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Neuroblastoma: helping children who are at greatest risk from this potentially life-threatening cancer

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Around 100 children are diagnosed with a type of cancer called neuroblastoma each year in the UK.1,2 Most are young – less than five years old.1,3 Many children with this cancer do well. Sadly, though, other children lose their lives despite going through gruelling treatment. Professor Deborah Tweddle, of Newcastle University, aims to help those children who are at highest risk. Her work could lead to better ways to identify children whose cancer is most likely to come back after treatment, which could guide treatment decisions. It could also lead to more effective, personalised treatments that save more children’s lives.

Action Medical Research and Great Ormond Street Hospital Children's Charity are jointly funding this research.​

How are children’s lives affected now?

When children are diagnosed with neuroblastoma, their chances of making a good recovery vary considerably. Most children with low-risk disease will be cured.4-5

Sadly, though, the outlook is not so good for children with high-risk disease. “Children with high-risk neuroblastoma need intensive treatment, which lasts for over a year and causes unpleasant side effects,” says Professor Tweddle. “Children normally have chemotherapy, surgery and radiotherapy, amongst other things, meaning they spend long periods of time in hospital.”

“Nearly half of children with high-risk disease will not respond to treatment or will find their cancer comes back,” says Professor Tweddle.4 “This is particularly hard when children have gone through such aggressive, prolonged therapy. Ongoing care after recurrence focuses on trying to control children’s disease, and maintain their quality of life, for as long as possible. Sadly, though, most of these children eventually lose their lives.”

More effective treatments for children with high-risk neuroblastoma are urgently needed.


How could this research help?

“We aim to find better ways to predict which of the children who’ve been diagnosed with neuroblastoma are most likely to have a relapse after treatment,” says Professor Tweddle. “The ability to identify these children at an early stage would enable doctors to adjust their treatment recommendations. Doctors might, for example, suggest that a child who’s at particularly high risk tries an experimental treatment if one’s available.”

The team’s work involves studying the genetic make-up and the clinical features of children’s tumours to determine the likelihood of relapse and the length of time the child survives following relapse. This is likely to vary from one child to another, so the researchers are investigating whether any particular genetic changes raise children’s chances of having a relapse and how long they survive after relapse.

“Our research could enable the development of new drug treatments, which specifically target the particular genetic changes that children have in their tumours at relapse, changes which may have been present at a much lower level at diagnosis,” says Professor Tweddle. “We hope this approach will one day help save the lives of more children with neuroblastoma.”


1. NHS Choices. Neuroblastoma. Website accessed 26 August 2015.

2. The Neuroblastoma Society. Home page. Website accessed 26 August 2015.

3. The Neuroblastoma Society. Neuroblastoma FAQs. Website accessed 26 August 2015.

4. Cole KA et al. New Strategies in Refractory and Recurrent Neuroblastoma:

Translational Opportunities to Impact Patient Outcome. Clin Cancer Res 2012; 18: 2423-28.

5. Park JR et al. Children’s Oncology Group’s 2013 Blueprint for Research: Neuroblastoma. Pediatr Blood Cancer 2013; 60: 985–993.






Project Leader Professor Deborah A Tweddle BSc MBChB PhD FRCPCH
Project Team Dr Richard JQ McNally BSc MSc DIC PhDMrs Nermine O Basta MB BCh MScDr Nick P Bown PhD FRCPath
Project Location Paediatric Oncology & Haematology Group, Newcastle Cancer Centre at the Northern Institute for Cancer Research, Newcastle University
Project Location Other Institute of Health & Society, Newcastle UniversityNorthern Genetics Service, Institute of Genetic Medicine, Newcastle University
Project duration 3 years
Date awarded 20 July 2015
Project start date 1 April 2016
Project end date 31 March 2019
Grant amount £196,219
Grant code GN2390

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