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Birmingham teenager first in UK to receive groundbreaking diabetes treatment

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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New AI tools set to advance early cancer detection and prevention

Birmingham researchers are to play a role in Cancer Research UK’s recently announced £10 million AI detection programme, jointly supported by the National Institute for Health and Care Research (NIHR) and the Engineering and Physical Sciences Research Council (EPSRC) and involving 18 institutions including BHP founder-member the University of Birmingham.

Over the next five years, the Cancer Data-Driven Detection programme will harness vast quantities of data, link datasets and develop new tools to predict cancer risk – ultimately increasing the number of people diagnosed with cancer at its earliest stages.

The programme aims to access and link data from different sources – including health records, genomics, family history, demographics, and behavioural data – to develop advanced statistical models that help scientists accurately predict who is most likely to get cancer. Alongside this, the programme will develop powerful new tools which use AI to analyse the data and calculate an individual’s risk of cancer throughout their lifetime.

Researchers from University of Birmingham will take on specific roles in the programme, alongside approximately 40 others working together collaboratively. Professor Sudha Sundar, gynaecological cancer surgeon and a clinical academic in the University’s Department of Cancer and Genomic Sciences, is advising as a clinical practitioner in the multi-cancer risk prediction area of the work. Dr Ameeta Retzer from the Centre for Evidence and Implementation Science will lead on the cross-cutting Equity, Diversity and Inclusion theme, drawing on her expertise in health inequalities and research equity.

Dr Ameeta Retzer said: “Across the whole programme we will work to embed equality, diversity and inclusion since we know that cancer doesn’t affect everyone equally. It is vital that we ensure our research will benefit everyone, across all communities, equitably and that’s why I look forward to ensuring this strand of work has prominence in all areas of the programme.”

Over the next five years, the funding will build the infrastructure required to access and link these datasets, train new data scientists, create the algorithms behind the risk models and evaluate the algorithms and AI tools to ensure that they are giving accurate and clinically useful information about cancer risk.

The models generated from this research could be used to help people at higher risk of cancer in different ways. For example, the NHS could offer more frequent cancer screening sessions or screening at a younger age to those at higher risk, whilst those at lower risk could be spared unnecessary tests. People identified as higher risk could also be sent for cancer testing faster when they go to their GP with possible cancer signs or symptoms. Individuals at higher risk could also access different ways to prevent cancer.

Professor Sudha Sundar, whose clinical practice is based at BHP member Sandwell and West Birmingham NHS Trust, commented: “With cancer cases on the rise, it is essential that we work to identify and diagnose cancers earlier so that patients can begin treatments soon, which in most cancers vastly improves their quality of life and chances of survival. Screening is one way of identifying cancer sooners. Multi-cancer earlier detection tests represent an exciting progression in the scope of cancer screening programmes and this is part of the Cancer Data-Driven Detection programme that is exciting to explore further.”

The scientific programme will be guided by partnerships with cancer patients, the public, clinical experts and industry, while addressing ethical and legal considerations to ensure that the models and tools work well in practice.

Professor Antonis Antoniou, Director of the Cancer Data Driven Detection programme and Professor of Cancer Risk Prediction at the University of Cambridge, said: “Finding people at the highest risk of developing cancer, including those with vague symptoms, is a major challenge. The UK’s strengths in population-scale data resources, combined with advanced analytical tools like AI, offer tremendous opportunities to link disparate datasets and uncover clues that could lead to earlier detection, diagnosis, and prevention of more cancers.

“The Cancer Data Driven Detection programme will build the partnerships and infrastructure needed to make data-driven cancer early detection, diagnosis and prevention a routine part of frontline healthcare. Ultimately, it could inform public health policy and empower individuals and their healthcare providers to make shared decisions. By understanding individual cancer risks, people can take proactive steps to stop cancer before it gets worse or even begins in the first place.”

