Author: Louise Stanley

Patient symptom and quality of life assessments must be inclusive and equitable

Written by Louise Stanley on 09/05/2022. Posted in Health inequalities.

Information reported directly by patients can be invaluable for assessing the impact of disease and treatment on patients’ symptoms and quality of life -but more needs to be done to include under-served groups to avoid rising inequalities in healthcare, say experts at BHP founder-member the University of Birmingham.

The information is commonly collected in both clinical trials and clinical practice and ensures that the patient’s perspective is at the heart of decision making. As use of these data become more commonplace, however, researchers in the University’s Centre for Patient-Reported Outcomes Research are calling for more to be done to ensure such information can be provided by everybody.

In an article published 5 May 2022 in Nature Medicine, researchers in collaboration with patients, regulators and international experts identify some of the barriers to participation such as access to technology, disability, language and cultural requirements and call for these to be addressed.

Lead author, Professor Melanie Calvert, said: “When we start to embrace new approaches to healthcare, such as use of patient-reported outcomes, we need to make sure that barriers to participation are addressed at an early stage. If we don’t do this, the gaps between advantaged and disadvantaged populations will only get worse.

“Representative diversity in clinical trials is vital to ensure new medicines and technologies are applicable to the population they are intended to serve. Targeted initiatives are needed to ensure that no groups are excluded from participation in patient-reported outcome data collection, both in research settings and routine clinical care.”

Specific recommendations in the article include:

- - Improve diversity by involving individuals who represent target populations when designing patient questionnaires.
  - Consider the type and severity of disease when seeking patient views, including cognitive or functional impairment that would limit participation.
  - Be aware of cultural needs or languages that might be a barrier, and address these sensitively and appropriately.
  - Include individuals with all levels of reading, writing and problem solving abilities by ensuring material is accessible and that assistance is available, if required.
  - Provide alternative methods of participation to promote digital inclusion
  - Where information is being used in drug development, make sure inclusivity is included early on in discussions about data collection.

Rav Verdi, a patient partner who co-authored the work, said: “As a patient and living in a cosmopolitan society it’s encouraging to see that all walks of life have been considered and a means of communication thought about to glean information from the patient and to provide information to the patient regarding their care and treatment. As worldwide issue, information could be shared with treatment centres to provide better care and understanding of different groups of the population.”

Roger Wilson, a patient partner adds: “Listening to patients reveals the range of diversity which must be understood and allowed for by careful design if a study is to offer benefits to society equitably.”

Professor Calvert adds: “Patient-reported outcome measures and data collection must be reflective of diverse and multicultural societies, to improve research and promote equitable clinical care for the benefit of all patients and the public as a whole.”

The team are supported by a number of funders including the National Institute for Health and Care Research (NIHR) Biomedical Research Centre Birmingham and Applied Research Collaboration West Midlands, Health Data Research UK and UK SPINE.

Revolutionary technology has potential to help children with asthma

Written by Louise Stanley on 05/05/2022. Posted in Inflammation and chronic disease.

BHP members Birmingham Women’s and Children’s Hospitals have initiated a new study which could potentially revolutionise care for young people with asthma using artificial intelligence technology.

Over the next two years, 50 children and families will take part in the Childhood Home Asthma Monitoring Study (CHAMP), which uses a small table-top electronic device designed by Albus Health, not dissimilar in size to an Amazon ‘Alexa’ virtual assistant, to personally monitor a child’s symptoms and breathing while they sleep.

Using sensors and a microphone, it measures breathing and heart rate by analysing coughs, wheezing and other noises, while also assessing environmental factors, such as humidity and air pollution levels. The data collected over a period of months will help form a unique and personalised set of triggers which is able to warn of a future asthma attack days before it potentially happens, allowing for action to be taken.

Around one in 11 children in the UK has asthma and it’s one of the most common chronic conditions which causes hospitalisations. The potential of this AI technology is potentially huge; positively impacting thousands in the future.

Dr Prasad Nagakumar, Respiratory Consultant, is the Chief Investigator leading this exciting £1.6million CHAMP study, funded by the National Institute for Health Research. He’s looking forward to working alongside partners including Asthma UK, Imperial College London, Oxford Academic Health Science Network and Royal Brompton Hospital, where patients are also being recruited to take part.

