Novel treatments and trials for Glioblastoma (GBM)
This section lists novel, alternative, and experimental treatments for glioblastoma, organised by how they work. Each entry describes what the treatment does, what research has been done to support it, whether it is currently available, and general context on costs or access.
The information here has come from the community and is for the community. These are therapies that we are often asked about. We are not recommending these treatments; we are signposting and sharing what information there is so that you can begin to have further conversations with your clinical team.
Wherever possible we have included the latest evidence that supports these approaches. A lack of evidence is itself important information; it is reasonable, and wise, to question the basis for any treatment being offered to you. We’d always encourage scrutiny of evidence and costs before pursuing any private treatment.
This brainstrust webinar gives a basic overview of the current landscape for GBM.
Working together to help people with a brain tumour
brainstrust and myTomorrows share the commitment to support people with brain tumours, and their families. We know identifying and pursuing a clinical trial can be challenging, and together we provide support on this journey. This support is always free of charge for people with brain tumours and healthcare professionals in the United Kingdom.
Immunotherapy
Immunotherapy aims to use or boost the body’s own immune system to recognise and attack brain tumour cells.
Updated June 2026
Immune checkpoint inhibitors (Keytruda / pembrolizumab, Ipilimumab, Nivolumab)
What they are: Drugs that release the ‘brakes’ on the immune system, allowing T-cells to attack tumour cells. Key targets include PD-1/PD-L1 (nivolumab, pembrolizumab) and CTLA-4 (ipilimumab).
Current evidence: Results in GBM have been largely disappointing in unselected populations. Combination approaches, particularly anti-LAG-3 plus anti-PD-1 (relatlimab + nivolumab), are in Phase II trials for recurrent GBM. Pembrolizumab is also being studied with Tumour Treating Fields (Optune) in newly diagnosed GBM.
Availability: Not routinely available via NHS for GBM. Access primarily through clinical trials.
Dendritic cell vaccines (including DCVax-L / ADCV)
What it is: A personalised vaccine made from the patient’s own immune cells (dendritic cells) loaded with tumour antigens. Designed to train the immune system to recognise the tumour.
Current evidence: DCVax-L showed a statistically significant overall survival benefit in a Phase III trial (2023), with a subset of patients achieving long-term survival.
Availability: Not currently available via the NHS. Northwest Biotherapeutics is pursuing regulatory approval.
Note: A private treatment with significant cost. Patients should seek independent clinical opinion and review published evidence carefully.
Personalised Neoantigen Vaccines (NeoVax / GAPVAC series)
What it is: A highly personalised vaccine using genomic sequencing of the patient’s own tumour to identify unique mutations (neoantigens). A customised vaccine is manufactured for each individual patient.
Current evidence: Early Phase I data from GAPVAC-101 and NeoVax trials: nearly 100% of patients developed a measurable T-cell response. Early data suggests median OS of approximately 24–26 months, compared to the historical average of 14–16 months.
Availability: Investigational only – within named clinical trials at academic cancer centres.
CeGaT Personalised Neoantigen Peptide Vaccine
What it is: A fully personalised peptide vaccine developed by CeGaT GmbH (Tübingen, Germany), manufactured individually for each patient based on genomic sequencing of their tumour. Tumour tissue is sequenced to identify unique mutations (neoantigens), the patient’s HLA type is determined, and a bespoke set of peptides is synthesised and administered as a vaccine series to train the immune system to recognise and attack tumour cells. This is distinct from CeGaT’s diagnostic profiling service (see Section 5), which provides the sequencing data that underpins vaccine design but can also be used independently to guide treatment decisions.
