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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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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A stem cell therapy trial at BHP founder-member University Hospitals Birmingham is having a life-changing effect on heart attack patients, with scans showing almost complete restoration of heart muscle function just weeks after the procedure. 

After a heart attack, around 30% of patients are left with severely damaged and weakened hearts which can, over time, lead to life-threatening heart failure, as the heart is unable to pump the blood around the body properly. The trial therapy aims to prevent this heart failure by regenerating the damaged muscle.

Developed by biotechnology company CellProthera, the therapy involves the application of a person’s own stem cells directly into their heart, through the femoral artery in the leg. The patient’s heart activity is then monitored for six months – at 1, 3 and 6 months – using echocardiography and magnetic resonance imaging (MRI).

66 year old Kim Smith is one of four patients recruited to the trial at UHB and was randomly allocated to the experimental arm of the study, meaning that she received the therapy as treatment following her heart attack. Just two weeks after receiving the therapy, Kim’s heart function had returned to almost normal (55%).

Kim said: “I now feel as though I can actually do what I used to do before. When I had the heart attack, I was worried that I was going to end up being stuck at home all day, but since having the stem cell therapy, psychologically and physically, I just feel so much better.

“I’m so grateful to have had this treatment, and I do hope that the research that they are doing goes forward because I think a lot of people would benefit. That was my reason for doing it in the first place – even if it does nothing for me, it could help someone else.”

So far, approximately 50 patients from the UK and France have been recruited to the trial, known as the EXCELLENT (Expanded Cell Endocardial Transplantation) study. The research is currently in its final phase, with results expected later this year.

Dr Sohail Khan, Consultant Interventional Cardiologist and Lead Investigator at UHB for the EXCELLENT study, said: “What we have seen so far is that actually the stem cells do seem to have a dramatic effect in terms of improving heart muscle function.

“Currently, there are few clinical options available that repair and regenerate heart tissue following a heart attack. As a result, the only option for many patients that have suffered a heart attack and developed advanced heart failure, is a fully invasive heart transplant. This is a very serious procedure for the patient, and very costly for society.

“The development of a cell therapy to regenerate cardiac tissue will be transformative for a considerable number of patients globally. A minimally invasive, cell therapy, that uses a patient’s own stem cells, could also considerably reduce treatment costs.”

Matthieu de Kalbermatten, CEO at CellProthera, said: “Bringing the therapy to market as a minimally invasive therapy is vital to tackle, from the root, the harmful effects of heart attacks and improve quality of so many lives.

“The impressive progress of the CellProthera EXCELLENT trial is a testament to the work of our team and our stakeholders. All of the trial sites in the UK and France are committed to admitting and treating the final patients as quickly as possible. In 2024, we will look to start the phase III trial, where we will be recruiting patients from across Europe, with the aim of potential future market authorisation and bringing this vital treatment to all patients.”