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Promising new steroid drug could offer safer arthritis treatment

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

Early research has found that a new steroid drug, used for treating Duchenne Muscular dystrophy, has shown significant promise in treating inflammatory diseases such as rheumatoid arthritis.

Research published in Rheumatology revealed that Vamorolone, a glucocorticoid, showed to be just as effective as standard glucocorticoids to treat inflammation but with minimised negative side effects.

Glucocorticoids are some of the most widely used drugs to treat patients with a diverse range of inflammatory diseases. Unfortunately, whilst they effectively reduce inflammation and pain, they can also cause severe side effects. These include muscle and bone loss that can increase the risks of falls and fractures.

Vamorolone is a unique metabolism-resistant steroid approved by the FDA which appears to provide significant anti-inflammatory benefits while causing fewer harmful effects on muscle and bone.

The study, funded by the Foundation to Eradicate Duchenne and utilising mouse models with chronic rheumatoid arthritis, shows Vamorolone could be a promising alternative treatment for patients living with the disease.

Dr Rowan Hardy, Associate Professor in Steroid Metabolism and Signalling at BHP founder-member the University of Birmingham, said: “If Vamorolone is effective in patients with rheumatoid arthritis, it would allow us to better control disease activity, whilst preserving muscle and bone to reduce the risks of fractures and falls.”

The study team have now secured further funding with the Foundation to Eradicate Duchenne that will allow the research team to better understand the processes whereby Vamorolone is able to protect muscle and bone in patients with inflammatory disease. 

Through their involvement with the Birmingham Rheumatology group under Professor Adam Croft, and the NIHR Birmingham Biomedical Research Centre’s Inflammatory Arthritis Theme, the team will now work with clinicians to examine the possibility for new clinical trials to examine Vamorolone in rheumatoid arthritis patients. 

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Birmingham teenager first in UK to receive groundbreaking diabetes treatment

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

A young patient identified by the ELSA study as having early stages of Type 1 Diabetes, has received treatment to delay the condition.

Sam, aged 14 and from Kings Norton, was the first to receive the new drug, Teplizumab, at the Clinical Research Facility at Birmingham Children’s Hospital.

Sam’s dad, Chris, has Type 1 diabetes and knowing that family members are more likely to develop the disease, Sam was screened for early stages of the condition and learnt he would develop Type 1 Diabetes. However, there was good news for the family when Sam was offered Teplizumab, a new treatment to delay the onset of the chronic illness. Past trials have proven that Teplizumab delays insulin-dependent diabetes for up to three years.

Sam’s mum, Louise, explained: “I know from Sam’s dad just how stressful life with Type 1 Diabetes is, having to constantly monitor your blood sugar and carry insulin with you at all times. We’re so happy that Sam doesn’t have to worry about his blood sugar yet, especially while he is doing his GCSEs. He can just be a normal teenager.”

Teplizumab is prescribed on a case-by-case basis for children who have been recently diagnosed with type 1 diabetes and are at early stages.

Dr Renuka Dias, a researcher from University of Birmingham’s Department of Applied Health Sciences and Consultant Paediatric Endocrinologist working at Birmingham Women’s and Children’s Hospital, said: “Being able to delay insulin-dependent diabetes will have a huge impact on a child’s life. It means we are letting children have a normal childhood for much longer.”

Dr Dias and her specialist team at the Clinical Research Centre who were involved in Sam’s care have also been integral to a first-of-its-kind study, led by the University of Birmingham, to screen children aged 3 to 13 to find out their risk of developing type 1 diabetes. The study aims to allow treatment to begin sooner and, as in Sam’s case, delay the start of the condition.

The ELSA study has screened over 20,000 children in the UK for Type 1 Diabetes in the last two years. The study is now scaling up throughout Europe.

Unfortunately, it often takes a child becoming seriously unwell for a diagnosis of Type1 Diabetes to be made. Through the study, families identified with a child who is at-risk can begin educating themselves about the condition and learning about the options for management before they find themselves in that crisis situation.

