what you will find on this page
Optimising treatment beyond standard care
For newly diagnosed glioblastoma (GBM), the most common type of malignant brain tumour, the best results usually come from combining several treatments. Research into GBM is ongoing, so there are also some additional options you might hear about.
IS THERE MORE?
Immunotherapy (in GBM) is promising but largely unproven in GBM
Cannabinoids and off-label drugs are widely discussed by patients but currently lack strong evidence
Emerging / Limited Evidence
Genomic Testing has growing evidence base and can guide targeted treatments
Moderate Evidence
Optune has robust trial data behind it
Strong Evidence
Evidence levels change over time. This spectrum reflects the best available information today, but research is ongoing — and evidence doesn’t always equal effectiveness for every individual.
- For a few of these options, there is good clinical evidence showing they can help certain patients.
- For others, the evidence is limited or mostly based on early research or personal experiences.
- New information and research are coming out all the time, so what’s known today may change in the future.
- Optune (a wearable device that uses electrical fields to target tumour cells)
- Immunotherapies (treatments that work with your immune system to fight the tumour)
- Genomic testing (looking closely at the tumour’s DNA to find targeted treatments)
- Cannabinoids (medicines made from the cannabis plant or similar compounds)
Tailored Medicine
Clinical Trials
PERSONALISED / EXPERIMENTAL
Sterotactic
Radiotherapy
Optune
Immunotherapy
ADVANCED OPTIONS
Surgery
Radiotherapy
Chemotherapy
STANDARD TREATMENT
- Surgery - to remove as much of the tumour as safely possible.
- Chemoradiotherapy - radiotherapy combined with the drug Temozolomide.
- Steroids - to help control swelling and reduce symptoms.
- Anti-epileptic drugs - to prevent or manage seizures.
If you want to understand the research behind Optune, you can read about the EF-14 trial here.
Read the trial evidence
Ask about financial support programmes and whether your health insurance might cover the costs.
Check funding options
Understand what’s involved in wearing the device every day, including scalp care and changing the pads.
Be prepared for the commitment
Click here to find doctors who can prescribe and support Optune use.
Find an experienced clinician
Visit www.optunesupport.uk for independent information and patient experiences.
Learn more
If you’re interested in Optune:
Optune is an innovative and proven treatment for glioblastoma when used alongside standard care. While it does require commitment and can be costly, it can be a valuable option for some people, especially if funding is available.
Mainly because of the high cost. Although it has proven survival benefits, the price is a barrier. The manufacturer is working to reduce costs, and some private insurance plans will cover it.
The device weighs about 2kg. Most people find it manageable once they get used to it, although wearing it for long periods can be tiring at first.
Offers practical tips, community support, access to support groups and detailed information about Optune for GBM (and other cancers).Disrupts cancers ability to divide and grow
www.optunesupport.uk
Online groups, email lists, and WhatsApp chats connect you with people who are also using Optune.
From other patients:
Optune’s team can help set up the device, teach you how to use it, and provide ongoing support.
From the company:
Commitment:
Optune needs to be worn for at least 18 hours a day. This means you’ll be connected to the device most of the time, with short breaks for things like showering or changing the pads.
Cost:
Optune is not routinely available on the NHS because it is very expensive - currently over £17,000 per month. Some private insurers may cover it, and the company offers financial support schemes in certain cases.
A large clinical trial called EF-14 found that people with newly diagnosed GBM lived longer when Optune was added to standard treatment (surgery, radiotherapy, and chemotherapy) compared with standard treatment alone.
For people whose GBM had come back, Optune was found to work about as well as chemotherapy, but with different side effects. Many doctors now use Optune alongside chemotherapy so the two treatments can work in different ways.
There are ongoing trials of Optune, which you might be able to access; however, it is worth stressing that these are exploring whether we can make Optune more effective; there is already good data for it having an effect.
Disrupts cancers ability to divide and grow
TUMOUR TREATING FIELDS
(TTFields)
Worn in a bag / backpack for daily use (18+ hours a day)
CONNNECTED TO A PORTABLE DEVICE
These deliver low-intensity electric fields directly to the tumour area
ELECTRODES ARE PLACED ON THE SCALP
Optune works by sending low-intensity electrical fields into the brain through self-adhesive pads placed on the scalp. These electrical fields slow or stop cancer cells from dividing and growing.
