Neuroblastoma
All cancer treatments must be fully researched before they can be used for neuroblastoma. This is so we can be sure that:
they work
they work better than the treatments already available
they are safe
To make sure the research is accurate, each trial has certain entry conditions for who can take part. These are different for each trial.
Hospitals do not take part in every clinical trial. Some trials are only done in a small number of hospitals, or in one area of the country. You or your child may need to travel quite far if you take part in these trials.
The following is a video about Amy and Poppy's experience of a clinical trial. It lasts just over 2 minutes.
Go to Cancer Research UK’s clinical trials database if you are looking for a trial for neuroblastoma in the UK. You need to talk to your child’s specialist if there are any trials that you think they might be able to take part in.
Some of the trials on this page have now stopped recruiting people. It takes time before the results are available. This is because the trial team follow the patients for a period of time and collect and analyse the results. We have included this ongoing research to give examples of the type of research being carried out in neuroblastoma.
We have also included some early research. These are clinical trials mainly looking at whether a new medicine is safe and what side effects it may cause.
Scientists are studying blood samples and samples of neuroblastoma tissue. This is to collect information about the changes in children’s cancers. The trial team would like to learn more about neuroblastoma. They also want to help find out if they can choose the best treatments based on any genetic changes.
Find out more about the StratMedPaediatrics trial
A large research study is looking at improving treatment for high risk neuroblastoma. High risk neuroblastoma can be difficult to treat, so researchers around the world are looking to improve treatment. They are looking at 3 main areas:
comparing 2 different combinations for
looking at if having an extra treatment of and 2 works better than transplant treatment
looking at the amount of radiotherapy needed for treating high risk neuroblastoma. The researchers want to see if adding an extra, or boost treatment of radiotherapy helps
Go to information about this clinical trial
Neuroblastoma that does not go away with treatment is called refractory neuroblastoma. Neuroblastoma that comes back after treatment is called relapsed neuroblastoma.
A study is looking at different combinations of treatment for neuroblastoma that either has:
continued to grow despite treatment
has come back afterwards
The researchers in this study are looking at different combinations of the following cancer drugs:
irinotecan
temozolomide
dinutuximab beta
bevacizumab
Irinotecan and temozolomide are types of chemotherapy. Bevacizumab and dinutuximab beta are types of .
Find out more about the BEACON2 trial
Targeted cancer drugs work by ‘targeting’ the differences that help a cancer cell to survive and grow.
Ribociclib is a type of targeted cancer drug called a . Researchers want to find out if this treatment works for children whose neuroblastoma has come back or not gone away with treatment.
Children taking part in this trial will have ribociclib in combination with chemotherapy. The chemotherapy drugs are topotecan and temozolomide. This is early research.
Other researchers are looking at existing targeted cancer drugs. These targeted drugs are already approved treatments for some types of cancers. There are several groups to this study and they want to see how well these drugs work for rare cancers like neuroblastoma.
Find out more about the DETERMINE trial
Immunotherapy uses the to fight cancer.
Researchers are looking at combining mIBG with the immunotherapy medicines. The immunotherapy medicines are nivolumab and dinutuximab beta. This is an early phase trial.
mIBG is already an established treatment for neuroblastoma. It is a type of . Radioactive iodine is attached to the mIBG. Neuroblastoma cells take up the mIBG treatment wherever they are in the body. The radioactive iodine then kills the neuroblastoma cells.
Get more information on this study
Researchers are looking at a monoclonal antibody called naxitamab with a . This early research is looking at how well this treatment works for children who have high risk neuroblastoma and either:
the cancer has come back
treatment has stopped working
Find out more about this trial
Researchers are looking at using CAR T-cell therapy to treat neuroblastoma.
CAR stands for chimeric antigen receptor. are good at fighting infection and disease like cancer. But it can be difficult for them to tell the difference between a cancer cell and a normal cell. So the cancer cells can hide away and not be recognised.
Scientists are trying to find ways to get T cells to recognise cancer cells. One possible way to do this is CAR T-cell therapy.
Find out more about CAR T-cell therapy
Researchers are also looking into the long term side effects of cancer treatment. They are doing this for all types of children’s cancers.
This research project has collected information from children who were:
diagnosed with cancer between 1940 and 1991
lived for at least 5 years after diagnosis
The researchers are interested in:
the long term health problems people have developed
how well treatment worked for people who had cancer as a child
This type of research takes many years to produce results. This is because the children need to be followed throughout their lives. Researchers today are still using information collected in the study.
This large study was set up in the early 1990’s to find out about the causes of cancer in children. It includes over 140,000 children diagnosed under 15 years of age.
The study team are now looking at the health and healthcare needs of these people diagnosed with cancer as children.
You can read about their findings in their publications section. There are many published articles. The information in these articles is not in plain English. It has been written for healthcare professionals and researchers.
Last reviewed: 04 Sept 2026
Next review due: 04 Sept 2029
Surgery, chemotherapy, immunotherapy and radiotherapy are the main treatments for neuroblastoma. Find out how doctors work out your child's treatment.
Get information on organisations and resources that can help you and your family cope with a diagnosis of neuroblastoma.
In the UK around 1,838 children (aged 0 - 14 years) get diagnosed with cancer each year. Find out out the most common types.
Our clinical trials aim to find out if a new treatment or procedure is safe, is better than the current treatment or helps you feel better.
Find out about what to expect when your child is first diagnosed.
Neuroblastoma is a rare cancer that affects children, mostly under the age of 5. Around 100 children are diagnosed each year in the UK. Find out more about the symptoms, tests your child might have, treatment, side effects, coping and research.
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