A new Cancer Research UK-funded clinical trial being sponsored by BHP founder-member the University of Birmingham has opened for recruitment – aiming to investigate the efficacy of a novel treatment for patients affected by a specific type of blood cancer.
PROMise, which is being co-ordinated via the Cure Leukaemia-funded Trials Acceleration Programme (TAP) hub at the University of Birmingham’s Cancer Research UK Clinical Trials Unit (CRCTU), will recruit patients aged over 16 who suffer with myelofibrosis (MF).
Over the next two years, 15 NHS centres will recruit MF patients who will be given a novel agent called PLX2853 in addition to the existing standard treatment of ruxolitinib.
Each year in the UK over 300 patients are diagnosed with MF, which is a blood cancer associated with debilitating symptoms including extreme fatigue, pain, weakness and shortness of breath. Around 10-20% of MF patients go on to develop acute myeloid leukaemia (AML) and consequently, a diagnosis of MF has a huge impact on both length and quality of life, with median survival from the time of diagnosis being just two years for patients with high-risk disease.
The only curative therapy for MF is stem cell transplant; however, this is only suitable for a small minority of younger patients who don’t present with comorbidities (the effect of all other conditions an individual patient might have – physiological or psychological).
The current NHS standard of care for those unsuitable for stem cell transplant is treatment with ruxolitinib, approved for use in 2011 and currently the only therapy approved with an indication for MF.
Professor Pam Kearns, Director of the University of Birmingham’s CRCTU, said: “Whilst ruxolitinib is already in widespread clinical use, many patients do not achieve an adequate response. Significant residual symptoms remain in most patients thus there is a major unmet clinical need and the PROMise trial is addressing an urgent need for improved therapeutic approaches for MF patients.”
Chief Investigator, Professor of Haematology at University of Oxford, Adam Mead, said: “The PROMise study is a really exciting study that has just opened in the UK and will be opening across 15 centres. This is introducing a new treatment called PLX2853, in combination with ruxolitinib, for patients with MF. The impact on patients for this combination of treatments, I hope, will be improvement of their symptoms, improvement of their quality of life, without causing them side effects.”
The initiative is designed to develop and deliver specific practical skills to address the growing need in the UK for skills in vaccine and advanced therapy medicinal product (ATMP) manufacturing.
As one of the three initial National Training Centres, BHP founder-member the University of Birmingham will deliver advanced technology courses on-site in collaboration with local companies and institutions to provide training complementary to the ATSTN initiative. These include modules on ATMP manufacturing design and clinical adoption, as well as documentation and implementation of ATMPs, which will be delivered as a combination of on-site, online learning and virtual reality modules.
The first courses will be launched in September 2021 and will cover the fundamental aspects of ATMPs and Good Manufacturing Practice (GMP). The University of Birmingham will also offer master’s degree level modules on GMP standards, which are a vital requirement for the authorisation, manufacture, and commercialisation of ATMPs.
Professor Phil Newsome, Director of Research in the College of Medical and Dental Sciences at the University of Birmingham, commented: “We are delighted to provide our leading expertise, playing such a pivotal role in delivering these much-needed skills for the UK’s advanced therapies sector. This collaboration between industry and academia will create a truly world-class offering, ensuring the UK remains at the forefront of the manufacture and delivery of vaccines and advanced therapies. Moreover, it will further strengthen and leverage the rapidly-growing health and life science sector in Birmingham and the Midlands.”
The National Training Centres are a part of the ATSTN initiative, which also includes an online training platform to provide remote learning for those currently working in the vaccine manufacturing and advanced therapy industry, and a ‘career converter’, which maps an individual’s transferrable skills from adjacent sectors into recommended roles for this industry.
The ATSTN, driven by industry and coordinated by the Cell and Gene Therapy Catapult (CGT Catapult), was launched in December 2020 with £4.7m in funding awarded from BEIS. The initiative was designed in collaboration with industry to create opportunities to upskill, through digital and practical on-site courses, current professionals and leverage skills from individuals coming from other sectors.