For brain tumour treatment:
The scalp needs to be kept shaved so the pads stick well.
The pads need to be replaced every 2-4 days.
The more hours you wear it each day, the more effective it is likely to be.
Optune is a cancer treatment that uses gentle electrical fields, called tumour treating fields (TTFields), to interfere with the way cancer cells grow and divide. It has the strongest evidence for helping people with glioblastoma (GBM), a type of brain tumour. Researchers are also studying whether it could help in other cancers, such as lung and ovarian cancer.
Optune is a portable device worn for most of the day. For GBM, this usually means wearing it for at least 18 hours every day to get the best results. Research suggests that it can improve survival when used alongside standard treatments like surgery, radiotherapy, and chemotherapy. Its effectiveness varies from person to person.
Brain tumours are complicated. Although surgery is still one of the main treatments, new technology is helping doctors understand them in much more detail. One important advance is genomic testing, which looks at the tumour’s DNA (genetic code) to find changes that might affect how the tumour grows and how it responds to treatment.
Extended genomic testing can give doctors a detailed “map” of the tumour’s genetic and molecular features. This can sometimes help predict how the tumour might behave and identify treatments that are more likely to work for you.
CLINICAL TRIALS
TREATMENT OPTIONS
COMPUTATIONAL MODELLING
Methylation profiling
METHYLATION
Copy number variation analysis
CNV
Next-generation sequencing
NGS
BIOPSY
In aggressive brain tumours, it’s common to find several different genetic changes. These changes can sometimes point towards specific treatments or clinical trials that may be worth considering.
Most patients will have some form of genetic testing on their tumour, but standard tests usually only look at 2–5 genes. While this can be helpful, it’s just a small snapshot of the 30,000 or so genes in the human genome. Extended next generation sequencing (often called NGS) can analyse many more genes at once, giving a much more detailed view of the tumour’s biology. This extra detail can sometimes reveal treatment options that would otherwise have been missed.
At the moment, not every hospital offers this kind of testing, and results need to be interpreted by specialists who have experience in this area.
Looks at every part of the DNA to find all mutations, including regions outside the protein-coding parts. However, it may not consider changes such as methylation or copy number variation in as much detail.
At the moment, WGS is made available for all patients with a primary brain tumour in England on the NHS. However, it requires patients to have fresh tissue frozen at time of surgery, and for that to be transported to the regional lab. For that reason, most centres have struggled to introduce it for everyone.
Checks hundreds or even thousands of genes at the same time to look for DNA changes that could be driving tumour growth. NHS tests for brain tumours often check around 200 genes. Some private labs can look at 600–750 genes, which may reveal more possible treatment options or clinical trials.
All the results are combined to understand the tumour more fully and guide treatment
Integration / Combined Analysis
Focuses on the tumour’s active genes to find more specific information
Exome Sequencing
Looks at whether genes are turned “on” or “off.” Helps reveal how the tumour is controlled
Methylation
Identifies when two genes are abnormally joined, which can drive tumour growth
Gene Fusions
Looks for missing or extra DNA. These changes can affect how the tumour behaves
Copy Number Variation (CNV)
Extended Genomic Testing
Gene amplification – the cell has too many copies of a gene. This can make the gene overactive, which may help the tumour grow faster.
Gene deletion – the cell has lost one or more copies of a gene. This can reduce or completely stop the gene’s activity, which can also affect how the tumour behaves or responds to treatment.
CNVs don’t always show up in standard sequencing tests. Special analysis is needed to detect them, which is why CNV testing is often done alongside other types of tumour profiling.
Every type of tumour has its own unique “methylation pattern,” a bit like a fingerprint. By comparing a tumour’s pattern to a large database of known tumour types, doctors can often make a more accurate diagnosis.
- A small piece of tumour tissue is tested in a local lab.
- The data are sent securely to a central database in Heidelberg, Germany.
- A detailed report is returned to the medical team, showing the most likely tumour type.
- Improves diagnosis - Helps confirm exactly what type of tumour it is, especially when standard tests are unclear.
- Better predictions - Can sometimes indicate how likely the tumour is to come back after surgery.
- Not just for gliomas - Originally developed for gliomas, it’s now used for other brain tumours like meningiomas too.
Why this matters:
- Tumour growth - Fusion genes can make cancer cells more aggressive.
