University of Manchester study identifies best way to treat rare childhood cancer

University of Manchester researchers believe that high doses of a drug named ifosfamide are the best way to treat rare childhood cancer refractory Ewing sarcoma, which affects bones and soft tissues.

Ifosfamide works by adding an alkyl group to DNA, leading to an eventual breaking of DNA strands and affecting cancer cells’ ability to multiply.

The current five-year survival rate for this cancer is approximately 15 percent. Ewing tumours are uncommon, with only about 60 children and young people diagnosed in the UK each year, and there is no standard treatment.

The phase II/III rEECur study was undertaken by the Manchester researchers by evaluating four chemotherapy regimens commonly used for this disease in a randomised trial on 451 patients. Two of the treatments were eliminated from the study due to lack of effectiveness.

Analysis showed that use of ifosfamide led to patients remaining free of progression or second malignancies for a longer period of time (five months, in comparison to three months for the other treatment, topotecan plus cyclophosphamide). The median overall survival for the children was 16.8 months for ifosfamide, versus 10.4 months for topotecan plus cyclophosphamide. Children’s quality of life on ifosfamide scored higher.

However, adults’ quality of life score was not higher, and a greater survival difference was registered in patients aged 13 and below. It was also noted that ifosfamide led more brain and kidney toxicity than topotecan plus cyclophosphamide.

The results were presented by Dr Martin McCabe, study lead and a clinical senior lecturer in pediatric and adolescent oncology at The University of Manchester, at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.

The trial was funded by the Cancer Research UK and European Commission with additional funding from the Aamu Pediatric Cancer Foundation, Australia and New Zealand Children’s Hematology and Oncology Group, Australia and New Zealand Sarcoma Association, Canteen, German Cancer Aid, Swiss Pediatric Oncology Group and the Zoé4life Other Foundation.