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Brain tumour research

All treatments must be fully researched before they can be adopted as standard treatment for everyone. This is so that we can be sure they work better than the treatments we already use. And so we know that they are safe.

First of all, treatments are developed and tested in laboratories. Only after we know that they are likely to be safe to test are they tested in people, in clinical trials. Cancer Research UK supports a lot of UK laboratory research into cancer and also supports many clinical trials.

There is research into the causes and diagnosis of brain tumours. There is a lot of research into treatment for brain tumours, including into treating children.


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Why we need research

All treatments have to be fully researched before they can be adopted as standard treatment for everyone. This is so that

  • We can be sure they work
  • We can be sure they work better than the treatments that are available at the moment
  • They are known to be safe

First of all, treatments are developed and tested in laboratories. For ethical and safety reasons, experimental treatments must be tested in the laboratory before they can be tried in patients. If a treatment described here is said to be at the laboratory stage of research, it is not ready for patients and is not available either within or outside the NHS. Cancer Research UK supports a lot of UK laboratory research into cancer.


Clinical trials

Tests in patients are called clinical trials. Cancer Research UK supports many UK and international clinical trials.The trials and research section has information about what trials are including information about the 4 phases of clinical trials. If you are interested in taking part in a clinical trial, go to our searchable database of clinical trials. If there is a trial you are interested in, print it off and take it to your own specialist. If the trial is suitable for you, your doctor will need to make the referral to the research team.

All the new approaches covered here are the subject of ongoing research. Until studies are completed and new effective treatments are found, these treatments cannot be used as standard therapy for brain tumours.

Here is a video on what it's like to take part in a clinical trial:

View a transcript of the video (Opens in a new window)


Causes of brain tumours

Cancer Research UK scientists are looking into the differences between the genes in brain tumour cells and normal cells. They have already found some common gene faults and are hoping that investigating these will lead to new ways of diagnosing and treating brain tumours. 

There is a study looking at gene changes in pituitary tumours. This trial is trying to find genes that may be important in the development of pituitary gland tumours. 

The UK CYAB study is a study of children and young people up to 24 years old, looking at factors such as diet, lifestyle, and history of infections, to try to find what might cause brain tumours in this age group. The researchers have completed the pilot study. From this, they feel that it will be possible to plan and run a much larger study looking at the causes of brain tumours.

You can find out more about these trials for brain tumours on our clinical trials database


Diagnosing brain tumours

Research is looking into improving scans and diagnosis. These include some new types of scan.

One recent trial tested a new type of MRI scan called DTI (Diffusor Tension Imaging). It is also sometimes called tractography. Standard MRI can’t show up important bundles of white nerve fibres. Damage to these white nerve bundles during surgery can have serious effects. Surgeons have to find out where these nerves are and if they are surrounded by the tumour. They can then avoid cutting through the nerve bundles. 

The research team found that DTI scans are useful for getting a more detailed picture of the brain than other types of scan. They also found that they could see the difference between nerve bundles, normal tissue and areas of tumour more clearly. The researchers say that it is early days for this new technique, but it may be useful for planning treatment in the future.

The only sure way to diagnose many brain tumours is to have a biopsy done. Looking at the cells under a microscope is the only guaranteed way to identify the type and grade of the tumour. The CNS 2004 10 trial is looking at a range of scanning techniques to help diagnose, treat and understand cancer in children and young people (aged 25 or below). 

The advanced MRI scan trial is looking at whether specialised MRI scans can help to decide how and when to treat brain tumours. The new types of MRI scan give information about the size and shape of the tumour. They also show up the blood flow in the brain as well as which chemicals are in the brain, and how they move around.

A study looking at how high grade gliomas behave is also using MRI scans. The researchers hope this study will further develop MRI scans as a tool in deciding treatment for gliomas.

Another study is looking at using MRI scans to see how low grade glioma brain tumours change over time. The researchers aim to combine different ways of measuring glioma growth using the MRI scan.

Another study is looking at using a combination of imaging methods to better assess brain tumours. The researchers are combining a PET scan with an MRI scan to assess gliomas in people who are going to have surgery. The researchers think that the combination of these scans might help show up certain features of the brain tumour such as how fast it is growing.


