Brain tumours in children
All cancer treatments must be fully researched before they can be used for everyone. 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 and your child may need to travel quite far if you take part in these trials.
Go to Cancer Research UK's clinical trials database if you are looking for a trial for children with brain tumours 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 children’s brain tumours.
Cancer cells have changes in their that make them different from normal cells. These changes mean that they behave differently.
Researchers want to learn more about genetic changes in children’s cancers including brain tumours. They hope by learning and understanding more about the gene changes in children’s cancers they can:
understand how genetic changes cause cancer in children
identify possible treatments
work out if treatment is working or not
replace taking samples (biopsies) with blood tests. They’ll compare the genetic information from a biopsy with a blood test
use this information to help design future clinical trials
Cancer Research UK and Blood Cancer UK have set up a national UK biobank called the VIVO Biobank. With the permission of patients and their families, the biobank collects and stores samples of tissue, blood or . Your child’s doctor takes these samples when they take other samples as part of your child’s routine care.
The aim of the VIVO biobank is to provide samples to help researchers understand more about the cancer and its treatment.
Find out more about the VIVO biobank
Researchers are looking at improving magnetic resonance imaging (MRI) scanning techniques. This is to help diagnose brain tumours in children and young people.
Earlier research suggests that new MRI techniques can give more information about:
the tumour such as the type
how quickly it is growing
whether certain treatments will work
Researchers are looking at how to improve treatment for children’s brain tumours. This includes improving treatments we already use. And seeing if new treatments work better than those we already have.
Researchers want to find out why some children have speech and emotional problems after surgery. This is for children who have had surgery to tumours at the back of the brain, called the posterior fossa area. Finding the cause can help the team find ways to prevent it in the future.
Other researchers are looking at:
improving MRI scans during surgery to help prevent problems after surgery
a pink drink that helps surgeons see and remove all of the brain tumour
A team is studying the potential of putting palladium beads into the brain during surgery. Palladium is a type of metal. The researchers think that this might help target the chemotherapy to the brain tumour. The palladium beads start the chemotherapy working once it reaches the tumour. So after surgery, people would have a drug which is only activated by the palladium beads.
This is early stage research in the laboratory. But doctors hope in the future people might be able to have higher doses of chemotherapy after surgery.
Medulloblastoma is a type of brain tumour. Children usually have surgery, radiotherapy and chemotherapy treatment.
Several trials are looking at the treatment for medulloblastoma. Researchers want to:
test different chemotherapy drugs that children have during radiotherapy
find out if some children can have less treatment
compare different treatments after surgery for children with high risk tumours
Doctors usually treat ependymoma with surgery. Afterwards your child might have radiotherapy and chemotherapy.
Researchers are:
adding chemotherapy after surgery and radiotherapy to see how well it works
testing chemotherapy before radiotherapy when surgery cannot remove all the tumour
adding valproic acid to chemotherapy to see if it improves treatment for those who cannot have radiotherapy
Radiotherapy is the standard treatment for diffuse midline glioma. Sadly, for many children, radiotherapy isn’t a cure and the tumour is likely to come back within a year.
Researchers think it might be better to give a alongside standard radiotherapy. Targeted cancer drugs work by targeting the differences that help a cancer cell to survive and grow. Researchers are looking at several targeted drugs including:
erlotinib
everolimus
dasatinib
Germ cell tumours are a rare type of brain tumour. There are many different types of germ cell tumour.
Doctors use surgery, chemotherapy and radiotherapy to treat germ cell tumours. Researchers want to learn more about the best ways to treat the different types.
One type of germ cell tumour is called a germinoma. Researchers are looking at how much chemotherapy children with germinoma have before radiotherapy. They want to know whether one chemotherapy drug before radiotherapy works as well as 3 drugs.
As part of this study children receive either carboplatin or vinblastine.
Doctors are always looking at ways to improve treatment for brain tumours that have not gone away (refractory) or have come back (relapsed).
Immunotherapy treatment uses our immune system to fight cancer. It works by helping the recognise and attack cancer cells.
Early research is looking at an immunotherapy called pembrolizumab. Doctors use this drug to treat a number of different cancers in adults. Researchers want to see if this treatment can help children and teenagers with advanced melanoma, lymphoma or certain solid tumours such as brain tumours. Your child’s cancer must make a protein called PD-L1. Their doctor will test for this if they think it’s an option for them.
Targeted cancer drugs work by targeting the differences that help a cancer cell to grow and divide.
Researchers want to improve treatment for people with rare cancers such as brain tumours. They are looking at existing targeted treatments that are already approved treatments for some types of cancer.
The targeted treatment your child has, depends on the gene changes in their cancer cells. Their doctor looks at a sample of their cancer tissue to find this out. This can then help the researchers decide which treatment is most suitable for them.
Researchers are looking for new treatments for children’s cancers when the stops or doesn’t work. Researchers for the eSMART trial are looking at a few different combinations of treatment. Which treatment your child has depends on the changes (mutations) found in the of the sample of cancer tissue. Your child would have one or more of the following:
targeted cancer drugs
immunotherapy
chemotherapy
This trial is looking at different children’s cancer types, including brain tumours.
Find out more about the eSMART trial
Researchers are looking at other targeted cancer drugs in UK clinical trials too. These include:
larotrectinib
tovorafenib (known as DAY101)
As well as looking at how well treatments work, researchers are also looking into the:
side effects of treatment
for children with brain tumours
Researchers have been looking at ways to support children and young people after treatment for a brain tumour. They want to understand, improve and support children and young people’s quality of life after treatment for a brain tumour.
The Centre for Childhood Survivor Studies is looking at the long-term side effects of all children’s cancers. They have split the study into different age groups.
The British Childhood Cancer Survivor Study (BCCSS) includes children who were diagnosed before the age of 15, between 1940 and 2006. Participants survived for at least 5 years after diagnosis and live in England, Wales and Scotland.
The Teenage and Young Adult Cancer Survivor study (TYACSS) includes people aged between 15 and 39 years who were diagnosed with cancer between 1971 and 2006. Participants have survived for at least 5 years after their diagnosis and live in England and Wales.
This type of research takes many years to produce results. Researchers need to follow up children, teenagers and young people throughout their lives. You can check their website for updates and to read any published results.
Go to The Centre for Childhood Cancer Survivor Studies
Cancer Research UK and the National Cancer Institute in the United States have set up Cancer Grand Challenges programme. This unites cancer research teams around the world. It is to take on some of cancer’s toughest challenges.
The Cancer Grand Challenges has funded researchers across the world to work together and look into children’s brain tumours. They will be looking at:
developing new targeted treatments
immunotherapy as a treatment for children’s cancers
Find out more about the Cancer Grand Challenges
Cancer Research UK is supporting research into childhood cancer, including brain tumours. You can read more about how Cancer Research UK is supporting research.
Find out more in about the progress made so far
The best person to speak to about your child joining a clinical trial is their hospital consultant.
There might also be a research nurse at their primary treatment centre you can talk to. Or you could chat through the options with your child’s clinical nurse specialist. They will try to answer any specific questions you or your child might have.
Last reviewed: 09 Nov 2026
Next review due: 09 Nov 2029
The main treatments for children’s brain and spinal cord tumours are surgery, radiotherapy and chemotherapy.
Brain tumours and their treatment can cause physical and mental changes. Understanding about what they might be can help you cope.
It is essential that parents and other close family have support. Find out what is availble and who can help.
Brain tumours are the second most common type of children’s cancer in the UK. Around 420 children are diagnosed with these tumours each year in the UK.
