsome, Director of the Midlands and Wales Advanced Therapy Treatment Centre and national clinical lead, commented: “This funding will accelerate the delivery of advanced therapy trials across the Midlands, Wales and beyond. It is an exciting time for patients, researchers and industry as new therapies are trialled and enter routine clinical care.”

The UK is a world leader in ATMP clinical research with 175 ongoing trials being carried out here, and with 9% of global ATMP trials having representation in the UK. Many more products are in development and further action is needed to ensure that the NHS is able to bring advanced therapies to patients at scale across the UK.

Through this further funding, and in close collaboration with NIHR infrastructure and the devolved equivalents, the ATTC network aims to build on its work on advanced therapy clinical trial readiness to ensure the UK maintains its position as a globally attractive location for clinical research.

Health Minister Andrew Stephenson said: “This investment reaffirms the UK’s position as a global leader in clinical research. It will help roll out revolutionary medical products more quickly, potentially treating the root cause of disorders and diseases like Alzheimer’s and cancer. Harnessing technological and digital innovations is one of our primary focuses under the first ever NHS Long Term Workforce Plan, enabling new and advanced ways of working.”

Dr Stella Peace, Executive Director for the Healthy Living and Agriculture Domain at Innovate UK, said: “From our initial investment to now overseeing the delivery of the new four-year programme, our goal is to ensure the UK maintains its global leadership in clinical research. Our commitment to fostering innovation and scientific advancements is crucial for sustaining this leadership. This drives medical breakthroughs, as well as strengthening the UK economy by attracting investments, generating high-skilled jobs, and positioning us at the forefront of transformative healthcare discoveries.”

Professor Marian Knight, Scientific Director for NIHR Infrastructure, commented: “The NIHR is committed to ensuring that the UK provides a research environment to enable rapid assessment of new advanced therapies with the potential to transform health and care. Partnerships such as these, linked with existing NIHR research infrastructure, will help ensure that the UK public is able to benefit from these ground-breaking new treatments.”

Matthew Durdy, Chief Executive of the Cell and Gene Therapy Catapult, added: “Advanced therapies have the potential to transform healthcare, providing a range of new, lifechanging treatments to patients. Thanks to far-sighted investments, like this commitment by NIHR and the on-going support of Innovate UK, the UK is recognised globally as a pioneer in advanced therapies. With the continued great work of the ATTC network, we hope to further build the reputation of the UK.”

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Understanding pregnancy: Accelerating the development of new therapies for pregnancy-specific conditions

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

During pregnancy, women and pregnant individuals who do not identify as women* can develop a range of pregnancy-specific conditions, such as preeclampsia and gestational diabetes, that can adversely affect both their own health and that of the developing foetus during the pregnancy. These conditions can affect the lifelong health of both mother and child. Despite the danger that these conditions present to mother and baby, there are few approved, safe and effective medicines to treat them, and limited investment in novel therapy development.

To map out key barriers and potential enablers of preclinical research and experimental medicine to support the development of new medicines for pregnancy-specific conditions, the Academy of Medical Sciences, Birmingham Health Partners, and Concept Foundation organised a multi-sectoral FORUM workshop in September 2023. People with lived experience joined representatives from academia, the commercial sector, clinical practice (including doctors and midwives), regulatory authorities, funding bodies, charities, and patient advocacy groups at the meeting.

The result of this workshop is a new report – Understanding pregnancy: Accelerating the development of new therapies for pregnancy-specific conditions – which highlights the need to raise awareness of the importance of research in pregnancy, and give women opportunities to participate.

> Understanding pregnancy: Accelerating the development of new therapies for pregnancy-specific conditions – view and download the report here

This work builds upon the BHP-led Pregnancy Policy Commission which in 2022 published its Healthy Mum, Healthy Baby, Healthy Future: The Case for UK Leadership in the Development of Safe, Effective and Accessible Medicines for Use in Pregnancy report, proposing a clear roadmap to improve the lives of millions of people, not just for women while they are pregnant, but for future generations.

Professor Peter Brocklehurst, Emeritus Professor at BHP founder member the University of Birmingham, commented: “We need to better understand the biological mechanisms of pregnancy-specific conditions so that we can develop therapies that target these processes. To do this, we need more health data and biological samples from women with those conditions.”

Forum participants identified the following six priority areas for next steps:

    1. A cross-sectoral and cross-speciality network or coalition, including women with lived experience, to provide a platform for collaboration and to coordinate efforts to promote the development of new medicines for pregnancy-specific conditions.
    2. Additional interdisciplinary research and cross-sector collaboration to address key knowledge gaps (including the biology of the placenta, of the early stages of pregnancy, and of pregnancy-specific conditions), to enable appropriate use of animal models and physiologically based pharmacokinetic (PBPK) modelling, and to leverage routinely collected health data and patient samples.
    3. The establishment of a more enabling environment for research in pregnancy, for example through development of a stronger research base and a more supportive regulatory environment.
    4. Greater engagement with women to raise awareness of the importance of research into pregnancy and of opportunities to participate in this research, including when women contact the healthcare system.
    5. Education and training of healthcare professionals, including midwives, to promote research in pregnancy.
    6. Advocacy to secure greater prioritisation of research in pregnancy (and women’s health more generally) by policymakers, funders, and higher education institutions.

