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Rhabdomyosarcoma is a soft tissue cancer, which usually forms in skeletal muscle tissue and hollow organs including the bladder and uterus. Despite survival rates for the first occurrence of disease increasing from 25% in 1970 to more than 70% today, around a third of patients will relapse at least once which further increases their risk of dying, as treatment options after relapse are currently limited and innovative new drugs for this disease have been lacking.

The funding will support evaluation of regorafenib, an oral cancer therapy from Bayer, in relapsed rhabdomyosarcoma patients, aiming to determine whether this drug combined with standard treatment can improve survival rates, reduce the chance of relapse and improve long-term quality of life for patients.

The product from Bayer is already approved as a monotherapy across certain forms of colorectal cancer, gastrointestinal stromal tumours, and hepatocellular carcinoma, and will now be investigated in this rare paediatric indication.

The study will also explore biomarkers to evaluate whether they can be used to predict relapse and improve prognosis. The new study arm is one part of the international multi-arm, multi-stage Frontline and Relapse RhabdoMyosarcoma (FaR-RMS) clinical trial, which is already underway at the University of Birmingham, funded by Cancer Research UK.

Dr Scott Z. Fields, Head of Oncology Development at Bayer, commented: “Bayer is committed to developing new cancer treatments for which there is a great medical need, such as for paediatric cancer patients.

“We are pleased to be a partner on this important study supported by academic experts at the University of Birmingham and the European Paediatric Soft-Tissue Sarcoma Group, and are looking forward to the next steps of this trial and the benefits it could bring in the future for patients.”

Professor Pamela Kearns, Director of the University of Birmingham’s Cancer Research UK Clinical Trials Unit (CRCTU), explained: “This new partnership with Bayer highlights our world-leading strengths in paediatric cancers and the excellence of our industry trials work at the University of Birmingham, and further evidences our strengthening capabilities with the forthcoming Birmingham Health Innovation Campus and its Birmingham Precision Medicine Centre (BPMC) Industry Trials Hub.

“Our internationally-leading expertise in complex clinical trial delivery will inform this trial throughout as we seek to validate more effective treatments to enable children with rhabdomyosarcoma to return to remission and lead longer, healthier lives.”

Life Sciences Minister Nadim Zahawi said: “Birmingham is poised to be one of the UK’s powerhouses for innovation and it’s why the government recently designated it a Life Sciences Opportunity Zone – helping to capitalise on its expertise and attract inward investment from leading pharmaceutical and life sciences businesses.

“Today’s multi-million funding from Bayer is evidence of this thriving innovation ecosystem in action, and will enable the University of Birmingham to carry out a ground-breaking clinical trial to help accelerate treatments for children with cancer and improve their quality of life, all while cementing the UK’s status a global leader in life sciences.”

Michelle Mitchell, Chief Executive of Cancer Research UK, said: “We know that, despite more children and young people surviving cancer than ever before, there is more that needs to be done as cancer still represents the leading cause of death by disease for this age group.

“It’s fantastic to hear that Bayer is investing in the FaR-RMS trial to help improve the lives of children and young people with this kind of rare cancer. It’s a testament to the strength of charity-funded research to be able attract this kind of high-level investment to the UK, which helps fuel innovations required to bring much needed new treatments to patients.”

Professor Meriel Jenney, Deputy Medical Director at Cardiff and Vale University Health Board, and Chief Investigator of the Far-RMS trial, commented: “We strongly welcome this partnership with Bayer.

“The FaR-RMS trial is designed to facilitate the efficient testing of new drug combinations for the highest risk patients with rhabdomyosarcoma across Europe and beyond, through its multi-arm, multistage approach.

“This exciting partnership is the first of what we hope may be several partnerships to bring targeted agents into the FaR-RMS trial. The focus on biomarkers alongside the clinical data will also help us to understand better which patients with rhabdomyosarcoma respond to treatment and to find new, smarter ways of assessing that response.”

Richard Torbett, Chief Executive of the ABPI, said: “We are delighted to see Birmingham leading valuable research to find new cancer medicines for children, and industry investing in the West Midlands. Our work with Birmingham Health Partners is to connect industry with Birmingham’s extraordinary life sciences capabilities to tackle global health challenges, and today’s announcement is exactly what we want to see.

“Partnerships like this between academia, companies and the NHS lead to amazing breakthroughs which benefit people not just in the West Midlands but across the UK and the world.”

The BPMC Industry Trials Hub offers comprehensive design of trials which are conducted to industry standard as well as critical trial data, curated to provide a ‘fit for filing’ package which offers accelerated progress to clinical adoption. BPMC will be part of the Birmingham Health Innovation Campus – the only science park in the region dedicated to health and life sciences, creating opportunities for transformative collaborations between the NHS, academia and industry partners.

Notes to Editors

  • For further information please contact either:
  1. Emma McKinney, Media Relations Manager, University of Birmingham
  2. Louise Stanley, Birmingham Health Partners, Communications Manager 

The University of Birmingham is ranked amongst the world’s top 100 institutions, and its work brings people from across the world to Birmingham, including researchers and teachers and more than 6,500 international students from nearly 150 countries.

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to drive sustainable development and generate a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2020, the Group employed around 100,000 people and had sales of 41.4 billion euros. R&D expenses before special items amounted to 4.9 billion euros. For more information, go to

Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research. Cancer Research UK’s pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives. Cancer Research UK has been at the heart of the progress that has already seen survival in the UK double in the last 40 years.Today, 2 in 4 people survive their cancer for at least 10 years. Cancer Research UK’s ambition is to accelerate progress so that by 2034, 3 in 4 people will survive their cancer for at least 10 years. Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses. Together with its partners and supporters, Cancer Research UK's vision is to bring forward the day when all cancers are cured. For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022. Follow us on Twitter and Facebook.

BHP is a strategic alliance between the University of Birmingham and two NHS Foundation Trusts – Birmingham Women’s & Children’s, and University Hospitals Birmingham – where members collaborate to bring healthcare innovations through to clinical application