Dr Nagakumar said: “I’m delighted that we’ve now started this exciting study, which has such a huge potential. Over the next two years we’ll be working hard to further understand and develop the use of this innovative monitoring and, importantly, prediction technology.

“Our aim is to build algorithms and clinical-supporting tools for the early detection of asthma attacks in children by capturing warning signs before patients or those giving care perceive them.”

Professor Jeremy Kirk, Clinical Director NIHR Clinical Research Network (West Midlands) and Research and Innovation Director at our Children’s Hospital, said: “Asthma is the most common chronic disease in childhood and blights many lives. This project utilises the very newest cutting-edge technologies to give us further understanding of this condition, hopefully enabling better monitoring, optimal care and a reduction in hospital admissions.

“Dr Nagakumar and the team are to be congratulated on being awarded this highly competitive and prestigious grant.”

Experts at Birmingham Women’s and Children’s develop new test to spot rare eye cancer in unborn babies

Written by Louise Stanley on 03/05/2022. Posted in Cancer outcomes, Early diagnosis and detection.

Experts from BHP members Birmingham Women’s and Children’s Hospitals have developed a life-saving test that allows doctors to spot a rare form of eye cancer in babies in the womb.

The test, which is being rolled out by the NHS in England this week, means that babies identified as being at risk of developing retinoblastoma can be monitored and treated sooner – increasing the chance of saving their eyesight and potentially their lives.

Symptoms of retinoblastoma are hard to detect and a diagnosis can normally only be made once the tumour has progressed and the eye can’t be saved.

The new non-invasive test can detect changes in the genes in DNA and is likely to identify around 50 infants with retinoblastoma each year, in the latest example of the NHS harnessing the power of genomics to diagnose and treat patients faster and more effectively.

Non-Invasive Prenatal Diagnosis (NIPD) also means parents can be informed early in pregnancy if their child is at risk.

The blood sample test is taken from the mother before birth and tested and analysed for mutations, which can determine with almost 100% accuracy if the baby will develop retinoblastoma.

Treatment can then start on the affected eye as soon as the baby is born, with doctors closely monitoring the other eye for any signs. The test can also predict if the disease might develop in their siblings and will be offered to families where there is a confirmed case of retinoblastoma in the family.

In addition to the cutting-edge new test, Drs Trevor Cole and Amy Gerrish, who have been part of our specialist retinoblastoma service, are also developing a non-invasive post-natal cancer test for retinoblastoma patients using eye fluid – which can also identify if a patient is at risk from other cancers later in life. It’s hoped that in the future, this could be eventually done by a simple blood test.

Dr Amy Gerrish said: “The introduction of this technology of cell free DNA analysis will revolutionise the management of all aspects of retinoblastoma from early detection, selection of the best treatments, identification of family members at risk of retinoblastoma and early detection and treatment of associated adult onset cancers.

“We also believe it will help address the huge discrepancy in retinoblastoma outcome for individuals in high income and low and middle income countries which has been highlighted by the World Health Organisation (WHO)”.

NHS Chief Executive Amanda Pritchard said: “The introduction of this pioneering new test is fantastic news for babies and their parents and has the potential to save hundreds of lives over the coming years.

“Cancer is such a terrible illness and a baby being born with it can have a huge impact on parents and families during what should be an incredibly happy time, but backed by world-class innovation and services like the NHS Genomic Medicine Service, through the Long Term Plan the NHS is developing and delivering more cutting edge treatments like this one to help save lives and keep families together”.

Mum Siani Bainbridge, 22, from County Durham, had retinoblastoma herself as a child and feared her baby boy, Oscar, might carry a faulty gene known as RB1 which causes the potentially deadly cancer.

But she was relieved when she took part in a new trailblazing test, where doctors were able to spot the previously hard-to-detect disease and allay her concerns with a programme of treatment straight after his birth.

Siani said: “This took away a lot of stress, knowing that if there was going to be anything wrong then he would be helped straight away.

“Given that the tumours were quite severe when he was born, the fact he could be treated straight away definitely affected his outcome. It was nice to know the day he was diagnosed it was ready, set go”.

Just a week after being born, Oscar started his cancer treatment, which involved chemotherapy and then laser therapy.

