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Neuroblastoma: helping children’s immune systems to fight high-risk cancers

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What did the project achieve?

“As a result of the Action Medical Research funding, we have successfully developed solutions that will be implemented into the next clinical trials of this cutting-edge immunotherapy for children with high-risk neuroblastoma,” says Professor Anderson of UCL Great Ormond Street Institute of Child Health. “These refinements are designed to lead to more effective and kinder treatments in upcoming clinical trials.”

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 many years. Despite enduring this gruelling treatment, it doesn’t always work and their cancer may come back later. Tragically, many of these children will lose their lives at a young age.

The researchers are aiming to develop a new treatment for children with high-risk neuroblastoma, which harnesses the power of a child’s own immune system to combat their disease. The approach – called chimeric antigen receptor (CAR) T cell therapy – has already proved effective for treating other types of blood cancer. It involves collecting immune cells called T cells from a child’s blood, genetically modifying them so they recognise and kill cancer cells, and putting them back into their bloodstream to fight the disease.

“During this study, we’ve been contributing towards and building on the results from the clinical trial of CAR T cell therapy* as they emerge – identifying ways to refine the genetic modification technology so that the patient’s T cells are more effective at distinguishing between cancer and healthy cells,” says Professor Anderson. “We hope this will boost the chances of the treatment working while also reducing the risk of unwanted side effects.” 

In the laboratory, the researchers carried out tests to find out how well different types of CAR T cells kill neuroblastoma cells, whilst avoiding healthy cells. They hope their results will ultimately benefit children with neuroblastoma – by refining the treatment to make it safer and more effective over time.

This research is contributing to a large effort from a number of groups in the UK and around the world seeking to improve understanding of CAR T cell therapy for neuroblastoma and other childhood solid cancers.

*Between 2014 and 2021, the team ran a clinical trial of CAR T cell therapy for children with high-risk neuroblastoma, which was funded by Cancer Research UK.


  1. NHS website, Neuroblastoma: https://www.nhs.uk/conditions/neuroblastoma/ [website accessed 5 May 2022]

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 While many children with this cancer do well, others lose their lives despite going through gruelling treatment that can have severe side effects. Professor John Anderson, of University College London’s Institute of Child Health, is developing a way to programme children’s own immune systems to recognise and destroy cancer cells, while sparing healthy cells. He hopes this will one day help free children from the side effects of existing treatments and save more children’s lives.

This is a joint project which is being funded by Action Medical Research, Great Ormond Street Hospital Children's Charity and Neuroblastoma UK.​

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.4-6 Sadly, though, the outlook is less certain for children with high-risk disease.

“Children with high-risk neuroblastoma need extremely intensive chemotherapy and radiotherapy, along with other treatments,” says Professor Anderson. “The children spend a long time in hospital and they can get a lot of side effects. For example, chemotherapy, which involves frequent injections, can make children feel sick and cause them to lose weight, sometimes meaning they have to be fed through a tube. Longer-term side effects are possible too.”

“Children’s treatment normally lasts for a year,” says Professor Anderson. “Sadly, despite the intensity of treatment, high-risk neuroblastoma can be hard to cure and many children still go on to lose their lives.”6

Safer, more effective treatments are urgently needed.


How could this research help?

“We’re developing a new treatment for children with high-risk neuroblastoma, which harnesses the power of children’s own immune systems to combat their disease,” says Professor Anderson.

The new treatment is called chimeric antigen receptor (CAR) T cell therapy. It will involve collecting immune cells called T cells from a child’s blood, modifying them so they recognise and kill cancer cells, and putting them back into the child’s bloodstream. This approach has already proved successful in leukaemia and lymphoma.

“We’re investigating, in the laboratory, how to make CAR T cell therapy as safe and effective as possible for children with high-risk neuroblastoma,” says Professor Anderson. “We think we’ve found a way to create CAR T cells that are better at killing cancer cells, which could give children more chance of a cure, and less likely to attack healthy cells, which could spare children from unpleasant side effects. If we’re right, clinical trials could soon follow.”


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.

6. American Cancer Society. Survival rates for neuroblastoma based on risk groups. http://www.cancer.org/cancer/neuroblastoma/detailedguide/neuroblastoma-survival-rates Website accessed 6 October 2015.




Project Leader Professor John Anderson BA MBBS PhD FRCPCH
Project Team Dr Jonathan PH Fisher MBBS MRCPCHDr Martin Pule MBBS PhD MRCPDr Karin Straathof MBBS PhD MRCPCH
Project Location Section of Cancer, University College London Institute of Child Health and Great Ormond Street Hospital, London
Project Location Other Department of Haematology, University College London Cancer Institute
Project duration 2.5 years
Date awarded 20 July 2015
Project start date 1 October 2016
Project end date 31 December 2020
Grant amount £197,027
Grant code GN2400


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