Research into children’s brain tumours

Researchers are interested in the diagnosis and treatment of brain tumours. There are a number of clinical trials for people to join in the UK. And many children and young people with brain tumours 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.

Here is some of the research that is happening in the UK.

Diagnosing brain tumours

Getting diagnosed

Researchers are looking into how to diagnose brain tumours earlier and more accurately.

HeadSmart is a campaign providing health professionals and the public with up to date information on:

  • the symptoms of brain tumours
  • what investigations or scans to do

The average time for a child to be diagnosed with a brain tumour has fallen from over 14 weeks to just under 7 weeks. Doctors and researchers want to continue to reduce this time.

Researchers are also interested in the ways teenagers and young people are diagnosed with cancer. The aim of the study is to identify possible delays in diagnosing cancer in teenagers and young adults. Finding any delays and the cause of delays will help to improve the way we look after teenagers and young adults with cancer in the future.


A team are looking into improving MRI scanning techniques to help diagnose brain tumours in children and young people.

A magnetic resonance imaging (MRI) scan is one of the tests children and young people have to diagnose and check how well treatment is working. This is for many types of cancer including brain tumours. This scan uses magnetism to build up a picture of the inside of the body. It can tell the doctors where in the body the tumour is and how big it is.

Functional imaging is similar to a MRI scan but uses a number of techniques. Some studies suggest that functional imaging may be able to give information about the tumour, how quickly it is growing and whether certain treatments will work. This study is aiming to find out more.

Inside brain tumour cells

Cancer cells have changes in their genes that make them different from normal cells. These changes mean that they behave differently.

Researchers are looking at the genes of thousands of brain tumours to pinpoint the mistakes that help them grow. They are also looking at how healthy brain cells develop. Other scientists are looking at how cancer cells and normal brain cells communicate and interact. This research is still in the laboratory.

In the future, scientists want to make new treatments by matching them to the biology of a child’s specific type of brain tumour.

You can watch a video about how Cancer Research UK is funding research into the biology of brain tumours here.

Researching treatment for children’s brain tumours

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 are looking at how the amount of information you and your child have before surgery affects the time in hospital and your experience of it. This might include:

  • more information about what the experience might be like
  • how long the team expect your child to stay in hospital
  • input from a dedicated discharge coordination team

Other research is being carried out to find out why some children have problems with speech and emotional problems after brain surgery. So, in the future we can work to prevent this.

Improving treatment with palladium

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 called an embryonal tumour. Treatment is usually:

  • surgery
  • radiotherapy
  • chemotherapy

An international clinical trial is looking at whether:

  • using the chemotherapy drug carboplatin with radiotherapy helps some children with medulloblastoma
  • children need the chemotherapy drug vincristine during radiotherapy
  • they can reduce the amount of chemotherapy for some children
  • children who at lower risk of their medulloblastoma coming back can have a lower dose of radiotherapy to the brain and spine

Doctors usually treat ependymoma by removing it with surgery. Afterwards your child might have radiotherapy to where the tumour was. But there is still a risk the tumour might come back in the same place in the brain or spine.

A trial team is looking at:

  • how well chemotherapy after surgery and radiotherapy works to stop ependymoma coming back
  • about the side effects of having chemotherapy after surgery and radiotherapy
  • how safe it is to give chemotherapy after surgery and radiotherapy

The standard treatment for diffuse intrinsic pontine glioma is radiotherapy. Unfortunately, for many children, radiotherapy is not a cure and the tumour is likely to come back within a year.

Researchers think that giving a targeted cancer drug alongside standard radiotherapy might be better. Targeted cancer drugs work by ‘targeting’ those differences that help a cancer cell to survive and grow.

Some targeted cancer drugs work by blocking pathways in cancer cells that tell them to grow and divide. These are called cancer growth blockers. Certain substances (biomarkers) in the tissue of the tumour show that these pathways are active.

In one trial researchers take a small sample of your child’s tumour tissue to look for these biomarkers. Depending on which biomarkers are present your child has one of the following as well as radiotherapy:

  • erlotinib
  • everolimus
  • dasatinib

We have more information about these targeted cancer drugs.

Some children and young people’s high grade glioma has a larger number of receptors for a protein called epidermal growth factor (EGF).

A trial team are looking at a new cancer drug called ABT- 414 to see if this helps these children.

ABT- 414 is a type of drug called an antibody drug conjugate (ADC). One part of ADC targets cancer cells by attaching to the epidermal growth factor receptor (EGFR) on the tumour cells. The second part is a toxin that goes into the cancer cell and kills it.

The researchers want to find out how safe ABT- 414 is and how well it works for children and young people with high grade glioma.

Low grade gliomas are brain tumours that start in the glial cells of the brain. They are usually slow growing tumours.

Doctors are looking at a targeted cancer drug called nilotinib alongside the chemotherapy drug vinblastine. Nilotinib blocks a protein that stimulates cancer cells to grow. It’s already a possible treatment for people with a type of leukaemia called chronic myeloid leukaemia (CML).

Doctors think it might help children with low grade glioma.

Research into treatment for children with a brain tumour that has not gone away with treatment or come back

Researchers are looking at the targeted cancer drug pomalidomide as a treatment for children with brain tumours. It works by stopping blood vessel growth in cancer cells.

At the moment, it is a possible treatment for adults with myeloma that got worse despite having had treatment with at least two other drugs. Researchers want to see if it helps children with brain tumours that have not gone away with treatment or have come back.

Another group of researchers are looking at the targeted cancer drug dabrafenib. Dabrafenib is a cancer growth blocker. It stops signals that cancer cells use to divide and grow. It’s only likely to work if the brain tumour cells have changes to the BRAF V600 gene. The change to the gene cause it to make an overactive BRAF protein. This makes cells grow and divide too fast.

It’s sometimes used to treat a cancer called melanoma in adults. Researchers want to find out if it’s helpful for some children with a brain tumour that has not gone away with treatment or has come back. And what the side effects are. 

Researchers are looking at other targeted cancer drugs in UK clinical trials too. These include:

  • larotrectinib
  • trametinib

Side effects and quality of life

As well as looking at how well treatments work, researchers are also looking into the side effects of treatment. And the quality of life for children with brain tumours.


BRIAN (the Brain tumouR Information and Analysis Network) is a new way for those affected by a brain tumour to learn from each other's experiences.

It’s been designed by the Brain Tumour Charity. It securely and anonymously stores data about people's treatment, tumour types, experiences, side effects and decisions. The team behind BRIAN want to learn on a large scale about what people living with a brain tumour have been through. Parents can sign up on behalf of their children.

Research into longer term 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.

How to join a clinical trial

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 or find out an answer for, any specific questions you or your child might have.

For information about clinical trials you can also call the Cancer Research UK nurses on freephone 0808 800 4040. They are available to help Monday to Friday, 9am to 5pm.

You can find more information about clinical trials into diagnosing and treating children’s brain tumours on our clinical trials database.

Last reviewed: 
13 Mar 2019
  • Sympathetic nervous system regulation of the tumour microenvironment

    SW Cole and others

    Nature Reviews Cancer, 2015. Volume 15.

  • Cancer Research UK Clinical Trials Database

    Accessed September 2018

  • Clinical Trials Database

    Accessed September 2018

  • Malignant Brain Tumours in Children: Present and Future Perspectives

    JT Rutka and others

    Journal of the Korean Neurosurgical Society, 2018

    Volume 61, Issue 3

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