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Research into neuroblastoma

Researchers are very interested in the diagnosis and treatment of neuroblastoma. There are a number of clinical trials for people to join in the UK. And many children and young people with neuroblastoma have their treatment as part of a clinical trial.

Individual trials close when enough people have joined. There might then be a period of time before the results are available. New trials then open based on the lessons learned.

Diagnosing neuroblastoma

Researchers are looking into different types of scans to see which is better at showing where neuroblastoma has spread in the body.

Scientists are also studying blood samples and samples of neuroblastoma tissue. This is to look at changes inside the cancer cells. Researchers would like to know if doctors can choose the best treatments based on these changes.

Treating neuroblastoma

Doctors are looking into improving the treatment for high risk neuroblastoma.

This video shows one way that Cancer Research UK is supporting research into neuroblastoma.

Targeted cancer drugs

Cancer cells have changes in their genes that make them different from normal cells. These changes mean that the cell might grow faster and work differently from normal cells. Targeted cancer drugs take advantage of this and target the specific gene changes the cancer cells have.

Several trials of new targeted cancer drugs and different ways of giving them have recently closed. New trials into targeted cancer drugs are being developed.

Researchers also want to find out if having a drug called bevacizumab can help children with neuroblastoma. Bevacizumab is a type of targeted cancer drug called a monoclonal antibody that targets the tumour’s blood supply.

This trial is also comparing different combinations of chemotherapy to see which works best. The trial is for children whose chemotherapy is no longer working and for those who have finished treatment but their neuroblastoma has come back.


A trial is looking at a new treatment using anti-GD2 T-cells. T cells are part of the immune system and help fight infection. The researchers are taking immune cells from the blood and changing them so they can attack the cancer cells. They change them by putting a gene into them. Genes are coded messages that tell cells how to behave. Doctors hope the new gene will help the T cells to recognise and attack the cancer cells.


Radiotherapy is a common treatment for neuroblastoma. It uses high energy x-rays to kill cancer cells. Doctors plan radiotherapy treatment so that cancer cells get a high dose of radiation and healthy cells receive a lower dose. But this can be hard to do using conventional radiotherapy.

Researchers have developed a new type of radiotherapy called Intensity Modulated Arc Therapy (IMAT). This allows radiotherapy to be directed more accurately at the cancer. Doctors think that using IMAT means a higher dose of radiotherapy can be given so the treatment might be better at killing the cancer cells.

Targeted radiotherapy

Researchers are testing drugs called somatostatin analogues. These drugs can target a protein called somatostatin on the surface of neuroblastoma cells. It is similar to the mIBG treatment that some children with neuroblastoma have.

LuDO is a somatostatin analogue that has a radioactive molecule attached to it. LuDO finds the neuroblastoma cells and then the radioactivity kills them.

Blood and bone marrow tests

A recent study looked at particular protein markers found in the blood at diagnosis. The study team concluded that these markers can help show which children with stage 4 neuroblastoma have high risk disease. And so they might not respond as well to standard treatment. Knowing this early on could help doctors decide to give newer treatments straight away. We need more research to see how helpful these blood tests are.

Researchers are looking at collecting tumour cells that have broken away from neuroblastoma. These are circulating tumour cells. They want to find out if it is possible to collect these cells from the blood and bone marrow. And if so, use these cells to help develop new drugs to treat neuroblastoma.

Research into side effects

Researchers are also looking into the long term effects of treatment for all types of childhood cancer. The Centre for Childhood Survivor Studies is carrying out the British Childhood Cancer Survivor study.

This research is looking at children who were diagnosed with cancer between 1940 and 1991 and lived for at least 5 years after diagnosis.

This type of research takes many years to produce results because the children need to be followed throughout their lives. So it will be some time before we know the results.

Find a clinical trial

Last reviewed: 
16 May 2018
  • Cancer Research UK Clinical Trials Database

    Accessed 2017

  • A Systematic Review of Models of Care for the Follow-Up of Childhood Cancer Survivors

    M Heirs and others

    Paediatric Blood Cancer, 2013

    Volume 60

  • Neuroblastoma mRNAs predict outcome in children with stage 4 neuroblastoma: a European HR-NBL1/SIOPEN study

    VF Viprey and others

    Journal of Clinical Oncology, 2014

    Volume 1, Issue 32

  • The information on this page is based on literature searches and specialist checking. We used many references and there are too many to list here. If you need additional references for this information please contact with details of the particular issue you are interested in.