Immunotherapy for Brain Cancer
Immunotherapy is an emerging treatment approach that works by helping the body’s immune system recognise and attack brain cancer cells more effectively.
What Is Immunotherapy And How Does It Work?
Immunotherapy for brain cancer is a type of cancer treatment that is being actively researched in brain cancer. It aims to help the body’s immune system develop to recognise and attack cancer cells. The immune system normally protects the body by detecting and removing infections and abnormal cells.
Cancer cells can carry changes in proteins on their surface that distinguish them from healthy cells, but they often develop ways to hide from or suppress the immune response. Immunotherapy works by boosting immune activity or by helping a patient’s immune cells better recognise and kill cancer cells; or by blocking proteins that suppress immune responses, allowing immune cells to better recognise and attack cancer cells, although normal tissues may also be affected.
However, the biological features of brain tumours have made immunotherapy particularly challenging, meaning that many treatments are still in development and undergoing clinical testing.
Immunotherapies currently being developed and tested for brain cancer patients
- Immune checkpoint inhibitors
- CAR T-cell therapy
- Oncolytic virus therapy
- Monoclonal antibodies
- Cancer vaccines (including mRNA, peptide, and dendritic cell vaccines)
Immunotherapies currently being developed and tested for brain cancer patients
- Intravenous (IV) infusion through a vein
- Oral medication (tablets or capsules)
- Topical treatment applied to the skin (for some skin cancers)
Immunotherapy is often given through an IV and is usually delivered as an outpatient treatment. This means patients can typically go home the same day. Treatment is given in cycles, often every 2–6 weeks, depending on the specific therapy or clinical trial.
In Australia, immunotherapy is not yet standard care for many brain tumours, so it is often offered through clinical trials, alongside treatments such as surgery, radiotherapy, or chemotherapy but not as a standalone option.
Immunotherapy Brain Cancer Treatment: Effectiveness and Success Rates
Many patients often ask, ‘How effective is immunotherapy?’ Immunotherapy has transformed treatment for some cancers, but in primary brain tumours its role is still being defined. For most primary brain tumours, especially glioblastoma, immunotherapy has not yet shown consistent benefit in large randomized clinical trials.
Many factors influence how well immunotherapy works. These include the tumour type and grade, the type and dose of immunotherapy used, individual biological factors including genetics and immune system characteristics, and whether immunotherapy is given alone or together with other treatments. Emerging research also suggests the gut microbiome may influence immune responses, although its role in brain cancer is still being studied.
Why Immunotherapy Works Better in Some Cancers
Immunotherapy has been more successful in cancers such as melanoma and in some subtypes of breast cancers, than in primary brain cancer. These cancers often have a higher number of genetic mutations, which can make them easier for the immune system to recognise and target.
Challenges in Treating Brain Cancer with Immunotherapy
In contrast, brain cancers, especially childhood brain cancers, are notably difficult to treat with conventional immunotherapy due to a combination of low genetic mutation rates and an active, immunosuppressive tumour microenvironment. Additionally, the brain and spinal cord are protected by the blood–brain barrier, which can limit the ability of immunotherapy drugs to reach the tumour cells.
Promising New Approaches in Immunotherapy
Some newer immunotherapy approaches have shown early signals of activity in brain cancer, although their effectiveness remains under investigation. These therapies aim to stimulate the patient’s immune system to better recognise and attack tumour cells, and in some cases may help generate longer-lasting immune responses. However, their ability to reliably prevent tumour recurrence has not yet been demonstrated. The brain tumour’s microenvironment can influence how well these treatments work. Brain tumours often create an immunosuppressive environment that limits immune activity and reduces treatment effectiveness.
Personalised cancer vaccines, designed to target a person’s own tumour antigens, have been associated with improved survival in glioblastoma, with very few serious side effects reported. CAR T-cell therapy may also be effective in situations where cancer cells change or hide their markers to avoid recognition by the immune system, a common strategy used by tumours. However, responses have been variable and remain under active study.
Combining Immunotherapy with Other Brain Tumour Treatments
When using immunotherapy for advanced brain cancers, treatment often involves a combination of therapies, such as surgery, radiotherapy, chemotherapy, and investigational immunotherapies. The goal of combining treatments is to improve tumour control, but this approach can also increase the risk of side effects. Adults and children can have different responses to immunotherapy because of differences in tumour biology and immune system characteristics. Immunotherapy research continues to explore how age influences treatment outcomes.