Earlier diagnosis of cancer saves lives. Yet according to analysis of NHS figures by Cancer Research UK, only 54.4% of cancers in England are diagnosed at stages one and two, where treatment is more likely to be successful. NHS England has set a target to diagnose 75% of cancers at stages one and two by 2028, and this will only be achieved with research and embracing new technologies to catch cancer earlier.

Professor Lucy Chappell, Chief Scientific Adviser at the Department of Health and Social Care (DHSC) and Chief Executive Officer of the NIHR, commented: “Detecting and diagnosing cancer earlier is key to improved survival and quality of life for patients. By leveraging AI to enable healthcare professionals to identify people at a greater risk of cancer, this initiative could improve the way patients are screened and diagnosed. This programme’s AI-driven insights could lead to more effective treatment and improved survival, helping patients to live longer, healthier lives.”

The Cancer Data Driven Detection programme is jointly supported by Cancer Research UK, the National Institute for Health & Care Research, the Engineering & Physical Sciences Research Council, Health Data Research UK, and Administrative Data Research UK.

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Most accurate ultrasound test could detect 96% of ovarian cancers

An ultrasound test that detected 96% of ovarian cancers in postmenopausal women should replace the current standard of care test in the UK, according to a new study by BHP members.

In a paper published in Lancet Oncology, researchers funded by the NIHR and led by Professor Sudha Sundar conducted a head-to-head comparison of all currently-available tests to diagnose ovarian cancer in postmenopausal women, in a high-quality diagnostic test accuracy study.

Of the six diagnostic tests investigated, the IOTA ADNEX model which looks at ultrasound features (how the lump looks on ultrasound) had the best accuracy of all and could detect up to 96% of ovarian cancers.

The ultrasound test outperforms the current standard of care in the UK significantly and so researchers recommend that the IOTA ultrasound ADNEX model should replace the current standard of care test in the UK which identifies 83% of ovarian cancers.

Sudha Sundar, Professor of Gynaecological Cancer at the University of Birmingham and consultant in gynaecological cancer surgery at Sandwell and West Birmingham NHS Trust – both BHP members – said: “This is the first time that a head-to-head study of all available ovarian cancer tests has been done in the same population. Here we studied their use with symptomatic, post-menopausal women who are most at risk of this cancer. Our trial found that the IOTA ADNEX ultrasound protocol had highest sensitivity for detecting ovarian cancer compared to the standard of care and other tests.

“The ultrasound test also performs well when delivered by a trained sonographer who has received specific training, certification and quality assurance, and as the vast majority of ultrasound scans are performed by sonographers it is important that a new standard is able to be delivered by as many clinical professionals as possible.

“We found that the higher sensitivity of the IOTA ADNEX model is likely to lead to some women who don’t have cancer also being flagged up as having a higher risk of cancer. We however did discuss this extensively with patients, cancer charity Target ovarian cancer and NHS experts who all agreed that in postmenopausal women who are at higher risk of ovarian cancer, picking up more women with cancer would benefit women overall.”

Annwen Jones OBE, Chief Executive at Target Ovarian Cancer said: “Early diagnosis of ovarian cancer is vital, and we are pleased to see this research demonstrate that there are more accurate ways of using ultrasound. The faster and earlier ovarian cancer is diagnosed, the easier it is to treat and the more successful the outcomes. Alongside this innovative research, we need to see greater awareness of the symptoms of ovarian cancer so that women know to come forward to their GP for testing and receive the best possible treatment as quickly as possible. It is crucial that new ways of working like this are rolled out as quickly as possible.”

The research team note that the IOTA ADNEX model achieved 96% accuracy when delivered by NHS sonographers who were appropriately trained and received quality assurance. As most scans worldwide are carried out by sonographers rather than gynaecologists, introductory free online resources have been created by the researchers for NHS staff to undergo the specialist ultrasound training to obtain certification and quality assurance.