Research into surgery for brain tumours

The GALA 5 trial is looking at 2 treatments for glioblastoma. The first treatment is 5-ALA or Gliolan. It is a dye that makes brain tumour cells glow red under ultra violet light. During surgery, it can help surgeons to check they are removing as much brain tumour as possible. The second part of the treatment is a chemotherapy implant called a Gliadel wafer that the surgeon puts into the area of the brain tumour. The aim of this treatment is to kill any tumour cells left after surgery.

The researchers want to see if it is safe to have 5-ALA and Gliadel wafers with standard brain tumour treatments. They also want to find out how many people are helped by this treatment and whether it has an effect on other treatments they have. 

The GALA-BIDD study is looking to see if the drug Gliolan can help surgeons see what grade a brain tumour is during surgery. The researchers hope the results of the study will help improve treatment for people with brain tumours in the future.

Treatment for low grade brain tumours can be challenging for doctors as the tumours can be present for years without causing symptoms. Some doctors prefer to closely monitor the tumours and offer surgery if the tumour grows or becomes more aggressive (watch and wait approach). But other doctors prefer to do surgery to remove the tumour early on, if possible.

A Norwegian study in 2012 compared the different ways of treating low grade gliomas at 2 hospitals. One hospital usually offered people with low grade gliomas surgery early on, whereas doctors at the other hospital preferred to monitor the tumours. Patients at the hospital that usually carried out early surgery lived longer than at the hospital that usually monitored tumours. Further follow up of these patients, and other studies, are needed to confirm these results and help doctors decide on the best treatment for some people with low grade brain tumours.


Radiotherapy research for brain tumours

The radiotherapy trials for brain tumours mentioned here are listed on our searchable clinical trials database. You will need to tick the boxes if you want to see the trials that have finished recruiting or that have results. Many of the trials are supported by Cancer Research UK.

Researchers are looking into the following

Stereotactic radiotherapy and radiosurgery

Stereotactic radiotherapy is a technique that gives very accurate and high doses of radiation directly into the centre of the brain tumour without damaging the normal surrounding brain tissue. A variation on this is called radiosurgery, which is a single, highly targeted dose of stereotactic radiotherapy. There is detailed information about stereotactic radiotherapy and radiosurgery in this section of the website.

Both stereotactic radiotherapy and radiosurgery are still being researched to find out exactly which tumours they can treat. They are mainly used for brain tumours that have come back or for other cancers that have spread to the brain. They are used for some non cancerous (benign) tumours of the brain, such as vestibular schwannoma.

This type of treatment is only suitable for a small number of brain tumours and for particular circumstances. It is less likely to be suitable for larger tumours or for widespread tumours. A number of clinical trials are in progress in the USA and other parts of the world. When the results from these are available, specialists will know more about which tumours these treatments can help.

Boron neutron capture therapy (BNCT)

Boron neutron capture therapy (BNCT) is an experimental way of giving radiotherapy. It has been used in trials with glioblastoma multiforme (grade 4 glioma), which is a type of brain tumour. Researchers are looking into using this treatment after surgery to try to reduce the risk of the brain tumours coming back. It has also been used for some types of advanced head and neck cancers. 

With BCNT, boron is injected into the bloodstream and collects in tumour cells. The next stage of the treatment is external radiotherapy with low energy neutrons. The boron molecules give off radiation within the brain tumour cells when the external neutron radiation hits them. This increases the radiation dose by about 2 or 3 times. So, a lower dose of neutron radiotherapy can be given. This makes it less likely that the neutrons will cause damage. However, there are concerns that the treatment may cause severe side effects, so trials are continuing in Europe, the USA and Asia. There are no trials for patients going on in the UK at the moment. 

There is an early phase trial in the USA for people with newly diagnosed glioblastoma multiforme. It is testing BNCT with a chemotherapy drug called temozolomide. Early phase trials look at side effects, doses and whether there seems to be a response for a particular cancer. It is only in later phase 3 trials that we can see if a new treatment works any better than an existing treatment. So it will be some years before we have any long term results for BNCT.

Proton therapy

One of the newer ways of giving radiotherapy uses a different type of beam called a proton beam. Protons collect energy as they slow down and travel through the body. They then release this energy at their target point – the tumour. This means they give a very high dose of radiation to the cancer, but only a low dose to the area around it. So there is less likelihood of damage to nearby healthy tissue.