Support and education has been made available to study participants. In addition, some children, like Sam, will be eligible to explore treatment options that could delay the onset of the condition.

Parth Narendran, Professor of Diabetes Medicine at the University of Birmingham and lead for the Type 1 Diabetes clinical service at the Queen Elizabeth Hospital Birmingham (QEHB), said: “We hope that the ELSA study will lead to the roll-out of Type 1 Diabetes early detection programme for children in the UK and that many more children could then benefit from potential treatments to delay Type 1 Diabetes in future.”

Birmingham Women’s and Children’s Hospitals, the University of Birmingham, and University Hospitals Birmingham – which operates QEHB – are founder-members of Birmingham Health Partners. Fellow BHP members Birmingham Community Healthcare NHS Foundation Trust is an ELSA Study partner.

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Aston University and Birmingham Children’s Hospital study shows diagnosis and treatment of preschool wheeze needs improvement

Written by Louise Stanley on . Posted in Inflammation and chronic disease.

A study led by Aston University’s Dr Gemma Heath and  Dr Prasad Nagakumar from Birmingham Children’s Hospital – both BHP member organisations – has shown that treatment and diagnosis for preschool wheeze needs more effective evidence-based guidelines.

Preschool wheeze affects approximately 30–40% of children under six. The condition is characterised by episodes of wheezing or breathlessness, with younger children being particularly susceptible due to their narrower airways. Although it can resemble asthma, preschool wheeze is often triggered by viral infections or allergies and does not always mean a child will develop asthma.

The UK has Europe’s second highest prevalence of preschool wheeze in two-year-olds and is a leading cause of emergency hospital visits and hospitalisations in the country. Repeated preschool wheeze attacks are frightening for parents, and result in significant morbidity, healthcare costs and impaired quality of life for both the child and parent.

There is currently no diagnostic pathway or definitive management guidelines for preschool wheeze. The research team interviewed affected parents and carers about their experiences, and found problems with diagnosis and treatment at multiple levels.

The first major issue identified by parents was inconsistent terminologies used by doctors, and confusing and conflicting diagnoses such as asthma, suspected asthma, viral wheeze and allergy. Some reported frustration at the lack of definitive diagnosis, an apparent lack of GP knowledge, sometimes false reassurance that the wheeze was viral rather than asthma, or that the cause was a “mystery”.

A common problem was that investigative tests did not occur until after multiple hospitalisations. Blood tests for particular markers have potential to identify whether asthma or an allergy is likely to have caused the wheeze, and therefore guide treatment. The parents in the study welcomed the idea of timely tests, but stressed that children should not be subjected to repeated testing.

Preschool wheeze is generally managed with steroid and salbutamol inhalers, as for asthma. While parents had concerns about the side-effects and long-term impacts of using the treatments, they deemed the medication “an acceptable cost”.

Parents reported being “terrified” while watching attacks of preschool wheeze, and significant psychological impacts when their child was admitted to hospital. Some had missed work or even given up work to care for their child, with high levels of anxiety, while others said they felt unable to go on holiday overseas due to concerns about healthcare access in the case of a wheeze attack.

Most parents preferred to access care at hospital rather that at doctors’ surgeries due to the perception of a lack of training for GPs and a lack of confidence. However, accessing necessary care can be difficult, including due to childcare difficulties, the cost of hospital parking and a lack of available ambulances.

The research team said that parents’ views highlight the problems and called for clinical trials to determine the efficacy of treatment decisions made according to the results of investigations.

Dr Heath said: “This research demonstrates an urgent need for preschool wheeze management policies and treatment pathways that are evidence-based and co-developed with parents. We have shown that use of investigations such as blood or allergy tests would be acceptable to parents, if they were shown to be helpful in guiding more effective and timely treatments.”

Dr Nagakumar said: “Preschool wheeze has significant impact on young children’s and their parents’ lives. Our research, involving parents with lived experience, will inform future studies to improve the care and reduce the impact of preschool wheeze on the already-stretched emergency health services in the UK.”