While doctors could not save the sight in one eye, they did avoid having his eyeball removed and crucially, he kept his perfect sight in the other eye – as well as avoiding the disease potentially spreading to the brain.

Consultant Clinical Scientist Stephanie Allen, at Birmingham Women’s Hospital, said: “An early diagnosis will allow clinicians to manage, monitor and prepare treatments much earlier which can transform the prognosis for the baby.

“It will also give the family certainty and allow them to prepare for the birth knowing the support the clinical team will give them”.

The NIPD is one of more than 15 new tests and amendments being added to the National Genomic Test Directory (NGTD), which outlines the genomic tests available via the NHS in England through the NHS Genomic Medicine Service (GMS).

The directory, which is the only one of its kind, covers more than 3000 rare diseases and over 200 types of cancer – demonstrating how the NHS is a world leader in harnessing the benefits of genomics, the study of the genes in our DNA and their function, to deliver better patient care.

Among the other additions to the directory are tests for gene mutations that cause forms of breast and endometrial cancer, acute myeloid leukaemia and several rare diseases. A genetic test for a particular type of advanced lung cancer has had a matching treatment recently approved by The National Institute for Health and Care Excellence (NICE), meaning more effective treatment for patients.

Professor Dame Sue Hill, Chief Scientific Officer and Senior Responsible Officer for Genomics in NHS England said: “This new test is a perfect example of how the NHS Genomic Medicine Service is harnessing cutting-edge technology to deliver genomic tests for cancers like this and many other conditions through the National Genomic Test Directory – meaning more comprehensive and earlier diagnoses and more targeted treatments sooner for all our patients”.

Patrick Tonks, Chief Executive of The Childhood Eye Cancer Trust (CHECT): “Any developments such as this new diagnostic test which has the potential to allow treatment to be started much sooner and therefore the real potential to improve patient outcomes is very exciting news for babies and for the families of anyone affected by retinoblastoma. We watch with interest as this new development is rolled out across the country”.

Health and Social Care Secretary Sajid Javid said: “Despite the unprecedented pressure put on the NHS because of the pandemic it is incredible to see continued life-saving innovation taking place, enhancing cancer care and diagnosis even before birth.

“Early diagnosis is vital to ensure these babies are given every opportunity to see, and the best chance of survival. New tests such as these will help clear the COVID backlog, ensuring patients are seen at the right time and provided the right care.

“Our 10-Year Cancer Plan will set out how we will lead Europe in cancer care, improving outcomes for patients across England”.

New spinout Healome Therapeutics to speed development for eye therapeutics

Written by Louise Stanley on 27/04/2022. Posted in Experimental medicine.

BHP founder-member the University of Birmingham has created a new spinout, Healome Therapeutics Ltd, to commercially deploy a platform that delivers a ‘pro-healing’ microenvironment for the leading causes of preventable blindness.

The company’s leading application will be in ocular surface diseases, which are notoriously challenging conditions to treat, and have progressively larger impacts on quality of life as the diseases run their course.

The Healome technology is a novel fluid-gel material that flows like a liquid, and self-structures into a thin, clear, protective layer over the surface of the eye which is gradually dispersed and cleared away by blinking over 2-8 hours (customisable depending on application).

The gel can be used alone, or as a ‘carrier molecule’ to deliver other therapeutics.

Studies have already shown that one of Healome’s formulations has anti-fibrotic (anti-scarring) activity and these healing properties are augmented by combining it with other therapeutics.

The technology was developed by a team led by Professor Liam Grover who is Director of the University’s Healthcare Technologies Institute (HTI). It is envisaged that treatments developed from this platform will come in the form of clear degradable ‘ocular bandages’ that can be applied like normal eye drops.

Professor Grover, who is also a co-founder of Healome Therapeutics, commented: “There are many cutting edge drugs on the market or in development for diseases that affect the surface of the eye. One of the biggest challenges is to keep therapeutics on the surface of the eye for sufficient time for them to have an effect and more generally to regain or replace all the functions of the tear film.”

The company’s founding directors already have prior experience in commercialisation and advancing therapies towards Phase I-III clinical trials and include Professor Anthony Metcalfe, Industrial Professor of Wound Healing, formulation engineer Dr Richard Moakes, and Dr Richard Williams, whose work at the HTI involves translating healthcare technology concepts to finished products ready to enter clinical trials.

