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Author: Louise Stanley

New research will target earliest stages of bone marrow cancer

A new programme, funded by Cancer Research UK and led by BHP founder-member the University of Birmingham, has set out to attempt to eradicate a major and incurable bone marrow cancer.

An interdisciplinary team of researchers will investigate the underpinning biology of the very early stages myeloma, a cancer that develops from plasma cells – a type of white blood cell – made in the bone marrow. This is game changing research because the early stage, called MGUS, is common and only an unpredictable minority of cases go on to develop the killer disease of myeloma.

MGUS patients have non-cancerous expansion of immune cells in their bone marrow. In most patients this will be stable for many years and will never cause significant harm. The problem is that, in some patients, the cells will become more aggressive and cause full-blown cancer.

For decades it has not been possible to sufficiently tell these two types of patients apart. Until now this challenge has been considered insurmountable resulting in all cases of myeloma being diagnosed very late. However, thanks to the funding by Cancer Research UK it is time to make a change. Using biological and bioinformatic research the team aim to devise ways of accurately predicting which patients with MGUS are truly at risk of developing life threatening myeloma and those that will not.

With the help of experts in the economic challenges faced by health providers such as the NHS the team will then will design the best possible screening strategies to detect the disease and the quickest possible route to clinical trials of ways to prevent myeloma occurring.

Lead researcher Chris Bunce is Professor of Translational Cancer Biology, in the School of Biosciences at the University of Birmingham. He said: “Despite decades of academic and pharmaceutical company research, costing eye watering amounts of money and human endeavour, a cure for myeloma remains elusive.

“All myeloma cases, however, arise from an easily diagnosed pre-condition that remains virtually ignored by researchers. This funding from Cancer Research UK reverses that stance and turns the focus onto the very early stages of myeloma with a view to stopping the disease in its tracks.”

The research aims to provide a deeper understanding of how specific chemicals in the blood change as patients transition from MGUS towards developing myeloma. These changes – first identified by researchers at the University of Birmingham – could help distinguish ‘high risk’ MGUS from ‘low risk’ MGUS, as well as helping to identify potential drug targets for treatment.

Mark Drayson, Professor of Clinical Immunodiagnostics in the University’s Institute of Immunology and Immunotherapy, and co-lead research for the project, said: “By bringing together different approaches and expertise we aim to overcome the existing barriers to developing an effective test for stratifying risk of progression from MGUS to myeloma that is recognised as both affordable and effective.”

Dr Marianne Baker, Science Engagement Manager at Cancer Research UK, said: “To beat cancers that are often diagnosed late, like myeloma, we need to understand the earliest stages of its development – the underlying biology of the disease. This is a historically underfunded area, so we’re excited to see what the project brings; the more our researchers discover, the less room cancer has to manoeuvre. It’s vital we translate results into innovations, like tests that can predict risk, and give treatment the best chance of success.”

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Improving screening in underrepresented groups for genetic condition with heart complications

Researchers from across BHP are collaborating with community groups and Amicus Therapeutics to improve screening and diagnosis of Fabry disease, a rare and inherited condition where complications affect the heart muscle, and can lead to premature death.

Through the project, the team – from University Hospitals Birmingham NHS Foundation Trust and the University of Birmingham – has begun to build advisory groups in collaboration with community leaders, healthcare professionals and patients from black, South Asian and other minority ethnic groups. These advisory groups will support researchers to understand patient perspectives on possible barriers to diagnosis, as well as possible approaches to breaking down these barriers. Once further funding is secured, these approaches will be trialled locally in Birmingham, a super-diverse city.

The incidence rate of Fabry disease in the general population is reportedly around 1 in 100,000, although this is likely an underestimate of its true prevalence. Although Fabry disease is very rare, as a genetic condition, patient’s family members have a higher likelihood of the disease that the wider population. Therefore, family screening and mapping is an important tool to identify more cases.

Identifying Fabry disease is important, since the earlier that a patient is diagnosed the better the prognosis for treatment. There are several therapies available, including enzyme replacement and methods to make the faulty enzyme that causes Fabry disease work better, all of which are more effective if started earlier. Without treatment, patients may develop significant cardiac, renal and cerebrovascular complications.

Project lead Richard Steeds, Honorary Professor of Cardiovascular Medicine and Deputy Director of Clinical Research within the Institute of Cardiovascular Sciences at the University of Birmingham and Consultant in Cardiovascular Imaging at University Hospitals Birmingham, explained: “Previous work has shown that Black, South Asian, and other minority ethnic groups, as well as patients from lower socioeconomic groups, are underrepresented in our clinic in Birmingham, despite it being a very diverse city. This knowledge helped us to identify a need for further research to understand and overcome specific barriers to family screening that are more prevalent within minority ethnic groups. If we can address these barriers, then we can support more people to benefit from beginning treatment for this rare condition sooner. Working with members of different community groups is essential to making this work.”

It is well established that minority ethnic groups face health inequalities from language barriers, lower health literacy and cultural difference that contribute to stigma and fear of discrimination that can impact on help seeking behaviours. These inequalities can lead to poorer health outcomes, so addressing them is vital.

Fabry disease can often take many years and several doctors’ appointments to diagnose because many of the symptoms are also attributed to more common causes. Family screening has the potential to help identify more cases sooner.