Archives of Disease in Childhood doi: 10.1136/archdischild-2024-327781

£3.4m boost for research into paediatric autoimmune brain inflammation

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

Dr Sukhvir Wright at Aston University‘s Institute for Health and Neurodevelopment (IHN) and Honorary Consultant Neurologist at Birmingham Children’s Hospital (BCH) – both BHP member organisations – has been awarded a £3.4m Career Development Award from Wellcome to research paediatric autoimmune encephalitis (AE), an inflammatory brain condition.

Every minute, someone in the world is diagnosed with encephalitis, which can be caused by an infection or have an autoimmune cause, where the body’s own immune system starts attacking the brain. The expert neuro-immunology team at BCH cares for children with autoimmune encephalitis all year round.

AE accounts for around a third of cases worldwide, with patients experiencing seizures, cognitive and sleep dysfunction and movement disorders. Although medical professionals are getting better at recognising and treating AE earlier, the long-term outcomes remain frustratingly poor, particularly in children under five.

Some symptoms of the disease, such as seizures, can resolve but others, such as problems with learning and memory, behavioural change and sleep disorders, can become chronic. Why some of these symptoms get better and others persist is not well understood. 

Dr Wright carried out a world-first preliminary study in a group of children with AE at least 18 months after they first developed the condition, using magnetoencephalography (MEG) brain scans. She found distinct long-term brain structure and network changes and believes that these brain changes are responsible for the chronic symptoms of the disease.

During her Career Development Award, Dr Wright will use laboratory models to characterise the mechanisms causing the chronic symptoms, examining the underlying changes from single brain cells to whole brain networks. She will also examine longitudinal brain network changes in children immediately following the acute attack of AE and for up to eight years afterwards using a new optically pumped magnetometer (OPM) MEG scanner.

IHN is an ideal location for the research project, as it houses the UK’s only paediatric clinical and research Wellcome Trust MEG laboratory. The MAG4Health OPM MEG scanner that will be used by Dr Wright was installed in 2024 following a Medical Research Council (MRC) equipment grant for £800,000 led by Aston University’s Dr Caroline Witton in partnership with BCH. The Aston-BCH OPM MEG uses an adjustable cap with sensors which is placed on the patient’s head, which allows some degree of movement and is therefore more acceptable for children.

Combining the data from the laboratory models and human patients will enable Dr Wright and her research team to identify commom pathophysiological targets, mechanisms and predictive biomarkers to reduce the adverse effects of AE and improve long-term outcomes.

Dr Wright is part of the expert neuroimmunology team at BCH, led by Professor Evangeline Wassmer, Paediatric Neurology Consultant. The AE research project will involve Professor Wassmer’s team, the BCH Psychology department led by Jo Horton, Professor Stefano Seri (neurophysiology) and Dr Laavanya Damodaran (liaison psychiatry).

Children and families with lived experience of AE will be directly involved with all aspects of the research to ensure it is answering questions that matter to them, including the family of one of the first AE patients ever treated by the neurology team at BCH. This patient and family involvement will be facilitated by the Epilepsy Research Institute’s Shape Network and Encephalitis International, two charities with which Dr Wright has strong links.

Dr Wright said: “We hope that this project will transform outcomes and optimise brain health in paediatric autoimmune encephalitis and beyond by delivering a significant shift in understanding the acute and long-term effects that autoimmune encephalitis has on children and young people.”

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Fitness app could transform care of rheumatoid arthritis patients

Written by Louise Stanley on . Posted in Clinical trials, Inflammation and chronic disease.

Researchers at BHP members Sandwell and West Birmingham NHS Trust and the University of Birmingham aim to transform the care of rheumatoid arthritis (RA) patients by developing an app which links to the Fitbit – a popular activity tracker.

The rheumatology medical team and researchers are leading the groundbreaking clinical trial to create the app which they hope will ease the pain of patients suffering from the crippling disease and provide extra support.

It’s called MISSION-RA which stands for MovIng to Support Sustained Improvement of Outcomes iN Rheumatoid Arthritis.