- Prognosis - Certain fusions are linked to how the tumour is likely to behave over time.
- Treatment options - Some fusion genes are “targets” for specific drugs, meaning they can guide doctors toward personalised treatments.
This combined view, sometimes called Extended Genomic Testing, can help guide more personalised treatment plans and may open the door to treatments or clinical trials that wouldn’t have been considered otherwise.
- Suggest personalised treatment options.
- See if you might be suitable for clinical trials testing new therapies.
- Better understand how your tumour might respond to different drugs.
Although this technology is still developing, it may reveal new options and more personalised treatment plans. Because each modelling platform uses different methods and data, results usually need to be interpreted by specialists who understand both the science and the limitations of the technology.
- Find patterns in the genetic changes that might be important for your tumour’s behaviour.
- Predict how your tumour might respond to different treatments, including targeted therapies and immunotherapies.
- Help doctors create a treatment plan that’s as personalised as possible to your tumour’s unique features.
- NTRK fusions - rare, but can be treated with specific targeted medicines.
- FGFR fusions - also rare, but drugs exist for these changes.
- BRAF mutations - more common in certain tumour types and treatable with BRAF inhibitors.
• Papillary craniopharyngioma is also linked to BRAF mutations and may respond to the same treatments.
Finding these changes can sometimes give you access to new drugs or clinical trials that wouldn’t otherwise be considered.
Stored samples - If you’ve already had surgery or a biopsy, your tumour sample is most likely stored in a hospital laboratory. Often, it will have been preserved in a way called formalin-fixed paraffin-embedded (FFPE) - which means it’s treated with chemicals and stored in wax. These samples can still be tested using certain techniques, such as NGS, CNV and WES. This means even samples stored for years can sometimes be re-tested to look for more detailed information.
3–4 weeks after CNV results
Integration & Modelling
2 weeks after NGS completes
CNV Testing
10–12 days after sample arrives
NGS (Next-Generation Sequencing)
week 9
week 8
week 7
week 6
week 5
week 4
week 3
week 2
week 1
First, the tumour tissue (from biopsy or surgery) needs to be located and sent to the testing lab. This can take several days depending on hospital processes.
Sample retrieval
If Copy Number Variation (CNV) analysis is also being done, this can add another two weeks.
Once all the test results are ready, the information is combined and reviewed, sometimes using computational modelling to get the clearest possible picture. This final stage can take another 3-4 weeks.
In total, it can take around 10 weeks from starting the process to getting the full set of results and interpretation.
- Point towards a targeted drug that works specifically on that change.
- Make you eligible for a clinical trial testing new treatments for that type of alteration.
- Not every result will lead to a new treatment option right away, but it can still give doctors more insight into the tumour and how it might behave.
Specialists look for patterns or “markers” in the DNA that are known to be important for treatment or prognosis. The most valuable findings are called actionable alterations - changes like mutations, gene fusions, or differences in the number of copies of certain genes.
- Comprehensive testing panels.
- Reliable analysis methods.
- Experience in brain tumour profiling.
However, recommendations can change as technology improves. The goal of extended testing is to find genetic changes that standard hospital tests might miss. Knowing about these changes can sometimes open the door to targeted treatments or clinical trials. Using NGS, CNV analysis, and in some cases computational modelling, helps create a treatment plan tailored to the individual – rather than using a “one-size-fits-all” approach.
- What type of testing is needed - for example, Next-Generation Sequencing (NGS), Copy Number Variation (CNV) analysis, or more advanced molecular profiling.
- The quality and size of the gene panel - larger panels can look at hundreds of genes, picking up more potential treatment targets.
- Speed and accuracy - how quickly results are processed and how clearly they are explained.
- Clinical support - some providers work closely with doctors to help interpret the results and suggest next steps.
Although the technology is promising, some tests are still in the research stage and their benefit in day-to-day treatment is not yet fully proven.
Still being studied:
The findings can be difficult to interpret and need to be reviewed by specialists in tumour genetics.
Complex results:
Access to this type of testing can depend on where you live, the hospital you’re treated at and the resources available in your healthcare system.
Not always available:
This is an exciting and fast-moving area of medicine but not every finding will have an immediate treatment available.
