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Rare disease trials to develop playbook for testing new treatments

Written by Louise Stanley on . Posted in Clinical trials.

Patients with rare diseases could benefit from a ‘revolution’ in clinical trials that could see one-stop studies designed to provide robust results even with small numbers of participants.

The CAPTIVATE node is part of the recently introduced UK Rare Disease Research Platform established as part of a £14 million investment over five years by the Medical Research Council (MRC) and the National Institute for Health and Care Research (NIHR).

Led by BHP founder-member the University of Birmingham along with collaborators at the Universities of Sheffield and Liverpool, the CAPTIVATE node will be developing a methodology to run a ‘one-stop-study’, which would encompass all phases of clinical trials to enable the efficient evaluation of treatments for rare diseases.

The CAPTIVATE node will bring together the UK’s leading trial experts with hospital researchers experienced in rare diseases, industry partners, policy makers and patient partners, and the resulting designs could speed up the approval of medicines for use in rare diseases.

Professor Lucinda Billingham at the University of Birmingham is the lead academic for the CAPTIVATE node of the MRC-NIHR UK Rare Disease Research Platform. She explained: “One of the biggest challenges with developing new treatments or improving existing ways of treating rare diseases is that the model of clinical trial that is used as standard is incredibly difficult to run where there may be only hundreds of people around the world at any one time with that disease.

“Added to this fact, in the UK three quarters of rare diseases affect children and more than 30% of children with a rare disease die before their fifth birthday.

“The CAPTIVATE node is looking to scale a revolutionary model of clinical trial that runs all the phases together and can get clinically significant results even with very small numbers of patients. We want to find ways that need as few people to take part as possible, that are quicker and that provide all the information needed for the authorities to approve a new medicine to be prescribed to people with rare diseases from one single trial.”

Bringing together strengths in rare diseases

The MRC-NIHR UK Rare Disease Research Platform is now getting up and running and will bring together UK strengths in rare diseases research to improve diagnosis and treatment through better understanding of the disease.

It is made up of a central coordination and administrative hub and 11 specialist nodes based at universities across the UK, including the CAPTIVATE node at the University of Birmingham.

The aim of the platform is to bring together expertise from across the UK rare disease research system to foster new and innovative treatments for those directly and indirectly impacted by rare conditions.

Professor Lucy Chappell, Chief Executive of the NIHR, commented: “The UK Rare Disease Platform marks a significant advance in accelerating rare disease research, supported through NIHR funding and our partners the Medical Research Council. The platform will enable greater collaboration between patients and those working across academic, clinical and industry research. By bringing the right people and expertise together, we will be able to provide better care more quickly to those living with rare diseases.

“The NIHR continues to lead essential ongoing research into rare diseases, including through our Biomedical Research Centres, and we are making it easier for people with rare diseases to take part in research opportunities via our Be Part of Research Service, which can now be accessed through the NHS app in England. We look forward to working with our partners further to accelerate our understanding and treatment of rare diseases affecting millions of people across the UK.”

Stem cell therapy for heart attack patients trialled at UHB

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

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.”

NIHR awards £4m to Birmingham Clinical Research Facility to enhance the delivery of research

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

The Birmingham NIHR Clinical Research Facility (CRF) has been awarded £4 million from the National Institute for Health and Care Research (NIHR).

This funding is part of a total investment of £96 million that has been awarded to NHS organisations across England, to enhance the delivery of research through improving research facilities for patients across the NHS, helping teams to find new ways to prevent and treat diseases.

The Birmingham NIHR CRF is a collaboration between three BHP members – University Hospitals Birmingham NHS Foundation Trust (UHB), Birmingham Women’s and Children’s NHS Foundation Trust (BWC) and the University of Birmingham (UoB).

In Birmingham, new equipment will enable the delivery of innovative health technology services, including a range of cell and gene therapies requiring specialist pharmacy facilities. For example, onsite isolators will support preparation within the research facilities, speeding up the delivery of CAR-T trials for patients with haematology (blood) cancers, as well as trials into cancer vaccines for patients with a variety of solid tumour cancers.

The funding will also provide new laboratory equipment, essential for storing samples from patients recruited to metabolic research studies. This will support researchers with ground breaking work in the prevention, treatment and management of metabolic disease for adults and children across Birmingham.

The bid was led by Jo Gray, NIHR Birmingham Clinical Research Facility Clinical Manager, who said: “This successful bid for equipment and upgrades to our facility is fantastic and will make such a difference to patients and research teams across all partner sites.