Sally Fenton, Chief Investigator for the study, said: “The app will be specifically designed for and by people with RA.

“This will be done in two ways. Firstly, a series of interviews, co-design studies and workshops will be carried out to develop the app. Then, artificial intelligence will be used within the app to provide personalised support for people living with RA, based on symptoms such as pain, fatigue and mobility. It is expected the app will be available to download for free so it can be used on a Fitbit in the future.”

RA leads to inflammation of the joints and surrounding tissues which causes flare ups – this is when symptoms become worse. One of the ways to help ease these episodes of pain can be through exercise, physiotherapy and occupational therapy.

Dr Sangeetha Baskar, Clinical Specialty Lead for Rheumatology and Principal Investigator for MISSION-RA explained: “Rheumatoid arthritis is an extremely painful, tiring, psychological and physically debilitating disease. Patients face countless challenges while doing basic daily activities such as dressing, cooking and walking and often become dependent on family members to perform some of their daily tasks. Through this innovative study and the mobile app, we are hoping to help patients increase their physical activity tailored for them and improve their quality of life.”

The first patient from the Trust has already been recruited into the study.

Ana Duarte, Clinical Research Practitioner (CRP) and also Associate Principal Investigator, said: “Overall, the MISSION-RA study has overcome our initial expectations. Patients are very keen and have enjoyed their experience so far. This has also been a great opportunity for the study staff which has led to progression in their careers. Most importantly, this we hope will place SWB’s R&D department and rheumatology team as international leaders in healthcare AI and machine learning.”

MISSION-RA is being delivered in partnership with the National Institute for Health and Care Research (NIHR) and National Rheumatoid Arthritis Society (NRAS).

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Patients with recent onset diabetes fast-tracked more effectively for pancreatic cancer screening

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

A recent study, funded by Pancreatic Cancer UK and conducted by researchers at BHP’s University of Birmingham, in collaboration with University of Oxford, University of Nottingham and fellow BHP founder-members University Hospitals Birmingham NHS Foundation Trust, developed a new prediction model as an effective way of identifying individuals suitable for fast-track abdominal imaging.

Weight loss and glycaemic control are known biomarkers that can indicate pancreatic cancer risk and in, accordance with NICE recommendations, people over the age of 60 years with recent onset diabetes and weight loss currently undergo urgent abdominal CT imaging to assess for pancreatic cancer.

Pancreatic cancer is known for its poor prognosis, with less than a quarter of patients surviving past one year after diagnosis. Early detection is important as patients with early-stage disease are more likely to be able to tolerate chemotherapy and therefore have an improved 5-year survival rate, but most patients are not diagnosed until the later stages of the disease. One way of detecting pancreatic cancer patients sooner is through screening patients with diabetes as there is a known association.

By looking at further potential biomarkers to determine which patients would benefit from referral for abdominal imaging, there is a chance of picking more cancers and reducing the cost of imaging those who are not so high-risk.

Dr Shivan Sivakumar, Associate Professor in oncology, specialising in pancreatic, liver and biliary tract cancer, said: “One in ten pancreatic cancer patients have new-onset diabetes and we know that some patients with newly diagnosed diabetes are worth exploring further to improve early detection of pancreatic cancer. We need to more accurately predict which of those patients should be referred for further investigation. We used health data records, from a larger patient population than has previously been studied, to develop a more nuanced method of stratification that could improve referral pathways.”

This study used large-scale, population-representative, linked electronic health data records to develop and evaluate a new prediction model that can be used to predict risk of developing pancreatic cancer within two years of a diabetes diagnosis. The new models used a variety of potential markers and were able to predict pancreatic cancer risk in patients aged between 30 and 85 years, rather than relying on the 60+ rule of thumb.

This study was the largest of its kind and offers improved accuracy compared to previous prediction models as it used a larger data set. The new prediction model could be more effective than current ‘rules-based’ referral guidelines. Further external validation and health economic assessment is recommended.

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