Keep expectations realistic
If the results suggest a possible treatment, your medical team may discuss your case at a specialist meeting and look at targeted drugs, immunotherapy, or clinical trials that could be suitable for you now or in the future.
Plan the next steps
Testing can find genetic changes that open up new treatment options, but sometimes it may find changes with no current treatment, or none at all.
Know what results might show
Your doctors will send the testing team important details, such as pathology reports, operation notes, and any previous genetic test results.
Share your medical history
Most tests use tumour tissue collected during surgery or biopsy. Sometimes stored samples can be used, but certain tests (like whole genome sequencing) need fresh tissue and a blood or saliva sample for comparison.
Access your tumour sample
If NHS testing isn’t available, your doctor can tell you about reputable private labs, how long results might take, and what the costs could be.
Understand your options
Some tests may be offered through the NHS, while others might need a referral to a specialist centre or be arranged privately.
Find out what’s available
Your medical team will look at your diagnosis, tumour type, and current health to decide if this type of testing could be useful, especially if standard treatments are limited.
Check if it’s right for you
If you’re thinking about having extended genomic testing:
Extended genomic testing is a powerful tool that can give doctors a much clearer picture of your brain tumour. By studying the tumour’s genetic makeup in detail, it may be possible to find treatments that are more targeted and better suited to you. As research moves forward, these tests are likely to become more accurate, easier to access, and more useful in everyday treatment.
Immunotherapy is a type of treatment that helps your own immune system recognise and attack cancer cells. It can work in different ways, such as boosting your body’s natural defences or teaching your immune system to spot cancer more effectively.
For some cancers, immunotherapy has been a major breakthrough, helping patients live longer and, in some cases, keeping the cancer under control for years.
For brain tumours, research is still at an earlier stage. Some studies have shown encouraging signs that it might help certain people, but there isn’t yet enough strong evidence to make it part of standard treatment for most brain tumour patients.
- Immune checkpoint inhibitors - medicines that help the immune system “switch on” and attack cancer cells.
- Therapeutic vaccines - treatments designed to train the immune system to recognise and respond to specific tumour markers.
- Oncolytic viruses - specially engineered viruses that target and kill tumour cells, while also helping the immune system join the fight.
Right now, there’s no agreed “best” type of immunotherapy for brain tumours. Most of these treatments are still in the research stage and not yet part of standard care. While doctors are learning more through early trials and patient experience, the evidence is still developing, and results can vary from person to person.
May be less precise than T-cell therapies
Early stages in brain cancer
Ongoing trials in solid tumours
Uses natural killer cells to kill tumour cells
NK Cell Therapy
Can cause severe immune reactions
Still experimental in GBM
Breakthroughs in leukaemia
Modify patient’s T-cells to target cancer
CAR-T Cell Therapy
Require time to produce and may be costly
Not yet widely available
Some success in early trials
Teach immune system to target tumour-specific proteins
Personalised Vaccines
Limited penetration into tumours
Still early for brain tumours
May boost immune response locally
Special viruses that infect and destroy cancer cells
Oncolytic Viruses
Can cause autoimmune side effects
Only modest results in GBM
Used in some cancers like melanoma
Block “off switches” on immune cells so they attack tumours
Immune Checkpoint Inhibitors
Known Issues
What’s Experimental
What’s Promising
How It Works
Immunotherapy Type
- Immune checkpoint inhibitors - drugs that block the “off switches” tumours use to stop immune cells working.
- Oncolytic viruses - specially chosen or engineered viruses that infect and destroy cancer cells, while also alerting the immune system to the tumour’s presence.
- Personalised vaccines - vaccines designed for the individual patient, aimed at training the immune system to target specific features of their tumour.
- Cell-based therapies (such as CAR-T cells or NK cells) - immune cells taken from the patient or a donor, modified or selected to make them better at finding and killing cancer cells.
Unfortunately, many cancers – including brain tumours – find ways to “hide” from the immune system. They can create a local environment around the tumour that switches off immune cells, stopping them from attacking.
- A phase I trial of pembrolizumab given before surgery in recurrent glioblastoma (GBM) showed some improvement.
- A small study found that nivolumab combined with bevacizumab improved survival in patients not receiving steroids.
- Early data suggest combining pembrolizumab with Optune may enhance efficacy, with supportive evidence from both GBM and lung cancer cohorts.