Proton beam radiotherapy machines in the UK are only able to treat cancer of the eye. The machines are not able to treat cancers deeper in the body. Some countries in Europe and the USA are testing and using proton beam radiation for deeper cancers, including a tumour called chordoma that can occur in the spinal cord or the brain stem. At the moment, if this treatment is suitable for you the NHS pays to send you for treatment abroad – usually to the USA.

Hyperfractionated radiotherapy (HFRT) 

The HIT - SIOP PNET 4 trial compared hyperfractionated radiotherapy (with 2 treatments a day) to standard radiotherapy (one treatment a day). It recruited children and young people from the age of 4 to 21 with medulloblastoma. The trial team found that HFRT was no better than standard radiotherapy as part of treatment for medulloblastoma. The researchers also found no significant difference between the side effects of the two treatments.

Side effects of radiotherapy

Radiotherapy treatment is planned very carefully and techniques such as intensity modulated radiotherapy (IMRT) can shape radiation beams to treat the cancer, while avoiding healthy tissue. Although this makes it very precise, radiotherapy can still cause damage to some healthy tissue. The VoxTox study is looking at the side effects of radiotherapy for cancers of the central nervous system and some other types of cancer. The researchers want to understand more about the side effects and to work out how much radiation has reached healthy tissue. The information the researchers collect may help to reduce side effects for people having treatment in the future.


Chemotherapy research for brain tumours

There are always clinical trials testing new combinations or single chemotherapy drugs for brain tumours. Trials also look at drug doses and the order in which you have them (the sequence).  

Many of the chemotherapy trials for brain tumours are listed on our clinical trials database. To find them, go to the trials database search page.

Current research is looking at 


Temozolomide chemotherapy is a possible treatment for newly diagnosed glioblastoma multiforme. 

The BR14 trial is looking at radiotherapy with and without temozolomide for anaplastic glioma. Anaplastic means that the cancer cells look less like normal glial cells than other gliomas. Anaplastic cancers often grow more quickly than other cancers. In this trial, some people won’t have temozolomide. Some will have temozolomide during radiotherapy and some will have it after radiotherapy. Some will have it both during and after radiotherapy. This trial has now closed and we are waiting for the results.

Some people with a brain tumour have changes in two chromosomes called 1p and 19q in the cell. One or both must be normal in everyone taking part in this trial. Doctors call this non 1p19q deleted anaplastic glioma.The aims of this trial are to find out if temozolomide during radiotherapy, after radiotherapy, or during and after radiotherapy is better than radiotherapy alone. And they want to learn more about the side effects. 

Chemotherapy for primary lymphoma of the central nervous system (CNS)

Primary CNS lymphoma is a very rare form of non Hodgkin lymphoma which usually only affects the brain. The first treatment for it is usually the chemotherapy drug methotrexate. Doctors think higher doses of methotrexate would work better, but it has serious side effects, including kidney damage and a reduction in blood cell counts. A drug called glucarpidase helps your body to break down methotrexate quickly and get rid of it. 

Doctors hope that glucarpidase will protect people from the worst of the side effects of methotrexate, and allow them to give higher doses to fight the lymphoma. A small trial of this treatment is looking to see how safe it is to give higher doses of methotrexate with glucarpidase, and what the side effects will be.

If CNS lymphoma does not respond to chemotherapy or comes back after treatment, there is currently no standard treatment that doctors use. So they are researching new combinations of treatment. The TIER trial is looking at the chemotherapy drugs thiotepa, ifosfamide and etoposide, and a type of biological therapy called rituximab. The aim of the trial is to find out the best dose of thiotepa and to see how well this combination of treatment works.


Biological therapy research for brain tumours

Biological therapies are treatments that use natural substances from the body, or drugs made from these substances to treat cancer. Research is looking into 

Cancer growth blockers

Some biological therapy drugs called tyrosine kinase inhibitors (TKIs) block signals that tell cancer cells to divide and grow. Imatinib (Glivec) is a TKI drug that works well in treating a type of cancer called gastro intestinal stromal tumour (GIST). Researchers have tried this drug for glioma brain tumours. But the results so far have been mixed, and further trials are needed to see if imatinib is helpful for brain tumours.