The workshop was chaired by Professor Peter Brocklehurst FMedSci, Emeritus Professor of Women’s Health at the University of Birmingham, and Dr Pauline Williams CBE FMedSci, an independent pharmaceutical medicine consultant and former Senior Vice-President and Head of Global Health R&D at GlaxoSmithKline.

* The Academy acknowledges that not all pregnant people identify as women. While the terms ‘woman’ and ‘mother’ are used here, many of the learnings from the workshop about obstetric/pregnancy-specific conditions are expected to be widely applicable. It is recognised that there will be specific experiences and challenges associated with obstetric conditions among pregnant individuals who do not identify as women that were not explored at the workshop given the lack of specific research in this area. 

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Major contract awarded for a concussion research programme from the US Department of Defense

Written by Louise Stanley on . Posted in Birmingham Health Partners, Clinical trials, Early diagnosis and detection.

Birmingham experts in neurology have been awarded a seven-year contract by the U.S. Department of Defense – which could be worth up to $15.5m – to undertake a major research programme aiming to transform the way concussion is identified and managed.

Some 890 people across the UK, aged 18 to 60, will take part in the study which will measure the ability of a range of biomarkers – such as blood and saliva, mental health, vision, balance and sleep – to predict long-term complications from mild Traumatic Brain Injury (mTBI), also known as concussion. The  mTBI-Predict study will see researchers measure effectiveness of various methods to predict outcomes of mTBI after six, 12 and 24 months.

mTBI can be caused by physical impact to the head through accident, injury, sport, or even from shockwaves following explosions. Led by the Royal Centre for Defence Medicine (RCDM) and the University of Birmingham, researchers will use the UK TBI Research Network to recruit both civilian and military participants to the programme.

mTBI Predict will be supported by Birmingham Health Partners and University Hospital Birmingham, as well as a range of research institutions across the UK.

Professor Alex Sinclair, from the University of Birmingham, who will lead the study said: “Concern around the long-term effects of concussion is mounting. Even a minor injury to the head can cause concussion, which leads to brain injury with potentially serious effects on both immediate and long-term health.

“We have no precise way to tell who will have a serious consequence after a concussion. This means we can’t tell which patients will need more intensive treatment and which will recover spontaneously. The mTBI Predict research program will identify new ways to accurately predict whether concussion patients will develop long-term complications.”

Concussion has been declared a major global public health problem, with 1.4 million hospital visits due to head injury annually in England and Wales. Some 85% of these are classified as concussion and it is also estimated that up to 9.5% of UK military personnel in a combat role are diagnosed with concussion every year.

Major General Timothy Hodgetts CB CBE KHS, Surgeon General of the UK Armed Forces, commented:

“UK Defence has funded the initiation of this research, but it would not be possible to complete without the support from US DoD. This is a prime example of our longstanding bilateral research collaboration where we have a common purpose to address a significant and shared clinical problem. This study will be definitive in helping us identify those who need the most help and resources following a very common injury.”

The research programme brings together a team of experts including neuroscientists, psychologists, sport and exercise scientists, software developers and statisticians – coordinated by Birmingham Clinical Trials Unit.

The study will recruit patients with concussion related to sports injuries, road accidents, cycling accidents, falls and accidents at work, and military personnel experiencing concussion during training or active duty. It will involve military patients and expertise from the Defence Medical Rehabilitation Centre Stanford Hall and Royal Centre for Defence Medicine.

Dr. David J. Smith, from the US Department of Defense, commented: “The US Department of Defense is excited to support this study and continue to identify threats to the brain, such as blast overpressure, head impact, directed energy, and environmental hazards.

“These threats may have a direct impact on brain health. Our aim is to reduce risks to the brain, monitor exposures, and document them for long-term review. The goal is to look for multiple protection strategies to decrease exposures and protect brains better. This research will play a pivotal role in continuing our research investments partnering with the UK to better understand mTBI and concussion to prevent and reduce their effects.”

Although classed as mild brain injury, concussion leads to a disproportionate impact on future health, with three in 10 patients unable to work 12 months after their injury. The consequences of mTBI are profound, with many patients suffering long-term disability due to persistent headaches, imbalance, memory disturbance and poor mental health.

mTBI-Predict will look at biomarkers to enable faster diagnosis and assessment of a concussion, leading to improvements in treatment and long-term management, enabling a quicker return to play, work or duty.

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University of Birmingham awarded £7m NIHR funding to provide strategic leadership for its Research Support Service

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

The NIHR has selected BHP founder-member the University of Birmingham to run the National Collaborative for its Research Support Service (RSS) from 1 February 2024. 