“We have ambitious plans in place to upgrade existing equipment, as well as increase our ability to support new research across the sites. New equipment will include a paediatric ‘peapod’, which measures body composition and growth in premature born infants, and biosafety cabinets, which provide an enclosed ventilated space for drug preparation.

“Our portfolio of gastrointestinal (GI) and respiratory studies will also be able to grow with the addition of new endoscopy equipment. One of the key GI trials to benefit will be the INCEPTION clinic for patients with Inflammatory Bowel Disease (IBD). This trial aims to improve diagnosis and prognosis, and make more informed decisions on IBD treatment through biomarker discovery and implementation, by understanding more about how different microbiomes affect gut and oral immune responses.”

Professor Lorraine Harper, NIHR Birmingham Clinical Research Facility Programme Director, added: “We are tremendously proud that our hospitals have been selected for this award from the NIHR to improve the delivery of research in our local population. The award, which builds on existing clinical-academic collaborations facilitated by Birmingham Health Partners, will benefit our adult and paediatric patients, improving their access to new therapies and treatments, which can potentially be life-changing.”

Dr. Jan Idkowiak, NIHR Birmingham Clinical Research Facility Director (BWC), said: “This is very exciting. This award allows us to expand our ability to deliver cutting-edge clinical research beyond the Children’s Hospital, as it will enable us to include pregnant women and their children at the Women’s Hospital. This will be a huge benefit for our patients and will offer great opportunities to develop new therapies.”  

Professor Philip Newsome, Director of the NIHR Birmingham Biomedical Research Centre (BRC), commented: “This is fantastic news for both the CRF and for the organisations that work with them, like the Birmingham BRC. The CRF has been a key partner in delivering many of our experimental research studies, and we collaborate closely on our training, patient and public involvement, and equality, diversity and inclusion programmes too. We are looking forward to continuing working together to deliver patient benefit.”

Birmingham’s world-leading cancer trials unit gets £10m boost

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

A new £10m grant from Cancer Research UK will ensure that adults and children with cancer continue to benefit from world-class clinical trials led by the University of Birmingham.

The news has been hailed as a ‘major boost for patients’ by both clinicians and cancer survivors.

The Cancer Research UK Clinical Trials Unit (CRCTU) at BHP founder-member the University of Birmingham has already achieved significant progress in the treatment of cancer in the UK and internationally, including establishing new standards of treatment for the rare bone and soft tissue cancer, Ewing Sarcoma.

Scientists at the centre have also transformed the management of some types of prostate cancer and introduced treatment innovations for patients with blood cancers.

The new grant will allow researchers working on more than 100 national and international trials to continue developing safe and effective treatments as well as new tests for cancer over the next five years.

Professor Pamela Kearns, Director of the University of Birmingham-based CRCTU unit and children’s cancer expert, said:

“The renewal of funding for cancer trials in Birmingham is a major boost for our research here and we are delighted to continue working with research teams and patients to find new solutions in cancer care. Our clinical research enables us to translate discoveries from the lab and accelerate the improvement of cancer treatments, giving more patients the best chance of beating their disease.

“As a paediatric oncologist, I am particularly pleased this funding will allow our unique Children’s Cancer Trials Unit at Birmingham to continue to design and run clinical trials to improve the care of children with cancer.

“For example, with support from Cancer Research UK, we are leading International trials for children and young people with difficult to treat cancers like FaR-RMS; a trial testing innovative new treatments for rhabdomyosarcoma and the BEACON 2 trial, testing a range of new combinations of therapies for children and young people with a type of childhood cancer called neuroblastoma, at a stage where they have failed to respond well to standard treatments.”

The Birmingham CRCTU will combine strengths in innovative clinical trial methods with outstanding scientific and clinical expertise nationally and internationally to deliver new clinical trials, across all age groups over the next five years. The CRCTU will work alongside the Birmingham Experimental Cancer Medicine Centre, which is also funded by Cancer Research UK and the National Institute for Health and Care Research on trials to tackle more complex types of cancer and for cancers of unmet need.

Professor David Adams, Head of the College of Medical and Dental Sciences and Pro-Vice-Chancellor at the University of Birmingham said:

“The CRCTU is a jewel in the crown of our research portfolio across the University and I am delighted that with this latest funding we will continue to conduct internationally leading research to find better treatments and tests for cancer.

“Together with the ongoing funding for our Experimental Cancer Medicines Centre, the University is ideally placed to continue advances in cancer research which has a hugely significant role in society today. With unprecedented challenges for our NHS and after the effect that the pandemic has had on waiting lists and access to care, we need more than ever to have quick, effective and safe care for cancer.”