Another TKI drug being tested is erlotinib (Tarceva). The Phase 1 NAG 2005 09 trial looked at how well this might work for children whose brain tumours have continued to grow, or have come back after treatment, and for children newly diagnosed with brain stem gliomas, which are very difficult to treat. They found that erlotinib was safe and were able to find the best dose. As this was a phase 1 trial more trials are now needed. 

The DORIC trial is comparing a combination of cediranib and gefitinib (Iressa) with cediranib alone in people who have glioblastoma that has come back after treatment. Cediranib is a type of biological therapy that stops the cancer growing new blood vessels (see the section on blood supply and brain cancer below). Gefitinib is a tyrosine kinase inhibitor. The aim of this trial is to find out if the combination of cediranib and gefitinib works better than cediranib alone for glioblastoma that has come back after treatment. This trial has now closed to recruitment and we await the results. 

PARP inhibitors

PARP is an enzyme that helps damaged cells to repair themselves. If PARP is blocked, cancer cells may not be able to repair themselves after chemotherapy. This could make chemotherapy work better. Olaparib is a PARP inhibitor being looked at in early studies for different types of cancer, including glioblastoma.

There is a phase 1 trial looking at olaparib with temozolamide for glioblastoma that has come back after treatment. The first aim of the trial is to see if olaparib is able to cross the protective blood brain barrier. If it does, then the researchers hope to find the best safe dose of olaparib to have with temozolamide. And to learn more about the side effects of having both drugs together. 

An early trial called PARADIGM is looking at olaparib with radiotherapy for people with glioblastoma who cannot have standard chemotherapy and radiotherapy. This includes people over the age of 70 and those who have poor health. These people often have radiotherapy alone. Olaparib may help radiotherapy to work better. The first part of this trial is to find the best dose of olaparib to have with radiotherapy. The second part is to see if the treatment helps people to live longer and to learn more about the side effects.

Blood supply and brain cancer

Angiogenesis means growth of new blood vessels. A cancer needs to grow its own blood vessels as it gets bigger. Without its own blood supply, it can't continue to grow. Anti angiogenic drugs stop tumours from developing their own blood vessels. This then deprives the cancer of the oxygen and food that it needs. Research using these drugs is ongoing for brain cancers. The drug being tried most in trials for brain cancer is thalidomide. Thalidomide hit the headlines in the 1960s when it was discovered that it caused birth defects by reducing the blood flow to developing limbs in babies in the womb. Since then it has been given to many people with cancer, without causing serious side effects. It is still important not to take this drug if there is any possibility you could be pregnant. Early studies show some promise for treating people with high grade gliomas. But we will need a lot more research to develop this further as a treatment.

The phase 1 EORTC 26054 trial looked at temozolomide with an anti angiogenic drug called enzastaurin, for people with advanced glioma, or newly diagnosed glioma that could not be treated with radiotherapy. The trial recruited 28 people in total. The trial team found that temozolomide and enzastaurin was a safe combination. The trial was too small to draw any firm conclusions about how well the treatment works but the researchers felt it could be a useful combination to treat glioma. But further trials are needed.

Monoclonal antibodies

Many different monoclonal antibodies (MABs) are being investigated for cancer treatment. MABs are proteins, made in the laboratory from a single copy of a human antibody. When these laboratory made antibodies are injected into patients they seek out cancer cells which carry abnormal proteins. 

Researchers are looking at a MAB called bevacizumab (Avastin) for high grade gliomas. There have been studies looking at it on its own, and in combination with chemotherapy or radiotherapy for glioblastoma that has come back. There is some evidence that shows it may increase the length of time before glioblastoma starts to grow again. But more studies are needed to find out about the side effects and how they affect people's quality of life, and to find out if it is better to have this type of drug on its own or with other treatment.

Some researchers are looking at bevacizumab after surgery for people with newly diagnosed glioblastoma. The HERBY trial is looking at adding bevacizumab to radiotherapy and chemotherapy after surgery for children with a high grade glioma.

Researchers are also looking to see if bevacizumab is helpful in treating grade 2 and 3 gliomas. The TAVAREC trial is comparing temozolomide and bevacizumab with temozolomide alone in people with grade 2 or 3 glioma that has come back after treatment. This trial has now closed and we are waiting for the results.