Immunotherapy Side Effects and Risks
Like other cancer treatments, immunotherapy brain cancer treatment can also cause side effects. These may occur during treatment or develop weeks to months after treatment has started or finished. Side effects can occur because immunotherapy activates the immune system to fight cancer, and in some cases, the immune system may also attack healthy tissues that share similar proteins (even though at low levels) with cancer cells.
Common side effects include:
- Tiredness (fatigue)
- Nausea
- Skin rashes
- Diarrhoea
- Fever or chills
- Muscle soreness or pain at the injection site (vaccines or subcutaneous therapies)
Letting your healthcare team know about side effects early helps them manage symptoms effectively. Doctors may prescribe steroids or other medicines to calm the immune system while continuing treatment when it is safe to do so.
Rare cases of immune-related reactions include:
- Cytokine Release Syndrome (CRS) (most commonly seen with CAR T-cell therapy)
- Inflammation of the bowel (colitis)
- Autoimmune conditions, such as type 1 diabetes
- Inflammation of organs, including the heart (myocarditis), lungs (pneumonitis), or liver (hepatitis)
- Neurological symptoms such as confusion, seizures, or inflammation around the brain
If side effects become severe, treatment may need to be paused while medications are given to calm the inflammation before resuming therapy.
Monitoring and Support During Treatment
Your healthcare team will closely monitor you throughout treatment. Reporting any changes early is the best way to prevent complications. Many side effects can be managed with simple measures such as staying hydrated, adjusting diet, using moisturisers or sunscreen, or taking prescribed medications.
You will not face these side effects alone, your care team’s close supervision helps ensure your safety, comfort, and ongoing treatment success.
Immunotherapy Clinical Trials in Australia
In Australia, immunotherapy is not currently a standard treatment option for primary brain tumours. However, some patients may be able to access immunotherapy by taking part in clinical trials. Your healthcare team will discuss whether a clinical trial is suitable for you and help identify the most appropriate option.
As of October 2025, immunotherapy treatments may become more accessible for Australians living with advanced cancer, following recommendations from the Pharmaceutical Benefits Advisory Committee (PBAC).
Several types of Immunotherapy procedures are currently being studied in clinical trials for brain tumours, including:
- Immune checkpoint inhibitors, such as nivolumab and pembrolizumab
- Cancer vaccines, including PEP-CMV and DCVax
- Oncolytic viral therapies, such as PVSRIPO and G47Δ
- CAR-T cell therapy, which uses modified immune cells to target cancer
For patients who are eligible, clinical trial participation usually covers the cost of immunotherapy treatment. These trials are supported by government funding and brain tumour charity organisations.
References
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- Chen, Q., Zhao, B., Tan, Z., Hedberg, G., Wang, J., Gonzalez, L., Mugabo, C. H., Johnsson, A., Negrini, E., Páez, L. P., Rodriguez, L., James, A., Chen, Y., Mikeš, J., Bernhardsson, A. K., Reitzner, S. M., von Walden, F., O’Neill, O., Barcenilla, H., Wang, C., Davis, M. M., Carlson, L.-M., Pal, N., Blomgren, K., Repsilber, D., Herold, N., Lakshmikanth, T., Kogner, P., Ljungblad, L., & Brodin, P. (2025, 2025/03/06/). Systems-level immunomonitoring in children with solid tumors to enable precision medicine. Cell, 188(5), 1425-1440.e1411. https://doi.org/https://doi.org/10.1016/j.cell.2024.12.014
- Gálvez MF, et al. Pediatric and adult tumor immunology: similarities and differences. Journal of Clinical Oncology. 2018.
- Liu, Y., Zhou, F., Ali, H. et al. Immunotherapy for glioblastoma: current state, challenges, and future perspectives. Cell Mol Immunol 21, 1354–1375 (2024). https://doi.org/10.1038/s41423-024-01226-x
- Morgado, M., Plácido, A., Morgado, S., & Roque, F. (2020, Oct 1). Management of the Adverse Effects of Immune Checkpoint Inhibitors. Vaccines (Basel), 8(4). https://doi.org/10.3390/vaccines8040575
- https://oncodaily.com/oncolibrary/immune-oncology/immunotherapy-for-brain-cancer
- https://www.cancerresearch.org/immunotherapy-side-effects