Current evidence: The most substantial published evidence for this approach in GBM comes from a 2024 real-world study (Latzer et al., Nature Communications) involving 173 GBM patients who received a personalised neoantigen peptide vaccine as an individual healing attempt alongside or after standard care. Median overall survival from diagnosis across all patients was 31.9 months — roughly double the historical standard-of-care figure of 14–16 months. A vaccine-induced immune response was detected in 90% of monitored patients, and those who mounted strong responses fared substantially better (median OS 53 months) than those who did not (27 months; p=0.03). Adverse events were predominantly mild (grade 1 or 2). A separate 2025 publication reported findings in 52 patients with IDH1-mutant glioma (grades 2–4) who received a neoantigen vaccine alongside standard care; immunogenicity against the IDH1 mutation was 89%, with robust and durable T-cell responses observed.
Important caveats: The 2024 study is a real-world observational cohort, not a randomised controlled trial. Patients were not randomly assigned to treatment or control, and those who pursued the vaccine may differ from the broader GBM population in ways that affect survival. Randomised trials are planned and will be needed to establish the size of any benefit. The survival figures, while striking, should be interpreted with care.
Availability: A private treatment. Patients travel to Tübingen, Germany for an initial series of four vaccines (the training phase), with subsequent vaccines given every 3–6 weeks. UK oncologists vary in their familiarity with and openness to this approach; some are experienced in working alongside CeGaT.
Timing: Starting the diagnostic and vaccine manufacturing process as early as possible is important. Genomic profiling takes approximately 4–6 weeks; vaccine manufacture takes a further 12 weeks from that point.
Cost: Tumour neoantigen diagnostics cost approximately €10,000. Total vaccine costs vary per patient depending on the number of peptides; one patient’s published account reports a total of approximately €42,000 for the full vaccine course. This does not include travel, accommodation, or immune monitoring costs.
Note: A private treatment with significant cost and logistical demands. Patients should seek independent clinical opinion, discuss timing with their oncologist, and review the published evidence carefully before proceeding.
Published evidence: Latzer P et al. A real-world observation of patients with glioblastoma treated with a personalized peptide vaccine. Nature Communications 15, 6870 (2024).
Immucura
What it is: A private immunotherapy service offering personalised dendritic cell vaccines and other immune-based treatments.
Note: A private treatment with significant cost. Patients should seek independent clinical opinion and review published evidence carefully.
IOZK Vaccine / Immunotherapy
What it is: A multi-modal immunotherapy programme at IOZK in Cologne, incorporating dendritic cell vaccination, Newcastle Disease Virus (an oncolytic virus), and other immunotherapies.
Note: A private treatment with significant cost. Patients should seek independent clinical opinion and review published evidence carefully.
CAR-T cell and adoptive cell therapies
CAR-T therapy involves engineering a patient’s own T-cells to recognise and kill tumour cells. This is one of the most rapidly developing areas in GBM research.
What is CAR-T Cell Therapy: T-cells are taken from the patient, genetically modified to carry a receptor targeting specific tumour antigens (EGFRvIII, IL13Rα2, HER2, B7-H3), then infused back.
Delivery: Locoregional delivery (intratumoral, intracavitary, or intraventricular) is showing more promise than intravenous infusion due to the blood-brain barrier.
Current evidence: Phase I trials in 2025 demonstrated safety and feasibility. One case showed 77–100% tumour reduction after 16 intracranial infusions, with clinical benefit lasting approximately 7.5 months. Multi-antigen (bispecific) CAR-T constructs are under development to reduce tumour escape.
CAR-NK cells: Natural killer cells are also being engineered as an alternative, potentially available ‘off the shelf’ without using the patient’s own cells.
Availability: Investigational only – specialist Phase I/II trials, primarily in the US and Europe.
Oncolytic virus therapy
Oncolytic viruses selectively infect and kill cancer cells while sparing healthy tissue and also stimulate an immune response.
Oncolytic viruses (G47Δ, DNX-2401, and others)
G47Δ (teserpaturev): A third-generation oncolytic herpes simplex virus. Phase II study in recurrent/residual GBM showed 1-year survival of 84.2% and median OS of 20.2 months trial stopped early for efficacy. Approved in Japan.
DNX-2401: An oncolytic adenovirus studied with pembrolizumab. Early data showed encouraging responses in recurrent GBM.