The team coordinates ground-breaking clinical trials across the UK and internationally, as well as regionally through Birmingham Health Partners (BHP) – a strategic alliance between seven higher education and health institutions including the University of Birmingham, University Hospitals Birmingham NHS Foundation Trust and Birmingham Women’s and Children’s NHS Foundation Trust.

Improving outcomes – Francesca’s story

Cancer survivor Francesca Williams was one of 640 patients across Europe to benefit from a trial led by the Birmingham centre that has significantly improved outcomes for children and adults with Ewing Sarcoma.

Diagnosed with a tumour in her rib bone just weeks after her 27th birthday in July 2017, Francesca had 15 sessions of chemotherapy and five weeks of radiotherapy back-to-back over ten months. This was followed by major surgery at Heartlands Hospital in April 2018 to remove the remainder of her tumour and rebuild her chest wall using muscle from her back.

Despite going through medically induced menopause and having no time to store any eggs for fertility treatment, Francesca is expecting her first baby in July.

“I feel so lucky to have been part of the trial,” said Francesca, a 32-year-old English teacher who now lives in Austria.

“The worst thing for me was thinking I wouldn’t be able to have children so to find out I was pregnant last year was incredible. I’m really excited about becoming a mum.”

The European-wide trial EE2012, run by the University of Birmingham’s Cancer Research Clinical Trials Unit, tested the standard chemotherapy treatment plan against a new experimental treatment plan in children and adult patients from ten European countries.

The trial – a shorter treatment than the previous standard – found that six per cent more patients were cancer-free after three years, with fewer toxic side-effects. Results were so conclusive that the trial finished early in 2019 and the new treatment adopted as standard across Europe.

“I was very dubious about the trial to begin with but I’m so glad my dad persuaded me to go for it,” said Francesca. “I had no sickness from the chemotherapy so it’s great to hear that the treatment is now being offered as standard. That’s why I feel so passionately about supporting research. Without improvements like this I wouldn’t be here now.

“It’s such a deadly cancer and it is so aggressive, there isn’t the biggest window of opportunity for treatment. It affects a lot of young adults and children who can lose limbs if it’s found in an arm or a leg, but treatment can be successful if it’s caught early enough.”

BHP members join new Mission for mental health research

Written by Louise Stanley on . Posted in Clinical trials, Early diagnosis and detection, Mental health.

BHP members the University of Birmingham, Birmingham Women’s and Children’s NHS Foundation Trust and Birmingham & Solihull Mental Health NHS Foundation Trust, are part of the Government’s new Mental Health Mission – designed to develop radical new treatments for mental health conditions.

The £42.7M investment into research aims to improve the speed and accuracy of diagnosis and increase the use of new technologies, as well as novel and targeted treatment approaches for those with mental illnesses – including young people at Forward Thinking Birmingham (FTB), the city’s unique 0-25s Youth Mental Health Service.

The Mission will be delivered through the National Institute for Health and Care Research (NIHR) Mental Health Translational Research Collaboration, a UK wide network of leading investigators specialising in mental health research.

In Birmingham, £9.9 million in funding will enable the establishment of the Midlands Translational Research Centre of Excellence, co-led by the Universityof Birmingham with Birmingham Women’s and Children’s Hospitals and Birmingham & Solihull Mental Health NHS Foundation Trust, with reach across the Midlands through the five years’ funding.

Research will focus on increasing recruitment to new studies to test and validate treatments in early psychosis, depression and children and young people. We will work with people with lived experience to comprehensively understand the best way to test novel treatments. There are also plans to train and support a network of new researchers, partners, NHS staff and young people in the Midlands.

Professor Rachel Upthegrove, Professor of Psychiatry and Youth Mental Health at the University of Birmingham and Mental Health Research and Development Lead at Birmingham Women’s and Children’s NHS Foundation Trust, said: “We’re delighted that the Government is making such a significant investment in mental health research. This funding will allow us to increase and lead large-scale early intervention trials aimed at delaying or preventing the onset of severe mental illness, and its impact, with evidence-based treatments and support.

“The Centre will put research where we need it most, focusing on young, superdiverse, and deprived populations, which may be unfairly missing out on access to research.”

Teams at the Research Centre will work with individuals with lived experience to understand the best way to test treatments. There are also plans to train and support a network of new researchers, partners, NHS staff and young people in the Midlands.  

Building on the priority healthcare missions launched in November 2022 as part of the Life Sciences Vision, the Mental Health Mission will promote collaboration across different sectors to bolster research and attract further investment from industry and research organisations.