Immune checkpoint inhibitors (ICIs) are drugs that block the “brakes” tumours put on the immune system. This can reactivate immune cells and help them attack cancer cells. ICIs have worked well in some cancers, such as melanoma, lung cancer, and kidney cancer.
For brain tumours, however, the benefits seen so far have been limited. Possible reasons include:
- Steroid use - many brain tumour patients need steroids to control swelling, but steroids can also weaken immune responses.
- Low tumour mutational burden - most brain tumours don’t have as many mutations as other cancers, making them harder for the immune system to recognise as abnormal.
- Previous treatments - some chemotherapy drugs or antibiotics may affect the gut microbiome, which plays a role in regulating immune function.
- Newcastle Disease Virus – studied for its ability to selectively kill tumour cells and stimulate the immune system.
- TOCA-511 – a genetically modified virus tested in several trials, including in Japan.
Practical challenges:
These treatments are only available in a small number of specialist centres. In many cases, patients must travel long distances – sometimes internationally – to receive them.
For example, IOZK in Germany offers an oncolytic virus-based therapy as part of a broader immunotherapy treatment package.
CAR-T therapy has shown impressive results in certain blood cancers, but its use in brain tumours is still in the early stages of research. This treatment is complex, highly resource-intensive, and can only be delivered in specialist centres. So far, only a small number of brain tumour patients worldwide have received CAR-T therapy. While some early outcomes are encouraging, significant questions remain around scalability, accessibility, and long-term benefit.
Other immune cell-based approaches, such as the use of Natural Killer (NK) cells, are even more experimental, with limited clinical data available at present.
In brain tumours, two main personalised vaccine approaches are currently under investigation:
- DCVax – Requires fresh tumour tissue collected at surgery. Dendritic cells are harvested from the patient’s blood, then exposed to tumour cells and immune-stimulating agents to create a personalised vaccine. The results of a large clinical trial have been published but remain the subject of considerable scientific and clinical debate.
- Personalised peptide vaccines – Use Whole Exome Sequencing (WES) to identify tumour-specific proteins. A custom vaccine is manufactured to target these proteins. This approach is generally less expensive than DCVax but has yet to be validated in large, controlled clinical trials.
One of the main centres currently offering personalised peptide vaccines is CECAVA (formerly known as CEGAT) in Germany. CECAVA has published recent data suggesting a potential improvement in survival, although further independent validation is needed - LINK TO PAPER.
You’ll need regular scans and check-ups to see how well treatment is working and to manage any side effects quickly.
Plan your follow-up
In research settings, immunotherapy is sometimes combined with other treatments like Optune or vaccines, but these approaches are still being tested.
Ask about combinations
Immunotherapy can cause inflammation in different parts of the body, which can sometimes be serious or last a long time. Tell your team straight away if you notice new symptoms.
Be aware of side effects
While there are some promising early results, there’s not yet strong proof that immunotherapy works well in brain tumours. Most patients have it as part of a clinical trial.
Understand the limits of the evidence
Many of these treatments are only available at specialist centres, and some are only offered abroad. This can mean planning travel and costs in advance.
Know where you can get it
Options include checkpoint inhibitors, oncolytic viruses, personalised vaccines, and cell-based therapies such as CAR-T or NK cells. The choice depends on your tumour type, test results, and where you can be treated.
Find the right type of immunotherapy for you
Steroids can reduce how well immunotherapy works. If it’s safe, your medical team may try to lower your dose or stop them before starting treatment.
Limit immune-suppressing medicines where possible
Most immunotherapy treatments for brain tumours are still experimental. You or your care team can search trial websites such as ClinicalTrials.gov or the EU Clinical Trials Register to find studies that might suit your tumour type, test results, and treatment history.
Check if you’re eligible for a trial
If you’re considering immunotherapy for your brain tumour:
Immunotherapy has transformed the treatment landscape for several cancer types, but its role in brain tumours remains investigational. Multiple clinical trials are ongoing, and future advances may yield more effective ways to harness the immune system against brain cancer.
At present, most immunotherapeutic strategies for brain tumours remain unproven and may carry significant risks, including immune-related adverse events. Both clinicians and patients should carefully balance potential benefits against these limitations, ideally pursuing treatment within a clinical trial or at a specialist centre with relevant expertise.