Some of this work has been funded through a collaboration project with Amicus Therapeutics.

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

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

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.”

Aston University launches SPARK The Midlands to support health tech innovation

Aston University, a member of BHP, has launched SPARK The Midlands, a network which aims to bridge the gap between medical research discoveries of novel therapeutics, medical devices and diagnostics, and real-world clinical use.

SPARK The Midlands is the first UK branch of Stanford University’s prestigious global SPARK programme. It comes as a result of Aston University’s active involvement in the delivery of the West Midlands Health Tech Innovation Accelerator (WMHTIA) – a government-funded project aimed at helping companies drive their innovations towards market success.

The SPARK scheme helps to provide mentorship and forge networks between researchers, those with technical and specialist knowledge and potential sources of funding. SPARK members have access to workshops led by industry experts, covering topics such as medical device regulations, establishing good clinical trials, and creating an enticing target product profile to engage future funders.

While SPARK The Midlands will be based at Aston University, it will support multiple projects across the West Midlands from other BHP members including the University of Birmingham (UoB), Birmingham Women’s and Children’s Hospitals NHS Foundation Trust (BWC), as well as other regional institutions including Warwick University. It is also supporting a number of regional companies.

12 projects have been funded as part of cohort one, including a cross-BHP initiative involving Aston, UoB and BWC. Led by Dr Jan Novak from Aston’s School of Psychology,  the project will develop a device to categorise paediatric brain tumours using artificial intelligence (AI) and machine learning. Based on 24 years of data, the device will be able to help determine what type of brain tumour a patient has and what surgical approach to take.

Aston University researchers are involved in a further four projects within cohort one of SPARK The Midlands.

Luke Southan, SPARK UK director, said: “It is a privilege to be launching the first UK SPARK cohort here in the West Midlands. We have a real opportunity here to support not only new, transformative, standards of care for areas of genuine unmet clinical need, but also to support the growth of a major innovation cluster for the region.”

Pasi Sorvisto, founder and director of SPARK Finland and director of SPARK Europe, said: “We are truly excited to have a SPARK programme in the UK. SPARK The Midlands is a perfect example of what coming together enables in the region.

“The beauty of our European network is that we have come together in supporting scientists, students and healthcare professionals in developing great new solutions and businesses. We share experiences of what works and what doesn’t, we provide joint webinars and mentoring, we have joint events, and we support each other in building new solutions and businesses to global markets. “Together” is maybe the most common word in discussions at European SPARK network. Since we have a global network of SPARK programmes associated with top-tier institutions, each and every project in Europe has opportunities to build high value connections through our global network.”

SPARK The Midlands will host its first educational workshop on Tuesday 13 February at 15:00 GMT. It will be a hybrid event, hosted at Aston University’s John Cadbury House, and online. It is open to anyone interested in developing a therapeutic, medical device or diagnostic for public use, including academics, researchers, scientists, healthcare professionals, and postgraduate students. For more information and tickets visit the Eventbrite page.

For more information about SPARK The Midlands, visit https://www.sparkthemidlands.co.uk/

New treatment combination may improve outcomes for children with rare cancers

Children who develop neuroblastomas, a rare form of cancer which develops in nerve cells, may benefit from receiving certain anti-tumour drugs as well as chemotherapy, a new trial has found.

The results of the BEACON trial conducted by the Cancer Research UK Clinical Trials Unit (CRCTU) at BHP founder-member the University of Birmingham found that combining anti-angiogenic drugs, which block tumours from forming blood vessels, alongside various chemotherapy drugs led to more young people seeing their tumours shrinking, from 18% in the control group to 26% among those on Bevacizumab.

The findings have been published in the Journal of Clinical Oncology. The trial saw 160 young people aged 1-21, from 43 hospitals in 11 European countries, randomised with half receiving the anti-angiogenic drug called Bevacizumab on top of conventional therapy. The group who received Bevacizumab had an increase in the likelihood of responding to treatment, from 18% among those who only had the established therapy to 26% for those with the additional drug. Patients who received Bevacizumab additionally had better one year progression-free survival rates.

The trial constituted one of many collaborations between the University of Birmingham and European expert groups SIOPEN (International Society of Paediatric Oncology European Neuroblastoma) and ITCC (Innovative therapies for children with cancer).

Simon Gates, Professor of Biostatistics and Clinical Trials at the University of Birmingham and senior lead author of the paper said: “These are very exciting results that hopefully get us closer to finding treatments for children who develop neuroblastomas. Currently, the outcomes are really poor for children who get this horrible cancer and so even seemingly small increases in the chance that a patient is going to be able to shrink their tumours is significant.

“We are delighted that the BEACON trial has helped to shape treatment for children with relapsed and refractory neuroblastoma going forward.”

Dr Lucas Moreno, Head of Paediatric Haematology and Oncology at Vall d’Hebron University Hospital, Barcelona, Spain and Chief Investigator for the study said: “BEACON was a hypothesis-generating trial that has served to identify active regimens that are now being further investigated. We are delighted that the data generated has been incorporated into the current UK Clinical Practice Guidelines and Bevacizumab is incorporated into standard treatment for relapsed neuroblastoma.”