Nivolumab is another type of MAB. It works by blocking a body substance called PD-1. This may help the immune system to work against brain tumour cells. A trial is comparing nivolumab with bevacizumab for glioblastoma that has come back.

Researchers are looking at new types of drugs called antibody drug conjugates (ADC's). There are 2 parts to these drugs. The first part targets cancer cells by attaching to a particular receptor on the surface of the cancer cell. The second part is a toxin that gets into the cancer cell and kills it. ABT-414 is one of these drugs. It attaches to the epidermal growth factor receptors (EGFR) on cancer cells. An earlier study suggested that people whose glioblastoma have a large number of EGF receptors may benefit from ABT-414. The INTELLANCE 2 trial is comparing ABT-414 with or without temozolomide and standard treatment for glioblastoma that has come back.

Gene therapy

A number of new gene therapy treatments are being researched. Some doctors and researchers are now using the term molecular therapy which is a more general term. It can include research into oncogenes, tumour suppressor genes, gene therapy and repair genes. You can read about these genes in more detail

By studying how changes in these genes cause normal cells in the brain to become cancerous, scientists aim to eventually develop gene therapy where damaged genes in the cancer cells can be replaced with normal ones.

Doctors have started to look into a gene therapy called cerepro. One trial used a variation of the herpes simplex virus to deliver gene therapy to people with high grade glioma that has come back or started to grow again since treatment. Another trial has looked at using cerepro after surgery. There are no trials looking at cerepro at the moment. But the company that make it hope to continue with further research in the future.


Cancer vaccines are an experimental treatment that helps the immune system kill cancer cells. A phase 1 trial looked at a new vaccine called IMA950 for glioblastoma. The people taking part also had a drug called GM-CSF. This is a growth factor which helps to stimulate the immune system so it is more likely to respond to the vaccine. The trial team found that the vaccine was safe. Because enough people showed a good immune response the researchers feel that the vaccine should be developed further. You can read the results of the IMA950 trial on our clinical trial database.

Another trial is looking at a personalised vaccine called DCVax-L for glioblastoma multiforme. The dendritic cells are mixed with proteins taken from your brain tumour to make the dendritic cell vaccine. It works by helping your immune system recognise and kill cancer cells.

A phase 3 trial called ACT IV is looking at a vaccine called rindopepimut for people with glioblastoma. Between a quarter and a third of all glioblastomas may contain a protein called EGFRvIII. Researchers want to see if rindopepimut can train the immune system to recognise this protein and kill the glioblastoma cells. This trial has closed and we are waiting for the results.

If you would like to look for current brain tumour trials, you can look on our clinical trials database.



Reolysin is a cancer treatment made from a type of virus called reovirus. Reovirus is common and causes only minor symptoms such as coughs, colds and diarrhoea. We know from research that Reolysin kills cancer cells. But we do not yet know if it can reach, or affect, cancer cells in the brain. The REO 13 BRAIN study is an early study looking at giving Reolysin before surgery for people with a primary brain tumour that has come back or who have cancer that has spread to the brain (secondary brain tumour). The researchers will look at the brain tumour tissue removed during surgery to see if the reovirus has affected the cancer cells. They will also monitor how your immune system responds to the virus.


Clomipramine research

Clomipramine is a drug used for depression, called a tricyclic anti depressant. Some researchers think it might help to treat glioma brain tumours. There has been some lab based research that showed the drug could encourage death of cancer cells, but not healthy brain cells. The researchers say this is encouraging.  

A lot more research is needed before we know whether clomipramine can help to treat people with gliomas. There have been no trials reported using clomipramine in people with brain tumours and we are not aware of any trials currently recruiting patients.


Tamoxifen for brain tumours

Tamoxifen is a type of hormone therapy. This drug is most often used to treat or prevent breast cancer. But it has also been tried for other cancers, including brain tumours. How it works on brain tumours is complex. One theory is that because this drug helps to protect against spread of the cancer to the brain for women with breast cancer, it will have a similar protective effect on people with certain types of brain tumours. Much higher doses are needed for people with brain tumours than for women with breast cancer. 