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‘Symptom triggered’ test can detect early-stage aggressive ovarian cancer in 1 in 4 cases

Symptom triggered testing, prompted by symptoms such as pain, abdominal bloating/swelling, and feeling full soon after starting to eat, can pick up early-stage aggressive ovarian cancer in 1 in 4 of those affected, according to new research.

A study published in the International Journal of Gynaecological Cancer and funded by the National Institute for Health and Care Research found that the UK’s protocol for picking up early-stage disease in women with high grade serous ovarian cancer—the most common, aggressive, and lethal form of the disease— is an effective way to diagnose even early-stage ovarian cancer.

The findings also show that complete surgical removal of the cancerous tissue is possible even in more advanced disease, providing that women with suspicious symptoms are expedited for investigation and treatment, they add.

A team of researchers led by BHP founding member the University of Birmingham analysed data for 1741 women taking part in the Refining Ovarian Cancer Test accuracy Scores (ROCkeTS) study, which involves 24 UK hospitals. The women had all been fast tracked for treatment under the symptom-triggered testing rapid access pathway.

Sudha Sundar, Professor of Gynaecological Cancer at the University of Birmingham and the Pan Birmingham Gynaecological Cancer Centre at BHP member Sandwell and West Birmingham Hospital NHS Trust said:

“Our figures demonstrate that in a real-world setting, symptom-based testing can potentially lead to diagnosis of high grade serous ovarian cancer with low disease spread and results in a high proportion of complete surgical removal of the cancer.”

“These findings challenge the assumption that the disease should always be considered to be in its advanced stages in women once they develop symptoms.

“More importantly, our findings emphasise the importance of increasing an awareness of ovarian cancer symptoms to facilitate earlier diagnosis via referral through the fast-track pathway to improve patient outcomes.”

Key findings

  • Among participants in the study, 119 (7%) were diagnosed with high grade serous ovarian cancer with an average age of 63, and 90% had gone through the menopause.
  • In most of these women (112; 94%) cancer didn’t hugely interfere with their daily lives, as they were classified with a performance status of 0 or 1, meaning they either were fully active, or were able to do everything but strenuous activities.
  • One in four (30; 25%) had early stage I or II disease.
  • Visible cancerous tissue was completely removed in 73 (61%) and almost completely removed in 18 (15%). The disease was only deemed to be inoperable in 9 (8%).
  • The extent of cancer was low in 43 of the 119 (36%), meaning that it was localised in the pelvis; moderate in 34 (29%), meaning that it had spread to the lower abdomen; and high in 32 (27%), meaning that it had spread up to any of the liver, pancreas, diaphragm or spleen. Information on disease extent wasn’t available for 10 (8.5%) women.
  • Surgery to remove as much of the tumour as possible, which is associated with longer survival, was carried out in more than three in four women (93 ,78%), with almost two thirds receiving surgery ahead of chemotherapy (78, 65%,). 36 (30%) were given chemotherapy to shrink the tumour ahead of surgery; 5 (4%) women didn’t have surgery, information on this was not available for 17 (14%) women.

The UK adopted symptom-triggered testing for ovarian cancer in 2011. Women, especially those over 50 years with these symptoms are tested for levels of the tell-tale protein CA125 in their blood and given an ultrasound scan. Abnormal results prompt a fast-track referral for hospital review by a gynaecologist within 2 weeks.

Ovarian cancer is the seventh most common cancer amongst women worldwide, and the sixth most common cause of cancer death in the UK. While most (93%) women diagnosed with early-stage disease (I or II) survive for more than 5 years, only 13% of those diagnosed with advanced disease (stages III or IV) do so.

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

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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Children’s brain tumours could be diagnosed with 10-minute scan

Children with the most common malignant form of brain cancer could see diagnostic wait times dramatically reduced thanks to new research that trialled a quicker and less invasive way of determining which type of tumour they have. 

The study, published in eBioMedicine, was conducted by a team of researchers led by the University of Birmingham (UoB) and Newcastle University, with Birmingham Children’s Hospital as the lead clinical centre, and funded by Children with Cancer UK and Cancer Research UK. UoB and Birmingham Women’s and Children’s NHS Foundation Trusts are both founding members of BHP.

