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. Some of the trials mentioned may no longer be recruiting children but the researchers are still following them up and collecting the results.

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.

Scans

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 an 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 team of researchers are 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.

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.

Surgery

Researchers are looking at the amount of information you and your child have before surgery. And how this 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 wants to find out why some children who have tumours of the posterior fossa have problems with speech and emotional problems after brain surgery. So, in the future the treatment team 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

Another trial is looking at comparing treatments after surgery for high risk medulloblastoma. Researchers are looking at different treatments to try and find what the best treatment is and which has the least side effects.

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.

Doctors want to find new and kinder ways to treat ependymoma. And ways to reduce the side effects of treatment.

A trial called SIOP Ependymoma Program II is looking at 3 separate questions to try and improve treatment for ependymoma. They have split the questions into pathways 1, 2 and 3.

Pathway 1 is looking at radiotherapy and chemotherapy after surgery. Half the children and young people taking part will have just radiotherapy. The other half will have chemotherapy after radiotherapy.

Pathway 2 is looking at giving chemotherapy before radiotherapy. Doctors usually treat ependymoma by removing it with surgery. Afterwards they might give radiotherapy to where the tumour was. Researchers think by giving chemotherapy before radiotherapy it might help those whose tumour wasn’t completely removed by surgery.

The last pathway is 3. The researchers want to know if adding a drug called valproic acid to standard chemotherapy will help improve treatment for those that can’t have radiotherapy. We know from research that valproic acid can help people with other types of brain tumours.

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 targeted cancer drug alongside standard radiotherapy. Targeted cancer drugs work by ‘targeting’ the 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.

Researchers in France are looking at a new targeted cancer drug called ONC201 with radiotherapy compared to everolimus (a targeted drug) and radiotherapy. Researchers already know that everolimus and radiotherapy is better at treating diffuse midline glioma than radiotherapy alone. So now they want to see if ONC201 and radiotherapy is better for relapsed diffuse midline glioma.

Researchers are looking at a drug called pegylated recombinant human arginase (BCT-100). They want to see if it helps children with high grade glioma that has come back or didn’t go away with treatment.  Arginine is a protein (an amino acid) that’s important for cells to grow and survive. Normally, healthy cells can make arginine using a protein (an enzyme) that is often missing in cancer cells. If the amount of arginine available is reduced, it starves the cancer cells and stops them from growing.

Healthy cells are able to survive much better than cancer cells when there is less arginine available. This is partly because they can make it. So, by reducing the amount of arginine available the cancer might stop growing. The researchers hope that by giving this drug it will lower the amount of arginine available and stop the cancer from growing.

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

Clinical trials for brain tumours that have not gone away or have come back can open and close quickly. Talk to your child’s doctor as they will know of open trials that may be suitable for your child.   

Dabrafenib and trametinib

Other researchers are looking at the targeted cancer drugs dabrafenib and trametinib. Dabrafenib and trametinib are types of cancer growth blockers. They stop signals that cancer cells use to divide and grow. This combination is only likely to work if the brain tumour cells have changes to the BRAF gene. The change to the gene causes it to make an overactive BRAF protein. This makes cells grow and divide too fast.

Researchers want to find out if these drugs are helpful for some children with a brain tumour that has not gone away with treatment or has come back. Also they want to see if these drugs help those with a slow growing glioma (low grade). The other aims are to find out:

  • about the side effects of these drugs in children
  • how the drugs work in children
  • if children are able to take the liquid versions of these drugs if they can’t take the capsules 

eSMART

Researchers are looking for new treatments for children’s cancers when the standard treatment Open a glossary item 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 DNA of the sample of cancer tissue. Your child would have one or more of the following:

  • targeted cancer drugs
  • immunotherapy Open a glossary item
  • chemotherapy

This trial is looking at different children’s cancer types, including brain tumours.

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

  • larotrectinib
  • tovorafenib (known as DAY101)

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

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.

ACT NOW study

The ACT NOW study is a trial about a type of cognitive behavioural therapy (CBT). ACT stands for Acceptance and Commitment Therapy. It is a clinical trial that recruited children and young people aged 11 to 24 years of age who have completed cancer treatment for a brain tumour.

The trial was run through a video call. People taking part in this trial either have an appointment immediately or they wait 12 weeks. Each therapy session was an hour and lasts for a total of 12 weeks. The aim is to help understand, improve and support children and young people’s quality of life after treatment for a brain tumour.  

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.

Research into understanding more about children’s cancer and their treatment

The Children’s Cancer and Leukaemia Group (CCLG) have set up a tissue bank based in Newcastle. With the permission of patients and their families, they are collecting and storing samples of tissue left over from biopsies or they may have a small amount removed during surgery. In some cases they may also collect a small amount of blood. This is for any child or young person with a solid tumour or lymphoma.

The aim of this study is to help researchers understand more about the cancer and its treatment.

Germ cell tumours

Germ cell tumours develop in germ cells. These are the cells in the body that develop into sperm and eggs. Germ cell tumours most often develop in the ovary or testicle because this is where most germ cells are.

But germ cells can sometimes be left behind in other parts of the body from when you developed in the womb. So these tumours can develop anywhere in your body where there are germ cells. Germ cell tumours that start in the brain and spinal cord are very rare.

Doctors use surgery, chemotherapy and radiotherapy to treat germ cell tumours. But there are different types of germ cell tumours and doctors want to learn more about the best ways of treating different types.

The aim of this study is to collect information about germ cell tumours that start in the brain. 

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 any specific questions you or your child might have.

For general 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.

  • Cancer Research UK Clinical Trials Database
    Accessed December 2022

  • Clinicaltrials.gov
    Accessed December 2022

  • Childhood brain tumors: current management, biological insights, and future directions
    I Pollack, S Agnihotri and A Broniscer
    Journal of Neurosurgery, March 2019. Volume 23, Issue 3, Pages 275 to 282

  • Brain Tumours in children
    A Gajjar and others
    Springer, 2018

  • Oxford Textbook of Cancer in Children (7th Edition)
    H N Caron and others
    Oxford University Press, 2020

  • 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. Please contact patientinformation@cancer.org.uk with details of the particular issue you are interested in if you need additional references for this information.

Last reviewed: 
04 Jan 2023
Next review due: 
04 Jan 2026

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