- Reducing nausea and vomiting - Cannabinoids can sometimes help control sickness caused by chemotherapy. However, there are other medicines for nausea that may work just as well or better, with fewer side effects, so these are often tried first.
- Possible anti-tumour effects - Laboratory and animal studies suggest cannabinoids might slow the growth of tumour cells, help them die, or reduce the tumour’s blood supply. But these results have not yet been proven to work in the same way for people with brain tumours. More research is needed before this can be considered an established treatment.
Cannabis and its derivatives – known as cannabinoids – are being studied for their possible role in helping people with brain tumours. Many patients are interested in this area, but it’s important to understand that the research is still in the early stages.
Most of what we know so far comes from laboratory studies or small-scale human studies. This means there are still many questions about how effective cannabinoids really are for brain tumours, what doses work best, and what side effects they might cause.
It’s also important to think about the type of product, whether it’s legal where you live, and how much it might cost before deciding if this treatment is right for you.
- Side effects – Cannabinoids can sometimes cause unwanted effects like sleepiness, dizziness, trouble concentrating, or changes in mood and perception. These effects are more likely at higher doses and can affect daily activities such as driving or working.
- Differences between products - Cannabis-based products can vary a lot in strength, ingredients, and purity. Non-medical or unregulated products may not be consistent or safe, which can make it harder to know what you’re getting.
- Cost – Prescription products like Sativex can be expensive, and they’re not always covered by insurance or the NHS. Even over-the-counter versions can add up in cost over time.
- Legal issues – Cannabis laws vary from place to place. In some countries or regions, it may be illegal or heavily restricted, which can affect whether you can access or travel with these products.
One small study in people with recurrent glioblastoma (GBM) found that using Sativex - a prescription mouth spray containing THC and CBD - alongside the chemotherapy drug temozolomide might help some patients live longer. The benefit was mainly seen in people whose tumours were MGMT-methylated. There is no evidence it helps people with MGMT-unmethylated GBM.
Cannabinoids have also been studied for symptom relief, such as improving appetite, easing pain, and reducing nausea. While there is ongoing research into whether they can also directly fight brain tumours, at the moment they are usually considered a complementary therapy rather than a main treatment.
- Choose regulated medical products - If you’re thinking about trying cannabinoids, it’s safest to use prescription products like Sativex. These are made to strict standards, so the dose and ingredients are consistent and checked for quality.
- Start low and go slow - Begin with a small dose and increase it gradually. This can help reduce the risk of side effects while finding the amount that works best for you.
- Think about combining with chemotherapy - Early research suggests cannabinoids might work best when taken alongside chemotherapy, especially in MGMT-methylated glioblastoma (GBM). This is still being studied, so it’s important to discuss it with your medical team.
- Weigh up the pros and cons - Consider the possible benefits alongside the known risks, side effects, cost, and legal rules where you live before deciding whether to try cannabinoid treatment.
Medical cannabis can be expensive and is not always covered by insurance.
Think about cost and access
This can help reduce the risk of side effects while finding the right dose for you.
Start low, increase slowly
If you’re struggling with nausea or vomiting, your care team can recommend other medicines that may have fewer side effects.
Look at other options first
So far, evidence of benefit is limited to MGMT-methylated GBM when combined with chemotherapy. There’s no proof it helps in MGMT-unmethylated GBM.
Check if it’s right for your tumour type
Medical-grade options like Sativex have consistent dosing, quality control, and safety checks. Avoid unregulated products where the strength and ingredients can vary.
Choose regulated products
Are you hoping to manage symptoms like nausea, or are you looking at it as a possible extra anti-tumour treatment?
Be clear about your goal
If you’re considering immunotherapy for your brain tumour:
There is some early evidence that cannabinoids might help in treating brain tumours, particularly in MGMT-methylated glioblastoma (GBM).
However, the research is still limited, and we don’t yet fully understand how effective or safe they are. A clinical trial is currently looking into this in more detail. If you’re thinking about trying cannabinoids, it’s important to talk it through with your doctor or care team so you can make an informed decision about whether it’s right for you.
Tzar Labs
DATAR Cancer Genetics
BillionToOne
Genomic Testing Cooperative
3. Imaging analysis using artificial intelligence
Some early-stage approaches use AI tools to analyse brain imaging in an attempt to infer tumour features. This field is still very much in its infancy, but one example of a company working in this area is Glioma AI.