AAV-based viral immunotherapy: A Brain Tumour Research-funded programme entered its first GBM patient in early 2026 using an AAV vector, designed as a one-and-done treatment.
Availability: G47Δ approved in Japan. Trials running in Europe and North America. Not available via NHS.
Dr Benjamin Gesundheit / ATGesundheit Institute
Dr Benjamin Gesundheit / ATGesundheit Institute
What it is: A Jerusalem-based consultancy run by Dr Benjamin Gesundheit (Rapo Yerape Ltd) that advises patients on investigational oncolytic virus (OV) immunotherapy for GBM and other solid tumours. Dr Gesundheit reviews individual patient medical data and evaluates whether a patient may be a candidate for last-resort OV treatment, working with a network of treating clinicians across Europe and Israel.
Published evidence: Dr Gesundheit is lead author on a 2020 case series (Frontiers in Oncology) reporting four GBM patients treated with combination OV immunotherapy regimens who achieved clinical and radiological responses with long-term survival of up to 14 years and good quality of life. A further case report on OV therapy for relapsed IDH-mutant Grade 3 astrocytoma was published in 2025.
How this differs from IOZK: IOZK (Dr Van Gool) is a clinic that administers multi-modal immunotherapy directly. ATGesundheit operates as a consultancy: Dr Gesundheit reviews cases and advises on eligibility for OV treatment, then works with a wider network of clinicians to deliver it. Both are private and involve significant cost.
Note: The published case series involves small numbers; patients should seek independent clinical opinion before pursuing any private treatment.
Viral Vector Gene Therapy
Gene therapy involves delivering genetic instructions into cells to alter their behaviour.
What it is: Therapeutic genes are delivered using viral vectors (modified viruses). New instructions can produce proteins that kill cancer cells or stimulate immune responses.
Current status: A Brain Tumour Research-funded programme entered its first GBM patient in early 2026 using an AAV vector.
Availability: Early clinical trial phase only.
Targeted molecular therapies
These treatments target specific molecular features of the tumour rather than attacking all rapidly dividing cells as chemotherapy does.
CeGaT Tumour Diagnostics (CancerNeo® / CancerPrecision®)
What it is: A molecular diagnostics service offered by CeGaT GmbH (Tübingen, Germany) that provides comprehensive genomic profiling of tumour tissue. CeGaT’s CancerNeo® analyses the tumour exome to detect tumour-specific somatic mutations across more than 700 cancer-relevant genes, determines the patient’s HLA type, and predicts neoantigens — confirmed by whole RNA sequencing of the same sample. CancerPrecision® provides broader tumour profiling to identify targetable mutations and potential treatment options. CancerAdvice® adds a clinical interpretation layer, matching findings to current evidence, off-label therapies, and open clinical trials.
How this differs from the NHS: NHS genomic profiling in GBM currently reports IDH status, MGMT methylation, and a limited panel of standard markers. CeGaT’s profiling is considerably more comprehensive and is specifically designed to identify the full mutational landscape needed for personalised vaccine design, trial matching, and off-label treatment conversations.
Two uses of CeGaT diagnostics:
The profiling results can be used in two ways, independently or in combination:
- To inform conversations with your oncologist about clinical trial eligibility, off-label treatments, or targeted therapies that match your tumour’s specific molecular profile.
- As the foundation for a personalised neoantigen peptide vaccine manufactured by CeGaT (see Section 1 for full details of the vaccine, its evidence, costs, and logistics).
Published evidence: CeGaT has published peer-reviewed work on biomarker-guided therapy outcomes in adult patients with nervous system tumours (2023), and its genomic profiling platform underpins the 2024 Nature Communications real-world GBM vaccine study (173 patients; Latzer et al.). Analytical validation data for their liquid biopsy panel (523-gene ctDNA assay) have also been published (2023).
Note: A private service. Profiling alone may or may not identify an actionable mutation or trial match; this is not guaranteed. It is worth discussing with your oncologist whether NHS Genomic Medicine Service pathways or other academic profiling programmes may offer equivalent information before pursuing private options.
