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Author: Louise Stanley

Treatment hope for patients with rare disorder following clinical trial

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

Patients with a rare hereditary disorder may soon benefit from a new treatment which has undergone a promising trial at Queen Elizabeth Hospital Birmingham (QEHB) – part of BHP founder-members University Hospitals Birmingham NHS Foundation Trust.

The experimental drug, called mRNA-3927, has been tested on patients for the first time as part of a study into propionic acidaemia – a serious metabolic disorder which means the body is unable to process certain parts of proteins and fats properly. This can lead to a build-up of harmful substances in the body and, without appropriate treatment, can be fatal.

Patients with this condition must follow a specific diet, including a low protein intake and specific food for life. Symptoms include: vomiting, lethargy, dehydration, and acid build up in the body. Liver and kidney transplant is a surgical option that can help reduce the frequency of acute metabolic episodes.

QEHB is the only adult centre in the world running this study whose initial findings have just been published.

Prof. Tarekegn Hiwot, Consultant in Inherited Metabolic Disorders at QEHB and Honorary Professor in the Institute of Metabolism and Systems Research at fellow BHP founder the University of Birmingham, led the trial and recruited patients for the study. He said: “We conducted a study of mRNA-3927 with 16 participants to find the safety, tolerability, and optimal dose. Our interim analysis has shown significant reduction of 70% in preventing severe metabolic crisis. The treatment was safe and well tolerated.

“In summary, this study explores a promising, first of its kind treatment for propionic acidaemia using mRNA-3927, aiming to improve patients’ health and reduce dangerous metabolic events.

“This study may also serve as a proof of concept in using mRNA treatment for other life limiting single gene genetic conditions in general.”

The interim results of the study were published in Nature.

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Center of Excellence Award Renewal for Allergy, Asthma and Immunology Center

Written by Louise Stanley on . Posted in Birmingham Health Partners, Inflammation and chronic disease.

The Allergy, Asthma, and Clinical Immunology Center in Birmingham has been successful in renewing its Center of Excellence status for another four-year term – re-affirming the Center’s international recognition for multidisciplinary scientific and clinical innovation – following a successful bid led by Professor Mamidipudi Thirumala Krishna, representing Birmingham Health Partners.

Renowned for its innovative services to accelerate education and research, the Institute of Immunology and Immunotherapy, and the School of Pharmacy at the University of Birmingham in collaboration with the West Midlands Allergy and Immunology service at University Hospitals Birmingham (UHB) NHS Foundation Trust has once again been designated as a World Allergy Organisation (WAO) Center of Excellence.

There are currently 82 Centers worldwide, and only three other similar Centers of Excellence in the UK.

According to the World Allergy Organisation (WAO), their Centers of Excellence accelerate multidisciplinary science and clinical innovation, training, and advocacy worldwide to deliver world-class education, research, and training to stakeholders in asthma, allergy, and clinical immunology.

Professor Mamidipudi Thirumala Krishna said: “This continued recognition of our Center of Excellence signifies our commitment to delivering world-class clinical services, fostering excellence in education and research, and nurturing collaborative relationships both nationally and internationally and contributing to global health.”

Professor Ben Willcox, Director of the Institute of Immunology and Immunotherapy at the University of Birmingham, commented: “We are proud to see our Center of Excellence renewed, guided by the visionary leadership of Professor Krishna. We have over 50 years of experience in world-leading immunology research, and this Center’s status renewal is testament to that.”

Professor Anthony Cox, Head of the School of Pharmacy and Professor in Clinical Pharmacy and Drug Safety, said: “Great news that the World Allergy Organisation Center of Excellence has been extended. The School of Pharmacy is proud to be working with the center on issues related to allergy and medicines – including inappropriately identified drug allergies which have a considerable impact on patient safety and the rational use of medicines.”

Dr Richard Baretto, Clinical Service Lead for the Department of Allergy and Immunology at UHB, added: “This prestigious award from the World Allergy Organisation is testament to the efforts of all the members of the clinical teams, providing excellent care for our patients with allergic disease. It emboldens us all to strive for continued improvement in our service through regular review, research and innovation.”

Professor Adel Mansur, Lead for Birmingham Regional Severe Asthma Service, commented: “On behalf of the Birmingham Regional Severe Asthma Service I am delighted of the news of extending our status as World Allergy Organisation Center of Excellence, recognising the excellent work done in Birmingham. Over the last three years tremendous work and development have been achieved to further strengthen our Center in areas of research, education and training and to lead on clinical excellence in allergy, asthma and clinical immunology.”

“We have adopted various initiatives to improve our patients outcomes, access and equity of service delivery and provide conducive environment for our trainees to learn and develop skills and further their career.  Our national and international contribution to research and education in these fields are also important developments and targets to progress further over the next 4 years which I look forward to with excitement.”

Dr Gareth Walters, NHS consultant in occupational respiratory medicine and interstitial lung diseases in Birmingham, also added: “On behalf of the Birmingham Regional NHS Occupational Lung Disease Service , we are very pleased that our status as a World Allergy Organization Center of Excellence has been renewed for a further 4 years. This is testament to the hard work and dedication of our multi-disciplinary team, who go above and beyond to diagnose and manage a range of occupational lung diseases.”

The Allergy, Asthma and Immunology Center will continue to lead global healthcare innovation and pursue its commitment to excellence in research and education for another term commencing in 2024 to 2028. 

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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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Launch of new international medical code for presymptomatic type 1 diabetes

Written by Louise Stanley on . Posted in Early diagnosis and detection, Inflammation and chronic disease.