It’s important to stress that none of these approaches are as informative as analysing tumour tissue directly. However, for people where tissue is not available, or where there is a need to track how a tumour changes over time or responds to treatment, they may still play a useful role.
2. Cerebrospinal fluid (CSF) testing
Another option is to analyse cerebrospinal fluid, which surrounds the brain and spinal cord. This requires a lumbar puncture, which is more invasive and less convenient than a blood test. However, because CSF is closer to the tumour than blood, it may contain more relevant material and can sometimes provide more useful information.
1. Blood-based testing (liquid biopsy)
This involves taking a blood sample and looking for genetic changes or circulating tumour material in the bloodstream. A number of companies offer this type of testing in both the UK and the US, and it’s possible for samples taken in the UK to be sent overseas for analysis. These tests typically cost around £3,000, although prices vary widely and the technology is evolving quickly. While this approach does not provide as much information as analysing tumour tissue directly, it may still offer some insight into the tumour’s characteristics.
All of the options discussed above rely on having a piece of the tumour available for analysis. However, around 1 in 5 people with glioblastoma (GBM) will not have a tumour sample taken. This may be because the tumour is in a difficult or high-risk area of the brain, or because the person is not well enough to undergo a biopsy.
In these situations, the question becomes whether it’s still possible to learn anything about the tumour. There are currently three main alternative approaches.
Some providers offering blood or CSF-based testing include:
Sometimes, doctors may look at treatments that aren’t usually part of standard brain tumour care. This could mean using medicines in a different way to how they’re licensed (off-label use), combining certain drugs, or trying newer medicines that are still being researched.
While these approaches may offer extra options, the evidence supporting them is often limited. They can also carry added risks, such as side effects, drug interactions or higher costs.
If these treatments are being considered, it’s important to talk through the possible benefits and risks with your care team, make sure you give informed consent, and have regular monitoring.
Researchers are always looking for new ways to treat brain tumours - not just through surgery, radiotherapy, and chemotherapy, but also by exploring other types of medicines. One area of interest is repurposing non-cancer drugs - medicines originally made for other conditions - that might also help slow tumour growth or work alongside existing treatments.
At the moment, there isn’t strong evidence to prove that most of these drugs are effective for brain tumours. Some are being studied in clinical trials, but we don’t yet know how much benefit they can offer.
Below, you’ll find a summary of some of the most talked-about options, along with an overview of what the research currently says. This information may change as new, better-quality studies are published.
Fluoxetine (also known by the brand name Prozac) is a common antidepressant used to treat depression and anxiety. Some early laboratory research has suggested it may also slow the growth of glioma cells.
A study from Stanford University found that people with glioblastoma (GBM) who were already taking fluoxetine when they were diagnosed lived longer, on average, than those who weren’t. However, this was an observational study, so we can’t say for certain that fluoxetine was the reason for the difference. More high-quality research is needed before it could be considered part of standard GBM treatment.
Fluoxetine is usually well tolerated. Side effects can include stomach upset, headaches, difficulty sleeping, or changes in sexual function. These can often be reduced by starting with a low dose and increasing gradually.
Valganciclovir is a medicine normally used to treat certain viral infections. It became a topic of interest in brain tumour research when early studies suggested a virus called cytomegalovirus (CMV) might be found in glioblastoma (GBM) cells.
Some researchers thought that treating CMV with valganciclovir could help people with GBM live longer. However, later studies haven’t been able to consistently find CMV in GBM tissue, and the survival benefits seen in the original research may not have been reliable.
Because the evidence is weak and results are mixed, valganciclovir is not currently recommended as a standard treatment for GBM and would usually only be considered as part of a research trial.
Metformin is a tablet commonly used to treat type 2 diabetes. Some early research suggests it might also affect how tumour cells grow and use energy.
In glioblastoma (GBM), higher blood sugar levels have been linked to poorer outcomes. This has led researchers to explore whether metformin could help as an extra treatment. So far, clinical trials haven’t shown clear proof that it improves survival in brain tumour patients.
Metformin is usually well tolerated, but it can sometimes cause side effects like bloating, stomach discomfort, or diarrhoea. A very rare but serious side effect called lactic acidosis can happen, especially in people with kidney problems.