The RSS was launched on 1 October 2023. The service provides expert research design, methodological support, advice, and collaboration to all researchers in England throughout the pre- and post-application/research process, regardless of geographic location and research interest. 

Since launch, the service has received more than 840 requests for support from researchers through its 8 hubs. The specialist centres in social care and public health have received a combined total of 171 requests for support.   

The new function

The new RSS National Collaborative will be run by the University of Birmingham. The university has demonstrated a clear commitment to work with all NIHR RSS hubs through collaboration. 

Among other responsibilities, the new function will:

  • provide strategic and operational leadership across the 8 individual NIHR RSS hubs
  • develop collaborative working with other components of the NIHR’s infrastructure. This includes the NIHR Clinical Research Network and incoming Research Delivery Network, in particular around the deliverability of studies within the chosen setting
  • identify, develop and share standards of good practice. The function will support their implementation, and provide the highest quality resources to support the development and delivery of health and care research
  • support Research Inclusion, and Patient and Public Involvement, Engagement and Participation in the hubs, to encourage a coordinated national approach

The aim of the new function is to enable the 8 hubs to provide consistency of service across the RSS. It will supply a core team, with dedicated senior academic, clinical and operational leadership. 

Professor Marian Knight, NIHR Scientific Director for Research Infrastructure, said: “The National Collaborative is critical to the successful delivery of the new Research Support Service, which since October has been providing expert advice and support to researchers across England. By leading the Collaborative, the University of Birmingham will work with the other RSS Hubs to ensure all researchers across England are provided with the tailored support they need and to develop an environment of continuous improvement across the service.”

Neil Thomas, Professor of Epidemiology and Research Methods, Operations Director of the RSS National Collaborative, said: “I am looking forward to leading this exciting collaboration alongside Professor Katie Morris, Director of the Birmingham Clinical Trials Unit, who is our Academic and Clinical Lead. We look forward to working with all 8 RSS Hubs and Specialist Centres, the NIHR and the wider research community in developing and delivering health and care research. Our vision of collaboration is wide ranging and we have ambitious plans to build capacity in our workforce and public contributors, contribute to the development of inclusive and innovative research methodologies, as well as harmonise systems across our service.”

Find out more about the Research Support Service.

LIBERATE heart attack trial treats first patient

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

The first patient has been treated in the LIBERATE clinical study to evaluate glenzocimab efficacy in myocardial infarction, which involves BHP members the University of Birmingham and University Hospitals Birmingham NHS Foundation Trust (UHB).

In 2022, the University  signed a partnership agreement with Acticor Biotech to evaluate glenzocimab efficacy in myocardial infarction in a new clinical trial called LIBERATE.

Having obtained full regulatory approval in August 2023, two clinical research sites, the Queen Elizabeth Hospital in Birmingham – part of UHB – and the Northern General Hospital in Sheffield, are involved in the study. The Queen Elizabeth Hospital opened to recruitment on 24th January 2024. It is expected that the Northern General Hospital in Sheffield will also open to recruitment by the end of February 2024.

The LIBERATE study, a randomiSed, double-blind Phase 2b trial, will enrol over 200 patients diagnosed with ST-elevation myocardial infarction (STEMI) and scheduled for percutaneous coronary intervention. The primary objective of the study is to evaluate both the safety and efficacy of glenzocimab at a dosage of 1000 mg compared to a placebo, specifically focusing on the reduction of myocardial infarct size at Day 90 post-treatment.

Professor Jon Townend, Chief Investigator of the trial who works across BHP members the University of Birmingham and UHB as Consultant Cardiologist and Honorary Professor of Cardiology, said: “We have entered the operational phase of the trial, and I extend my gratitude to the entire team in Birmingham and Sheffield for their outstanding efforts in managing patient recruitment in these critical emergency care settings, as well as for gathering qualitative data for subsequent analysis.”

Dr Mark Thomas, Associate Professor of Cardiology at the University of Birmingham and Honorary Consultant Cardiologist, who designed the trial and led its development, said: “This is the first time worldwide that this class of medication has been investigated in patients with heart attacks, after showing great promise in patients with stroke. We are grateful to our patients for helping us in our mission to find new treatments that may help to reduce the damage done by heart attacks.”

Professor Robert Storey, Professor of Cardiology at the University of Sheffield and Honorary Consultant Cardiologist and Director of the Cardiovascular Research Unit at Northern General Hospital, said: “This study is exploring the potential of glenzocimab in reducing the type of blood clotting responsible for heart damage during heart attacks. This exciting collaboration with University of Birmingham and Acticor Biotech holds the potential to bring significant benefit to people suffering from a heart attack.”

Adeline Meilhoc, Head of Global Clinical Development of Acticor Biotech said: “We are delighted to witness the operational start of the study, and we reiterate our complete confidence in our partners as crucial contributors of its success. Acticor Biotech is dedicated to advancing treatments for the acute phase of thrombotic diseases. Glenzocimab application in ST-segment elevation myocardial infarction (STEMI) represents a significant focal point for Acticor Biotech’s commitment to medical advancement.”