Although Healome will initially concentrate on Dry Eye Disease, in the long-term the company aims to partner with healthcare companies to co-develop new therapeutics for delivery to the surface of the eye.

The gels respond to shear stress, which allows it to change back and forth from a liquid to a soft-solid consistency according to the physical forces applied to it, such as extrusion from a container, or blinking.

Its mechanical and drug diffusion properties can be ‘tuned’ by physical rather than chemical changes to the base polymers. These attributes mean that pre-clinical or early clinical safety studies for new formulations will not need to be repeated and will reduce the time and cost to bring new products to market.

CEO of Healome, Dr Richard Williams, commented: “Ocular surface diseases leading to Dry Eye have a disproportionately large impact on health, well-being and the ability to enjoy life. These conditions can also be very expensive for patients to manage. There are many unmet patient, clinical and industrial needs in this area, which Healome Therapeutics is well-placed to address. Pre-clinical safety of the platform is well-established, GMP manufacturing has been set up to supply planned phase 1 trials and we have brought in significant executive experience in eye care to accelerate plans.”

The researchers behind Healome have already raised £2.8m grant funding from the Medical Research Council (MRC) to progress the original concept from lab bench to completing phase 1 human trials. The developed platform and supply chain was then applied to help tackle challenges in ocular surface diseases via a £1.3m grant from the National Institute of Health and Care Research (NIHR) Invention for Innovation programme. The platform has also shown early promise in dermal and orthopaedic applications.

Healome Therapeutics has already raised £400k funding from Innovate UK and SFC Capital, and is now establishing its own laboratories at the Birmingham Research Park, which has been nurturing high-growth biomedical companies since 1986.

Phase I human trials to test the core technology in combination with therapeutics known to prevent corneal scarring and manage severe dry eye will commence in Q2 2022, supported by the University of Birmingham GMP manufacturing facility.

Urine test for bladder cancer could replace thousands of invasive procedures each year

Written by Louise Stanley on 25/04/2022. Posted in Cancer outcomes, Clinical trials, Early diagnosis and detection.

Birmingham researchers funded by Cancer Research UK and liquid biopsy company Nonacus have developed a new urine test for bladder cancer, which could reduce the need for invasive and time-consuming procedures to diagnose the disease.

The test will use highly sensitive liquid biopsy technology developed by Nonacus in conjunction with a panel of biomarkers developed and validated by Mr Rik Bryan and Dr Douglas Ward from the Bladder Cancer Research Centre at BHP founder-member the University of Birmingham, to detect the presence of bladder cancer by finding DNA from tumour cells present in the urine.

The biomarker panel, which consists of 443 genetic mutations that are common in bladder cancer has been validated in a deep sequencing study recently published in European Urology Oncology.

In this study, which was funded by Cancer Research UK and the Medical Research Council, the researchers used the test to analyse urine from 165 people with bladder cancer that had experienced haematuria (blood in the urine), and successfully detected the disease in 144 of them (87%).

The researchers also looked at using the test in 293 patients who had already been treated for bladder cancer and were being monitored for the cancer returning. In this setting, the test returned a higher proportion of false positive results compared to when used in the haematuria clinic (37.5% vs 15.2%), with 99 positive urine tests without a tumour being seen by cystoscopy on the same day. However, during their follow up monitoring, the patients who had those positive results had almost 3-times higher (11% vs 4%) rates of the cancer returning within 24 months indicating that the test could help detect recurrent disease before it is visible by cystoscopy (the camera inspection of the bladder). Further research is needed for the test to be used for surveillance.

Lead researcher Mr Richard Bryan said: “Even though cystoscopy is good at detecting bladder cancer, it’s invasive and time consuming for patients, so we need a better way to diagnose patients. In the future our test could be an easier way to get people with bladder cancer diagnosed faster, and could mean that tens of thousands of cystoscopies on healthy patients can be avoided each year.”

Iain Foulkes, Executive Director of Research and Innovation at Cancer Research UK said “These findings show that this urine test could help diagnose bladder cancer more easily. Early detection of cancer is key for improving patient outcomes and research like this could help identify the patients that need treatment soonest, while easing the pressures of diagnostic procedures on the NHS. We look forward to seeing how the test performs in the next clinical trial.”