Researchers think that they need to get a large amount of tamoxifen into the brain in a short time. So they are investigating starting with small doses and increasing the dose fairly quickly. We still don’t know whether people should remain on these high doses or if they should have a lower maintenance dose. We also don’t know how long people with a brain tumour would need to carry on taking tamoxifen.

Generally, tamoxifen has few side effects, although it may have more in the high doses used for brain tumours. It can increase the risk of blood clots and people with brain tumours also have a higher risk of blood clots. So it needs to be used with care. There is some evidence to suggest that it should not be taken with the anti epileptic drug called phenytoin. Results from early clinical trials have shown that tamoxifen does not help everyone with a brain tumour but it may help a few. We’ll need larger clinical trials before we will know whether it can help to treat brain tumours.


Photodynamic therapy for brain tumours

Photodynamic therapy (PDT) is a type of treatment that makes cells light sensitive and then shines a very bright light onto them. The National Institute for Health and Care Excellence (NICE) has said that there is not yet enough evidence to recommend this as a standard treatment for brain tumours but it can be used as part of clinical trials. 

PDT has been tried for brain tumours that have come back after standard treatment. But a recent trial looked at PDT for newly diagnosed brain tumours. The people taking part in this trial had glioblastoma multiforme and had either surgery and radiotherapy, or surgery, PDT and radiotherapy. The researchers found that it took longer for the brain tumours to start growing again in people who had the PDT. In this group of patients, the researchers also found there was more of an improvement in how well they were and how many activities they were able to carry out 6 months after treatment. This was only a small trial of 27 people, and so the researchers suggest this treatment should be looked at in larger trials.

Another trial is comparing different laser wave lengths in PDT for the most common types of brain tumour (glioblastoma multiforme or anaplastic astrocytoma). This trial is looking at giving PDT after surgery for brain tumours. The aim is to kill any cells left behind. The trial team are comparing 2 different PDT laser wave lengths. One shines deeper into brain tissue than the other. Doctors want to know which wave length of laser is better at stopping brain tumours coming back after surgery. This trial has now closed to recruitment and we are waiting for the results. 


Electric field treatment

An experimental treatment that uses electric fields to disrupt and interfere with the division of cancer cells has been developed. It was used in the phase 3 EF-11 trial in the USA, Europe and Israel. The device is battery powered and gives low amplitude alternating electric fields through disposable electrodes stuck to the scalp. In the trial, 120 patients with glioblastoma that had come back after several treatments used the electrical field device for about 20 hours each day. This group was compared with a similar number of patients who received more chemotherapy for recurrent glioblastoma.

The trial seemed to show that the electrical field therapy may work as well as chemotherapy for glioblastoma that has come back after previous treatments. The researchers say that the electrical field therapy seems to be safe and the only side effects reported were skin irritation from the electrodes. However, some researchers were concerned about the design of the trial. We need more research into electric field treatment before we know how helpful it may be for brain tumours.



The main ingredients of the drug Sativex are tetrahydrocannabinol (THC) and cannabidiol (CBD). Both of these come from the cannabis (marijuana) plant. An early trial is looking at Sativex with temozolomide for glioblastoma multiforme that has come back after treatment. The researchers want to find out how well this combination of treatment works and what the side effects are. The trial has closed to recruitment and we are waiting for the results. 


Research into treatment effects on children

A study is looking to learn more about the experiences of people who had radiotherapy as a child or young person for medulloblastoma. By gaining a better understanding of the impact of treatment, doctors may find a way of reducing side effects in the future and improve the quality of life of children and young adults who've had treatment for this type of brain tumour.

The British Childhood Cancer Survivor Study is following children who’ve had successful cancer treatment. The researchers are looking at their health in the years following their cancer treatment to see how it has been affected. They are also monitoring their general quality of life, how they do in school, and whether or not they go on to have children of their own. It is important that we understand the long term effects of treatments so that we can improve them in years to come. If we can identify some of the risks, doctors may be able to take steps to prevent other health problems from developing.

Children who’ve been treated for other types of cancer in the past may have an increased risk of getting a brain or spinal cord tumour in the future. This type of study can help doctors to predict who is at risk. If they knew that, they may be able to take action to reduce the risk for these patients.

There is more about the potential effects of brain tumour treatment on children in this section of the website.

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Updated: 27 October 2015