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Neuroblastoma

Treatment for neuroblastoma that has not gone away or has come back

Neuroblastoma that does not go away with treatment is called refractory neuroblastoma. Recurrent (or relapsed) disease is neuroblastoma that comes back after treatment.

Treatment

It can be more difficult to control recurrent or refractory neuroblastoma. But there are treatments available. Researchers are looking into how to improve treatment for children living with neuroblastoma.

The treatment your child needs depends on a number of factors including:

  • where it has come back

  • the original risk group they were in

  • the treatment they have had before

  • the seriousness of any side effects they have or have had before

Low or intermediate risk group

For children with low or intermediate risk disease that has come back where it started. They might need surgery again, with or without chemotherapy.

High risk group

For children with high risk neuroblastoma, treatment is usually more intense and is likely to include a combination of treatments again.

The type of chemotherapy your child will have is likely to be different from their first treatment. The type of chemotherapy depends on a number of factors. This includes what previous treatments your child had and any side effects they had before.

Targeted radiotherapy is another treatment your child’s doctor might suggest. Targeted radiotherapy is similar to the mIBG scan used to diagnose neuroblastoma. As a treatment your child will have a higher dose of radioactive iodine than they did with the scan. The cancer cells pick up the mIBG and die.

Some children might go on to have other treatments such as:

  • an immunotherapy drug such as dinutuximab beta

  • 13-cis-retinoic acid (isotretinoin) – this is a substance related to vitamin A

Your doctor might suggest your child has treatment as part of a clinical trial. This is likely to be trying newer treatments.

Clinical trials

Researchers are looking into different ways of treating relapsed or refractory neuroblastoma. Below is an overview of some this research going on.

Comparing chemotherapy with molecular radiotherapy

There is no standard treatment for children whose cancer continues to grow and does not respond (refractory disease) to their first treatment (induction treatment). Doctors would like to know which treatment or treatment combinations, work best in these situations. So one research project is comparing two different types of intensive treatment. These are:

  • high amounts of thiotepa – a type of chemotherapy

  • 131-I-mIBG (a type of molecular radiotherapy) with topotecan (a type of chemotherapy)

Find out more about this trial

Targeted cancer drugs and immunotherapy treatment

Targeted cancer drugs work by targeting the differences in cancer cells that help them to grow and survive. Immunotherapy drugs help the immune system to attack the cancer.

Bevacizumab

Bevacizumab is a type of targeted cancer drug called a monoclonal antibody. It targets the tumour’s blood supply. By cutting off the tumour’s blood supply the cancer cells will die. Researchers have been looking at this drug alongside chemotherapy drugs. They recommend that children with relapsed or refractory neuroblastoma have bevacizumab alongside irinotecan and temozolomide.

Another possible combination is topotecan and cyclophosphamide, or topotecan and temozolomide.

Targeted cancer drugs

Doctors are looking into other targeted cancer drugs. They are looking at them on their own or with chemotherapy. Some of these drugs include:

  • venetoclax

  • lorlatinib

  • idasanutlin

  • LY3295668 or Erbumine

  • entrectinib

Other treatments

Researchers have been looking at a new drug called pegylated recombinant human arginase (BCT-100). 

Arginine is an important protein that helps cancer cells grow and divide. BCT-100 is a drug they can give to lower the amount of arginine. By doing this doctors hope this will help starve cancer cells and stop them from growing.

The trial is no longer recruiting patients. You can find out more about this trial and the results when they become available on our clinical trials database. 

Go to clinical trials database

How to join a clinical trial

Your child’s specialist doctor is the best person to talk to about your child’s treatment. This includes talking to them about joining a clinical trial. 

You can find a clinical trial on our clinical trials database.

Go to information about this trial

This page is due for review. We will update this as soon as possible.

Last reviewed: 31 Aug 2022

Next review due: 31 Aug 2025

Neuroblastoma main page

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.

Treatments for neuroblastoma

Surgery, chemotherapy, immunotherapy and radiotherapy are the main treatments for neuroblastoma. Find out how doctors work out your child's treatment.

Research and clinical trials

Researchers are looking at improving the diagnosis and treatment of neuroblastoma. Find out about the research that is happening.

Coping with neuroblastoma

Get information on organisations and resources that can help you and your family cope with a diagnosis of neuroblastoma.

My child has just been diagnosed with cancer

Find out about what to expect when your child is first diagnosed.

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