Nationally, the Mental Health Mission will be chaired by Kathryn Abel and Husseini Manji. In a joint statement, they said: “We are delighted to be working together to make the new Mental Health Mission a truly revolutionary force behind mental health research. We want the Mission to create tangible differences to the lives of patients, both in the UK and internationally. Between us, we bring a wealth of experience in mental health research and innovation, and a commitment to genuine collaboration with patients, industry and healthcare staff.

“Bringing together the public sector, patients and industry as equal partners, the Mission will work with the Office for Life Sciences and the National Institute for Health and Care Research (NIHR) to support the NHS and NIHR to capitalise on its size and scope, and on the depth of its data resources. Alongside additional investment in mental health research and infrastructure, the Mission will foster a step change in the way we think about mental health, mental illness and its treatment. This will support development of the critically needed treatments across the spectrum of mental illness.

“We want the UK to be the most attractive place to conduct robust, high impact mental health research, ensuring people have access to the best, and newest, treatments. We are confident that the Mission will be unique in its ability to convene and challenge national partners to make this happen.”

 

Cannabinoid-based drug trial for brain tumours launches in Birmingham

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

A major UK clinical trial of an oral spray containing cannabinoids to treat recurrent glioblastoma has opened in the UK. Funded by The Brain Tumour Charity and coordinated by the Cancer Research UK Clinical Trials Unit at BHP founder-member the University of Birmingham, the three-year phase II trial  will investigate whether combining nabiximols and chemotherapy can help extend the lives of people diagnosed with recurrent glioblastoma.

Anyone interested in this study, which is called ARISTOCRAT, should speak to their medical team first to ensure they are eligible to participate.

It will recruit more than 230 glioblastoma patients at 14 NHS hospitals across England, Scotland and Wales in 2023 including Birmingham, Bristol, Cambridge, Cardiff, Edinburgh, Glasgow, London, Liverpool (Wirral), Manchester, Nottingham, Oxford and Southampton.

Professor Pamela Kearns, Director of the Cancer Research UK Clinical Trials Unit (CRCTU) at the University of Birmingham, which is co-ordinating the trial, said:

“ARISTOCRAT represents a significant step in our journey towards finding safe and effective treatments for the most aggressive brain tumours. By testing innovative combinations of drugs we hope to improve the outcome for this challenging disease.

“We’re immensely proud to be able to bring this trial to patients with the support of the Brain Tumour Charity and thanks to the generosity of all those who gave to the crowdfunding campaign.”

Glioblastoma is the most aggressive form of brain cancer with an average survival of less than 10 months after recurrence.

In 2021, a phase I clinical trial in 27 patients found that nabiximols could be tolerated by patients in combination with chemotherapy, and has the potential to extend the lives of those with recurrent glioblastoma.

Should the trial prove successful, experts hope that nabiximols could represent a new, promising addition to NHS treatment for glioblastoma patients since temozolomide chemotherapy in 2007.

In August 2021, a fundraising appeal by The Brain Tumour Charity, backed by Olympic champion Tom Daley, raised the £450,000 needed for this phase II trial in just three months, and Jazz Pharmaceuticals has generously agreed to provide nabiximols and matched placebo free-of-charge to patients on the ARISTOCRAT trial.

Participants will self-administer nabiximols or a placebo spray and will undergo regular follow-ups with the clinical trial team, including blood tests and MRI scans. This will also be one of the first trials to integrate with The Brain Tumour Charity’s app BRIAN.

Principal Investigator, Professor Susan Short, Professor of Clinical Oncology and Neuro-Oncology at the University of Leeds, said:

“We are very excited to open this trial here in Leeds and very much look forward to running the study which will tell us whether cannabinoid- based drugs could help treat the most aggressive form of brain tumour.

“The treatment of glioblastomas is extremely challenging. Even with surgery, radiotherapy and chemotherapy, nearly all of these brain tumours re-grow within a year, and unfortunately there are very few options for patients once this occurs.

“Cannabinoid-based drugs have well-described effects in the brain and there has been a lot of interest in their use across different cancers for a long time now. Glioblastomas have receptors to cannabinoids on their cell surface, and laboratory studies on glioblastoma cells have shown these drugs may slow tumour growth and work particularly well when used with temozolomide.

“We now have the opportunity to take these laboratory results, and those from the phase I trial and investigate whether this drug could help glioblastoma patients live longer in this first-of-a-kind randomised clinical trial.”

How can I take part in the trial?

Your treating oncologist will be aware of the study if it is open in your hospital or can refer you to a treating centre if necessary. Please speak to your treatment team about eligibility for the trial.

For more information visit the ARISTOCRAT web page on the Cancer Research UK Clinical Trials Unit website.