The collaborative team identified how the four different groups of medulloblastoma, a malignant children’s brain tumour, had a specific profile based on their individual metabolism. Taking cell samples from 86 tumours, a laboratory test was used to accurately identify metabolic markers including chemicals specific to the different tumour groups.  

The study also validated previous research that found that glutamate, a metabolite present across all of the tumour cells, is linked closely with tumour prognosis. 

Significantly, the research could pave the way for using MRI scanning combined with machine learning to assess medulloblastomas for their ‘signature’ metabolic profiles without the need for invasive biopsy and could rapidly reduce the current 3-4 week wait from presentation to full diagnosis. 

Andrew Peet, Emeritus Professor of Clinical Paediatric Oncology at the University of Birmingham and an Honorary Consultant at Birmingham Women’s and Children’s NHS Foundation Trust, who is lead author of the study said: “Time is so important in cancer diagnosis so our findings on different types of medulloblastoma having a detectable signature metabolism could be game changing for quickly diagnosing, and then offering the best possible treatment for children.”

Professor Steve Clifford, Chair of Molecular Paediatric Oncology at the Newcastle University Centre for Cancer, who jointly led the study said: “Providing a rapid diagnosis using innovative scanning and AI (artificial intelligence) techniques, has the potential to revolutionise patient management, allowing early non-invasive diagnosis, tailoring of treatment decisions and reducing the period of uncertainty for patients and parents while awaiting a full diagnosis. Further, our biological findings provide critical new insights into the metabolism underpinning these tumours, and the potential to exploit these therapeutically.”

The latest findings could be game changing for children like Jack Bourne, aged six, from Birmingham who was diagnosed with medulloblastoma in March 2023.

Jack’s dad Tom said: “We’ve been through 13 months of treatment but six weeks of that was just waiting to find out what type of tumour he had. We were so scared.”

Within weeks of starting school, Jack had started experiencing sickness and headaches which doctors put down to possible separation anxiety or vertigo. But when parents Tom and Tom and Suzanna noticed that he was struggling to walk, they sought a second opinion and Jack was referred to Birmingham Children’s Hospital the same day.

“When they told me the results of the MRI scan, I didn’t know what to feel,” said Tom. “As we were trying to digest everything, they were asking us to sign consent forms because they wanted to operate first thing the next morning. You’re reading these forms and all you see is – he might not make it out alive. It’s heartbreaking, it really is.”

Jack pulled through the ten-hour operation to remove the tumour, but it would take more than four weeks for doctors to figure out what specific type of medulloblastoma he had in order to effectively treat it.

“The research that’s going into diagnosing tumours is really important,” said Tom. “In Jack’s case there was quite a delay while they sent his tumour to Great Ormond Street to be analysed. During that time Jack was given some chemo just to start things off because they just wanted to do something rather than just wait. But all you want is for your child to be given the best possible treatment right from the start.” 

Christiana Ogunbote, Head of Research at Children with Cancer UK said: “We are incredibly proud to help fund this innovative medulloblastoma research and are excited to see how it could change the experiences of children diagnosed with this disease and their families. Discovering new ways to improve outcomes for children with cancer is at the heart of what we are trying to achieve. Through continued and sustained investments in research we look forward to a day where every child can survive their cancer diagnosis.” 

Dr Laura Danielson, Children’s and Young People’s Research Lead at Cancer Research UK, said:  “Developing quicker, less invasive ways to accurately diagnose the different types of medulloblastoma, the most common malignant brain tumour in children, is a crucial step in improving outcomes for young patients. 

“This important study has identified a new way to distinguish between the four subgroups of medulloblastoma. This discovery paves the way for the development of simple imaging tests that could quickly and accurately diagnose the different types of medulloblastoma. 

“This kind of discovery research is important to drive new and improved ways to better detect and treat cancers affecting children and young people.”

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