Getting care overseas can be expensive and complicated, especially if you don’t have family or friends nearby to help with accommodation, transport, and day-to-day needs. We’ve seen people successfully take part in trials abroad - often in the United States - but in most cases they had local family support in place. Without that, the cost and practical demands can make overseas treatment very difficult to manage.
If you’re thinking about trying one of these drugs, your medical team will look at:
- The specific type and molecular features of your tumour
- Your overall health and any other medical conditions
- How the drug might interact with your current treatments
- Your personal priorities and treatment goals
Because there’s still a lot we don’t know, it’s important to have open conversations with your care team about the possible benefits and risks, and to be monitored closely for both side effects and signs that the treatment is working.
Consider the costs, travel, and whether you have local support. Treatment abroad can be challenging without friends or family nearby.
If thinking about care overseas
Make sure there’s a clear plan for checking whether the treatment is helping, and when to stop if it isn’t.
Plan how to monitor progress
Joining a well-run study can be the safest way to try newer or repurposed treatments.
Look into clinical trials
These treatments may not be available on the NHS or through your usual healthcare system, so you may need to pay privately. Discuss the possible benefits, risks, and costs with your care team.
Weigh up pros and cons
Your tumour type, test results, other health conditions, and past treatments can all affect whether a therapy is suitable for you.
Think about your own situation
Ask your medical team whether the treatment could clash with your current medicines or cause unwanted side effects.
Check for safety and interactions
Some of these treatments are supported only by early research or small studies. Make sure you know how strong - or limited - the data is.
Understand the evidence
Are you hoping to directly target the tumour (for example, using a drug usually given for another condition) or to improve symptoms and quality of life?
Be clear about the aim
If you’re considering options outside the usual brain tumour treatments, it’s important to make sure you have the right information and support:
If you’re exploring treatments that aren’t part of the usual brain tumour care pathway, it can sometimes be hard to find clear guidance or support. It may help to connect with doctors or centres that have experience with these treatments, or to ask your care team if they can consult with specialists who do.
You can also reach out to trusted charities that provide reliable information about clinical trials, practical tips for managing care, and emotional support for you and your family:
Patients family
Access to a website like this would have been a breath of fresh air during the chaos and confusion that followed my husband’s diagnosis. Having clear, specialist-led information in one place would have helped us better understand what was happening, what options existed and how to navigate decisions with more confidence. At a time when everything felt overwhelming, clarity and compassion in how information was presented would have made a real difference. I’m really glad that this website now exists for others facing a similar situation.
Patients family
Tackling a GBM diagnosis is extraordinarily overwhelming, the stats bulldoze you & researching treatment options online is sole destroying. Having one consolidated place to connect with other patients, understand additional treatment options & potential trials & follow others journeys is incredibly comforting and very much needed. Thank you. It’s a tough journey and we only get through it by all sharing our discoveries to make each others experiences that little bit easier.
Thank you for putting the time aside for this website. Being a caregiver to a 10momths in GBM patient I can honestly say that this website will benefit future patients enormously.
Patient
When everything changed, I didn’t want medical jargon or endless links. I just wanted clear, honest information I could actually understand.
Having it all in one place helped. Finding the information together was a huge relief. We could stop Googling and start getting a clearer picture of what was going on.
I could come back when I was ready. Some days I read a lot. Some days I couldn’t read anything at all. Knowing I could come back without pressure really helped.
Knowing more made things feel less chaotic. Understanding my options didn’t fix everything, but it helped things feel a little less out of control.
This was about more than treatment. This affected my whole life - not just my health. Seeing emotional and practical support included made me feel like that was understood. I didn’t feel like I was doing this alone. It felt like someone had already done the hard work of pulling this all together for me - and that meant a lot!
This website is an independent resource, developed by the Horizons in Neuro-Oncology (HINO) team in the UK. Initial development was supported by Dr Matt Williams and Lillie Pakzad-Shahabi, with grant funding from Novocure to support ongoing work.
HINO maintains editorial independence. While the team collaborates with a range of healthcare organisations and receives grant support from Novocure, all content is created and reviewed by the HINO team and reflects their combined clinical expertise, professional experience, and lived experience as patients and caregivers.
While content is based on UK clinical practice, much of the information may be relevant to international readers. It is provided for general guidance and should not replace medical advice from your own healthcare team.
Please use this information to support discussions with your local oncology team, or see our advice on obtaining a second opinion.