The researchers are working in partnership with Nonacus, a provider of genetic testing products for precision medicine and liquid biopsy, to turn their approach into a clinical test for patients to be used within the NHS, and will start a clinical study funded by Cancer Research UK and involving over 3000 patients to evaluate just how powerful the test is at reducing the number of cystoscopies.

Each year, over 300,000 cystoscopies are carried out in England, however, around 80% of patients with haematuria who’ve had cystoscopy are found to have no cancers or abnormalities^1,2. The researchers believe that using the urine test in haematuria clinic could reduce the number of patients requiring a cystoscopy by at least 45%.

Civilians and military take part in study to improve concussion prognosis

Written by Louise Stanley on 08/04/2022. Posted in Clinical trials.

A major UK study to identify new ways to accurately predict if patients will develop long-term complications as a consequence of concussion has been launched, led by experts at BHP founder-member the University of Birmingham and the Defence Medical Rehabilitation Centre, in collaboration with the Defence Medical Services.

With year one funded by the Ministry of Defence (£2m) and projected to run over eight years, the multi-faceted study will include a trial involving 400 civilians and 400 military personnel aged over 18 with a new diagnosis of concussion (also known as a mild traumatic brain injury or mTBI) which has resulted in them needing hospital treatment or rehabilitation.

At specific time intervals over two years, the participants will take part in nine different areas of research using a variety of medical techniques and assessments to establish if these can be used routinely by medics as ‘biomarkers’ to indicate prognosis and long term impact of concussion. Medical techniques and assessments being trialled include brain imaging and function, analysis of blood and saliva samples, and headache measures, as well as mental health, vision, balance, and cognitive performance.

mTBI is common and has been declared a major global public health problem, with 1.4 million hospital visits due to head injury annually in England and Wales – 85% of which are classified as mTBI. It is also estimated that up to 9.5% of UK military personnel with a combat role are diagnosed with mTBI annually.

The research will involve 20 University of Birmingham experts working across disciplines, including neurology, psychology, sports medicine, mathematics and academics within the University’s Centre for Human Brain Health, and will be coordinated by Birmingham Clinical Trials Unit. It will also be driven by experts at the Defence Medical Rehabilitation Centre Stanford Hall; Aston University, Imperial College London; University of Westminster; University of Nottingham; Royal Centre for Defence Medicine; and University Hospitals Coventry & Warwickshire.

Alex Sinclair, Professor of Neurology at the University of Birmingham and Chief Investigator of the project, called mTBI-Predict, explained: “Although classified as mild, and many recover, the consequences of concussion can be profound with many patients suffering long-term disability due to persistent headaches, fatigue, imbalance, memory disturbance, and poor mental health including post-traumatic stress disorder, while it can have a significant impact on the economy through loss of working hours and demand on the health system.

“Identifying those patients most at risk of these disabling consequences is not currently possible. There is therefore a pressing need to develop accurate, reproducible biomarkers of mTBI that are practical for use in a clinical setting and can predict long-term complications. Our programme of research will deliver a step change in the care of patients with mTBI, enabling a personalised medicine approach to target early intervention for those most in need but also identifying those with a good prognosis who can return rapidly to activities of daily living.”

Co-Chief Investigator, Air Vice-Marshall Rich Withnall QHS Director of Defence Healthcare, UK Ministry of Defence said: “I am delighted that the Defence Medical Services, including the Defence Medical Rehabilitation Centre at Stanford Hall, will be working hand-in-glove with class-leading civilian colleagues and the National Rehabilitation Centre Programme. I fully support this ground-breaking research which I am confident will lead to significant clinical innovation to benefit military and civilian patients, and have translational positive impact for sporting activities from grass-roots to elite levels.”

Peter McCabe, Chief Executive of Headway – the brain injury association, said: “We know that even a seemingly minor head injury can have a major impact on a person’s life – and often the lives of those closest to them. This is particularly the case if the brain injury goes undiagnosed or its effects are mistaken for other conditions. The frustration of not having an accurate diagnosis or receiving the right support can be compounded by the lack of a clear recovery pathway or timeline. We therefore welcome this study in the hope that it can advance our understanding of concussion and mTBI.”