Matthew Durdy, Chief Executive Officer, Cell and Gene Therapy Catapult, commented: “The UK ATMP industry is expanding rapidly, and to support that tremendous growth the industry needs skilled people. The UK is making one of the biggest commitments anywhere in the world to training in this sector, keeping it as the “go to” place for the cell and gene therapy industry. As part of the ATSTN programme, the University of Birmingham is well placed to develop and deliver high-impact training courses utilising its world leading delivery technologies”.
People with ‘healthy’ obesity have a 66% increased risk of chronic kidney disease compared to metabolically healthy individuals with normal weight, finds a new study led by the University of Birmingham.
Those that are metabolically healthy (do not suffer with metabolic conditions such as hypertension, diabetes, high cholesterol or cardiovascular disease) and are overweight but do not have obesity are at a 30% risk of chronic kidney disease compared to healthy people with normal weight, the study found.
The research also found that the increased risk was greater in those aged under 65, while also the more the number of the metabolic complications a person has the higher risk of chronic kidney disease even in the normal weight range.
The research, published in the American Journal of Kidney Disease, was carried out in collaboration with University Hospitals Birmingham NHS Foundation Trust and the University of Warwick.
The retrospective population-based cohort study examined the GP records of around 4.5 million individuals from the UK and tracked their health over an average of almost five-and-a-half-years. Of the 4.5 million individuals, 1,040,921 (23.4%) and 588,909 (13.2%) were metabolically healthy overweight and metabolically healthy obese, respectively.
Chronic kidney disease has a major impact on global health and cost the UK National Health Service £1.45 billion in 2009-2010. In 2017, 697.5 million people in the world had the disease, and it accounted for 1.2 million deaths. The prevalence of chronic kidney disease increased by almost 30% between 1990 and 2017, while the number of deaths caused by the condition rose by 41.5% in the same period. Similarly, the prevalence of obesity is also on the rise, tripling between 1975 and 2016.
First author Dr Jingya Wang, Research Fellow at the University of Birmingham, said: “Our results demonstrate that individuals with metabolically healthy obesity might have a higher risk of developing chronic kidney disease compared with normal weight individuals, especially those younger than 65 years.
“Looking to the future, a clinical trial of a weight loss intervention could be considered in these individuals to help us establish whether this can reduce their high risk of chronic kidney disease.”
Dr Abd Tahrani, Senior Lecturer in Metabolic Endocrinology and Obesity Medicine at the University of Birmingham, says: “It is likely that weight loss in people with metabolically healthy obesity is likely to reduce their risk of chronic kidney disease as it will also reduce their risk of future type 2 diabetes, and hypertension, both of which can worsen kidney function and result in chronic kidney disease, however this needs to be examined in future trials.
“In addition, we know from previous trials that weight loss in individuals with normal weight can reverse non-alcoholic fatty liver disease for example, hence weight loss could offer an important strategy to prevent obesity complications in this population.”
Dr Krish Nirantharakumar, also of the University of Birmingham, said: “Furthermore, our results suggest that individuals with normal weight who have metabolic abnormalities are also at a higher risk of chronic kidney disease and as such might benefit from meticulous metabolic control to reduce the risk of developing the condition.
“Ultimately, chronic kidney disease is largely preventable, and therefore, it is important to identify and treat the underlying modifiable causes and risk factors.”
This new study follows previous research led by the University of Birmingham and published in 2017 in the Journal of the American College of Cardiology which showed that individuals living with obesity who are metabolically healthy have an increased risk of cardiovascular disease events compared to those who are normal weight without metabolic abnormalities.
Neil Thomas, Professor in Epidemiology and Research Methods at the University of Birmingham, adds: “This new study and our previously published study together demonstrate that individuals who are metabolically healthy can develop organ damage over time.
“Therefore, metabolically healthy obesity should not be considered ‘benign’ or harmless and addressing obesity in metabolically health people might reduce organ damage including chronic kidney disease.”
Children and young people living with cancer from across the West Midlands, Oxfordshire and parts of Southern England will now benefit from improved genetic testing, thanks to a seven-figure donation to Birmingham Children’s Hospital Charity from Children with Cancer UK, in partnership with Kwik-Fit.
Home to one of the largest children’s cancer centres in the UK, BHP member Birmingham Children’s Hospital cares for over 200 patients with cancer, leukaemia and brain tumours every year, from the West Midlands and beyond.
