World-first research in Manchester could revolutionise treatment for children with rare bone cancer

Children with Ewing sarcoma, a rare type of cancer in the bone, or surrounding tissues, are part of pioneering research being delivered in Manchester, that could transform treatment, to improve outcomes and save lives.

Although Ewing Sarcoma can be diagnosed at any age, it occurs most frequently in children and young adults. Approximately 30 children in the UK develop Ewing sarcoma each year. Long term survival for patients diagnosed with localised cancer (where it has not spread to other parts of the body) is approximately 60 per cent. Around five to six children with the condition are treated annually at Royal Manchester Children’s Hospital (RMCH).

The “International clinical research programme to improve outcomes in newly diagnosed Ewing Sarcoma – Trial 1 (INTER-EWING-1)” study is being led internationally by Chief Investigator, Professor Bernadette Brennan, Paediatric Oncologist, RMCH, part of Manchester University NHS Foundation Trust (MFT).

Treatment usually includes chemotherapy (where medicine is used to kill cancer cells), surgery and/or radiotherapy (where high doses of radiation is used to kill cancer cells). The study researchers are aiming to find if adding the drug regorafenib, improves the effectiveness of chemotherapy. They also want to find the optimal dose of radiotherapy, and the benefit of an additional six cycles of “maintenance” chemotherapy treatment to prevent cancer returning.

RMCH recruited the first patient in the world to the INTER-EWING-1 study which also aims to better understand Ewing sarcoma and look at how radiological scans may help predict how the body responds to treatment.

Over the next eight years, INTER-EWING-1 will examine several new treatment strategies, with different strands (randomisations) of the research opening over that time. Initially, all patients recruited to the study will receive extra scans or “maintenance” chemotherapy and in 2025 research investigating radiotherapy benefits will be added.

In the UK, the multi-centred trial, led by the University of Birmingham’s Cancer Research UK Clinical Trials Unit (CRCTU) and has been fully funded by Cancer Research UK.

Harvey’s story

Eleven-year-old Harvey was diagnosed with Ewing sarcoma in the left side of his jaw in June 2024 and is the second person participating in the INTER-EWING-1 study at RMCH. Harvey lives in Swinton with his parents, dad James, mum Kelly, and 14 year-old sister Laila.  

After unsuccessful treatment with antibiotics, further tests and scans revealed Harvey had Ewing sarcoma and was referred to, consultant paediatric oncologist, Professor Brennan at RMCH.

Dad, James said: “We realised there was something wrong one evening when we noticed a huge mass in Harvey’s cheek, that was pushing over his back teeth. It was really alarming and something that seemed to have occurred almost overnight.

“Professor Brennan gave us the treatment plan which involved chemotherapy followed by radiotherapy. We had initially believed Harvey may have had to have very invasive, life-changing surgery, which could have involved removing a significant part of his jaw, so this was reassuring news.”

When Professor Brennan discussed the study with the family, which for Harvey would involve some extra scans and x-rays, they were happy to be part of the research.

Professor Bernadette Brennan, Paediatric Oncologist at RMCH and study lead, said: “Treatments for Ewing sarcoma have not changed much over the past 30 years so more research in this field is vital, and I am proud to be leading this important study at RMCH and internationally.

“Although around six in every 10 people with bone cancer will live for at least five years from the time of their diagnosis, and many may be cured completely, survival rates are lower if the cancer has spread to other areas of the body. Only through the development of personalised treatments can we hope to improve outcomes for our Ewing sarcoma patients globally. We hope the results from this study will help improve the treatment and quality of life of children and adults with Ewing sarcoma in the future.”

James added: “Clearly this research needs be done. When we considered that we could potentially help other children and other families in similar situations, the decision to take part was a simple one. We are thankful to have this wealth of knowledge and expertise on our doorstep and it’s great that this important research is taking place in Manchester.”

Professor Brennan is supported by a team of Haematology and Oncology Research Nurses, Trial Coordinators, pharmacy professionals and radiologists to deliver this study in Manchester.

Michelle Bullock, Senior Paediatric Haematology and Oncology Research Nurse, Paediatric Oncology and Haematology Research Team said: “Harvey’s treatment will involve 14 cycles of chemotherapy, which he will receive over more than six months. He was able to return home recently after his third cycle of chemotherapy and will come back to RMCH for a few days at a time for each cycle.

“We are very grateful to Harvey and to all our patients and their families who help to support research.”

Dr Laura Danielson, Research Lead for Children and Young People’s Cancer at Cancer Research UK, said: “The INTER-EWING-1 trial is a crucial opportunity to change the outlook for children and young people with Ewing sarcoma. Not only does it focus on improving current treatments, but it also introduces regorafenib, a new drug to be trialled as a frontline treatment for this type of cancer.

“This research could mark a significant step forward in the treatment of this cancer, offering fresh hope to patients of all ages and their families.”

INTER-EWING-1 is open to all ages over two years old.  Approximately 900 participants will be recruited internationally. The trial will run across Europe and Australasia. The study will run until 30 November 2032.