Researchers at BHP founder-member the University of Birmingham have partnered with NHS England to produce a diagnostic code tailored for individuals in the early phases of type 1 diabetes, enhancing patient prospects for timely healthcare and access to cutting-edge treatments.

Today marks the introduction for a new SNOMED CT code specifically for presymptomatic type 1 diabetes, which will be integrated into the standardised and multilingual set of clinical healthcare terminology. SNOMED codes are crucial in electronic health records, being used to identify a person’s underlying medical conditions. This system acts as the most precise and extensive list in clinical health terminology globally.

Type 1 diabetes progresses gradually through three stages, with the initial two stages termed presymptomatic type 1 diabetes. Individuals in this phase exhibit biological markers, or autoantibodies, indicating the onset of the immune attack that targets insulin-producing beta cells. Given the absence of symptoms, detection relies heavily on screening initiatives such as the ELSA study, a trial led by Professor Parth Narendran at the University of Birmingham,  screening children for type 1 diabetes. Screening initiatives such as these will allow for early identification.

Lauren Quinn, who co-leads the ELSA study and assisted in the development of the of the SNOMED code, commented: “The introduction of this SNOMED code facilitates clinical care and follow-up for individuals with presymptomatic type 1 diabetes. It also allows researchers to identify people who could benefit from novel therapies to delay the onset of type 1 diabetes and recruit them to clinical trials of immunotherapies.”

“This will transform type 1 diabetes research by fast-tracking recruitment, unravelling how the condition develops and progresses, and bringing us closer to licensed disease-modifying treatments in type 1 diabetes.”

Dr. David Shukla, a GP and Clinical Research Fellow involved in code development, highlighted its practical implications: “The inclusion of a code for the diagnosis of presymptomatic type 1 diabetes will highlight to healthcare professionals involved in their care the individuals who are at high risk of developing type 1 diabetes. This will help ensure that when these people progress and develop symptomatic type 1 diabetes, it will be picked up and treated at a much earlier stage.”

“This reduces the risk of them presenting or being diagnosed late and developing diabetic ketoacidosis, an emergency complication of type 1 diabetes that can be fatal. This timely pick up and initiation of prompt treatment will lead to substantial improvements in their diabetes and future care.”

Hilary Nathan, Director of Policy and Communications at JDRF UK, added: “This recognition of presymptomatic type 1 diabetes with a SNOMED code is a crucial step towards the implementation of population screening programmes for early detection of type 1 diabetes. Early detection leads to short and long-term health benefits, improved quality of life and cost savings for healthcare providers.”

“The new code will unlock better monitoring, follow-up and education for people in the earliest stages of type 1. It will also help facilitate recruitment into clinical trials of emerging treatments, enabling people developing type 1 diabetes to access therapies that have the potential to claw back valuable time free from the burdens of type 1 diabetes management.”

The code for type 1 diabetes in SNOMED is ‘Diabetes mellitus type 1 – 46635009’. The new code presymptomatic type 1 diabetes, known as ‘‘Presymptomatic diabetes mellitus type 1 – 1290118005′, has now been introduced for inclusion in individuals’ electronic health records. 

These codes are now part of the ‘Health conditions’ category in the NHS app, allowing individuals and their families to access them as well. 

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New Inflammatory Bowel Disease testing protocol could speed up diagnosis

Written by Louise Stanley on . Posted in Early diagnosis and detection, Inflammation and chronic disease.

Patients with suspected inflammatory bowel disease (IBD) could benefit from improved testing protocols that could reduce the need (and lengthy wait) for potentially unnecessary colonoscopies, a new study has found.

In a paper published in Frontline Gastroenterology, researchers from the National Institute for Health and Care Research (NIHR) Birmingham Biomedical Research Centre (BRC) tested a new protocol to improve IBD diagnosis combining clinical history with multiple home stool tests.

In the two-year study involving 767 participants, patients were triaged and had repeated faecal calprotectin (FCP) tests. The research team found that the use of serial FCP tests were able to strongly predict possible IBD as well as Crohn’s Disease and Ulcerative Colitis, and observed that a second FCP test was a strong indicator of a potential need for further investigation including colonoscopy; although the researchers observed that only 20% of patients had two samples submitted prior to referral to secondary care.

Dr Peter Rimmer from the NIHR Birmingham Biomedical Research Centre and corresponding author of the study said: “Patients who experience symptoms associated with inflammatory bowel diseases often have a long wait until getting a diagnosis, and current testing is under immense strain.

“Using a comprehensive 13-point symptom checker and multiple FCP tests, we have been able to identify much more accurately patients who had IBD and other diseases. The rollout of this protocol could reduce the time taken to get a diagnosis and start treatment for IBDs as much more of the screening and testing can be done through primary care. The sensitivity of multiple FCP tests can be used to flag those patients who urgently need referral into secondary care.”

Dr Rachel Cooney, Consultant Gastroenterologist at University Hospitals Birmingham NHS Foundation Trust, researcher at the NIHR Birmingham BRC and co-author of the study, added: “In its simplest form, this study may help improve referral triage for IBD patients.

“But as we plan new care pathways, it could open up new exciting possibilities: with the growing availability of home FCP testing, these tests’ results combined with simple symptom questionnaires could feed into algorithms that allow patients to self-refer to secondary care services, reducing strain on primary care.

“This is something we’re going to explore in a large follow-up study we’re currently initiating.”

The NIHR Birmingham Biomedical Research Centre is hosted by BHP founder-member University Hospitals Birmingham NHS Foundation Trust in partnership with fellow BHP founder-member the University of Birmingham. Its roster of partner organisations also includes BHP members Birmingham Women’s and Children’s NHS Foundation Trust; Sandwell and West Birmingham NHS Trust; and Aston University. 

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