Genomic testing for patients diagnosed with cancer is incredibly important and over the last decade, large scale sequencing projects have identified pertinent DNA changes, which have enabled scientists to develop new and improved cancer drugs and treatments to specifically target these variations.
Sequencing all possible DNA changes in a single test gives scientists the best possible chance of detecting the genetic changes driving a child’s cancer; and knowing the genetic makeup of a child or young person’s cancer, or tumour, allows clinicians to offer a more tailored treatment.
Targeted therapies have improved the number and different types of treatment offered to children and young people, aimed at saving more lives and improving the quality of life for patients living with cancer.
The NovaSeq 6000 is a state-of-the-art piece of DNA sequencing equipment which has enabled the rapid expansion of cancer genetic testing at the West Midlands Regional Genetics Laboratory.
Now a donation of just over £1million to Birmingham Children’s Hospital Charity has allowed the laboratory to purchase a high-throughput next generation sequencing platform. This investment is possible thanks to the incredible fundraising efforts of Kwik-Fit’s staff, customers and suppliers who raised the substantial sum after Kwik-Fit employees chose Children with Cancer UK as the company’s national charity partner.
The NovaSeq 6000 is a state-of-the-art piece of DNA sequencing equipment which has enabled the rapid expansion of cancer genetic testing at the West Midlands Regional Genetics Laboratory.
Initial forecasts provided by NHS England and NHS Trusts across the West Midlands, Oxfordshire and parts of Southern England suggest that approximately 560 children and young people per year could benefit from large cancer panel genetic testing following the installation of the NovaSeq.
The significant gift also allowed for the refurbishment of the laboratory in which the NovaSeq will sit.
The Chairman of Birmingham Women’s and Children’s NHS Foundation Trust and its Charity, Professor Sir Bruce Keogh, former Medical Director of the NHS in England, said: “The NovaSeq 6000 has dramatically enhanced our ability to identify diagnostic and prognostic biomarkers for childhood and young people’s cancers, thereby enabling our expert scientists and doctors to quickly optimise and develop more effective and less toxic treatments for children and young people with cancer.
“We’re incredibly thankful to Children with Cancer UK and all the employees at Kwik-Fit for enabling us to expand our cancer genetic testing capability in this way. It really will make a difference to thousands of families living with a cancer diagnosis.”
Children with Cancer UK, the charity dedicated to the fight against childhood cancer, is a long-term supporter of Birmingham Children’s Hospital and is making its second £1million donation to the hospital’s charity. The first helped ensure the opening of a brand new Children’s Cancer Centre in 2018.
Children with Cancer UK Trustee, Nick Goulden, said: “We are delighted the new NovaSeq 6000 has been installed at the West Midlands Regional Genetics Laboratory, ready to serve its young cancer patients across the region. By offering personalised treatments through genetic sequencing, it’s another step towards our ultimate goal of saving the life of every child and young person diagnosed with cancer.
“We are also incredibly grateful for our year-long partnership with Kwik-Fit who shared this vision with us. Through their innovative and dedicated fundraising, Kwik-Fit employees raised £1million to make all of this possible and the legacy of our relationship will benefit the lives of thousands of children and young people in the future.“
Kwik-Fit’s partnership with Children with Cancer UK, which took place before the pandemic, saw its staff participate in events such as the Virgin Money London Marathon and the Simplyhealth Great North Run. It also organised various fundraising activities including bake sales, static bike challenges, car washes and sponsored walks. In addition, staff swapped cars for cycles as part of the company’s first-ever ‘Tour De Branch’ – a nationwide bike ride in which over 80 employees covered 2,500 miles between 120 Kwik-Fit centres.
Mark Slade, managing director of Kwik-Fit, said: “We were delighted to hit our fundraising target of £1million which has enabled Birmingham Children’s Hospital Charity to invest in such an important piece of equipment.
“I would like to congratulate and thank all of the Kwik-Fit staff for their tremendous effort and dedication and all our customers and partners who supported us – we would not have been able to achieve this target without them. We hope that this vital testing equipment will make a massive difference to the outcome for many families in the future.”
