Biomarker evaluation of neuroblastoma mRNAs in children enrolled in the BEACON trial When combinations of drugs are given to children, it is not always known which ones are providing benefit and which ones are contributing to toxicity alone. Biomarkers from blood tests can help predict which patients will respond to a particular therapy. In this study, blood and bone marrow samples from 120 children treated in the BEACON-Neuroblastoma trial will be collected in order to analyse levels of neuroblastoma mRNAs – molecules that carry codes to make proteins – and the results will be evaluated looking at which children responded to therapy. If the biomarkers are validated in this research study, they can be used in future to predict which children are likely to respond to a therapy before they are treated. Principal investigator: Sue Burchill, MD, University of Leeds Project grant: £41,500 Date of award: October 2016 Background Less than 10% of children with relapsed neuroblastoma survive, making new treatments a vital need. Studies have shown mRNAs in peripheral blood or bone marrow at diagnosis have prognostic value and can identify children with better outcome at relapse. This project seeks to evaluate the prognostic value of certain biomarkers and ascertain the correlation of these biomarkers with drug efficacy in children with neuroblastoma. Results of this study will validate the use of these biomarkers to inform patient selection for particular drug therapies in future. What difference will this project make? Evaluating the prognostic role of mRNAs in children with relapsed/refractory neuroblastoma could serve to identify populations of children who are more likely to benefit from treatment with chemotherapy or bevacizumab. This would allow the incorporation of mRNAs into future neuroblastoma clinical trials, helping with patient selection and stratification, providing increased patient benefit and accelerating drug development. What makes this project stand out? This project has significant potential to help the children of tomorrow, by allowing the incorporation of mRNAs into future neuroblastoma clinical trials and helping with patient selection and stratification. This would mean children who are unlikely to respond could be saved from undergoing significantly toxic therapy unnecessarily, and perhaps have an opportunity to begin other experimental treatments earlier. Sue Burchill, director of the Children’s Cancer Research Group at the University of Leeds said: "We have developed a simple blood test that can identify, at the time of diagnosis, children with neuroblastoma who are unlikely to respond well to current treatment. Being able to spot these children early means they could be offered alternative treatments that might be more effective. It is now important that this is validated and we define how best to use the test in the clinic for the benefit of these children." "With funding from Solving Kids’ Cancer, we are currently investigating how this blood test can be used in children with drug refractory or relapse disease to improve outcome by adapting treatment in real-time depending on patient-specific characteristics and response. This important research would not be possible without funding, and the hard work, support and commitment of Solving Kids’ Cancer supporters and fundraisers. Thank you." About the research team Professor Sue Burchill, MD, PhD, is Professor of Adolescent and Paediatric Cancer Research and Director of Postgraduate Research at the University of Leeds. She received her BSc (Hons) in pharmacology in 1982 and her PhD in medicine in 1986. Prof Burchill is a member of the American Association of Cancer Research, the British Association of Cancer Research, the Children’s Cancer and Leukaemia Group, the International Society of Paediatric Oncology, the Society International of Oncology Paediatric-Neuroblastoma, Teenagers and Young Adults with Cancer, and The Royal Society of Medicine.