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

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What did the project achieve?

“Our study has improved the understanding of the genetic and biological factors linked with the risk of a child’s neuroblastoma returning,” says Professor Deborah Tweddle of Newcastle University. “These findings are important for future clinical trial planning and will help pave the way to the development of new life-saving treatments for children with high-risk disease.”

Every year in the UK, around 100 children are diagnosed with a type of cancer called neuroblastoma – and around half of these children are diagnosed as high-risk.1 A child with high-risk neuroblastoma may need extremely intensive treatment that can last for years. Despite enduring this gruelling treatment, it doesn’t always work and their cancer may come back (relapse) later. Tragically, many of these children will lose their lives at a young age.

Professor Tweddle reviewed clinical information from 260 children with high-risk neuroblastoma from 11 centres across the UK, including 48 children whose cancer was not responding to treatment.

“We found that children whose neuroblastomas don’t respond well to treatment are more likely to be older than other high-risk patients – and their tumours are also less likely to have a change in a gene called MYCN, which plays an important role in driving cell growth,” says Professor Tweddle. “Sadly, nearly half of these children lose their lives within five years of diagnosis.”

The researchers also used cutting-edge genetic sequencing technologies to compare the genetic make-up of tumour samples from 37 children – collected at diagnosis and after relapse – to search for genetic changes that could be involved in driving the growth of neuroblastomas that come back after treatment.

“We identified an average of 20 new genetic changes in relapsed tumours,” says Professor Tweddle. “Some of these genes are now being investigated as potential targets for the development of new drug treatments that could help improve children’s survival.”

This research was completed on

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. http://www.nhs.uk/conditions/neuroblastoma/Pages/Introduction.aspx Website accessed 26 August 2015.

2. The Neuroblastoma Society. Home page. http://neuroblastoma.org.uk/ Website accessed 26 August 2015.

3. The Neuroblastoma Society. Neuroblastoma FAQs. http://neuroblastoma.org.uk/families/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 July 2020
Grant amount £196,219
Grant code GN2390


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