Cost: Tumour neoantigen diagnostics approximately €10,000. Turnaround approximately 4–6 weeks.
Vorasidenib (IDH-mutant glioma)
What it is: A dual IDH1/IDH2 inhibitor — an oral targeted therapy for IDH-mutant low-grade glioma. Primarily relevant to Grade 2–3 IDH-mutant glioma rather than IDH-wildtype GBM.
Current evidence: INDIGO Phase III trial showed significant improvement in progression-free survival. FDA-approved 2024. Scotland SMC positive 2025; England NICE decision pending.
Repurposed Drugs
What they are: Existing licensed medications (metformin, statins, chloroquine, valproic acid and others) being investigated for anti-cancer effects in GBM.
Evidence: Highly variable. Few have strong Phase III evidence in GBM.
Platform and adaptive trials
Platform trials test multiple treatments simultaneously, using adaptive methods to allocate more patients to the most promising treatment arms.
GBM AGILE
What it is: The Glioblastoma Adaptive Global Innovative Learning Environment is an international Phase II/III adaptive randomisation platform trial evaluating multiple therapies simultaneously in newly diagnosed and recurrent GBM.
Why it matters: Rather than testing one drug at a time, GBM AGILE compares multiple experimental treatments against standard of care simultaneously, continuously reassigning patients to the most effective arms.
Availability: Active in the US, Australia, and participating European sites. Ask your oncologist about eligibility.
★ INSIGhT Trial
What it is: The Individualised Screening Trial of Innovative Glioblastoma Therapy — a Phase II platform trial using Bayesian adaptive randomisation to match newly diagnosed GBM patients to experimental treatments based on molecular tumour profiling.
Availability: US-based. Published in Journal of Clinical Oncology (2023).
Technology-based treatments
This section covers treatments that use physical technologies e.g. electric fields, laser heat, and sound waves, to target tumour cells. These are distinct from drug therapies.
Tumor Treating Fields (TTFields / Optune)
What it is: A wearable device (Optune, by Novocure) delivering low-intensity, alternating electric fields to the scalp to disrupt tumour cell division. Worn continuously alongside maintenance temozolomide.
Current evidence: Phase III EF-14 trial showed improved survival in newly diagnosed GBM. Now being combined with pembrolizumab in further trials.
Trials open: The EF-41/KEYNOTE D58 study, combining Optune with maintenance temozolomide and pembrolizumab in newly diagnosed GBM, is recruiting to UK sites. Data expected approximately 2029.
Availability: TTFields is NOT currently available on the NHS. In August 2024, NICE confirmed it would not prioritise TTFields for review, citing limited evidence, cost, and ongoing trials. brainstrust and other brain tumour charities are actively campaigning to change this. Private access costs approximately £20,000 per month. Some insurers will cover this; many will not. Access via the TRIDENT trial is now closed to recruitment.
AutoLITT - Laser Interstitial Thermal Therapy (LITT)
What it is: A minimally invasive surgical technique in which a laser probe is placed stereotactically into the tumour and delivers heat to ablate it under real-time MRI thermometry guidance. AutoLITT (Monteris Medical) and Visualase (Medtronic) are the main systems. It is particularly useful for deep-seated or hard-to-access tumours, or where open surgery is not appropriate.
Current evidence: LAANTERN prospective registry (14 US centres) showed safety and feasibility in both newly diagnosed and recurrent IDH-wildtype GBM, with median OS comparable to open resection studies when followed by standard chemoradiotherapy. LITT also causes transient disruption of the blood-brain barrier, which may enhance subsequent drug delivery. A 2026 study showed the technique can be combined with open craniotomy in the same session for multifocal GBM.
UK availability: Available at a small number of specialist neurosurgical centres in the UK. Not routinely commissioned and requires individual case assessment.
Sonodynamic Therapy (SDT)
What it is: A non-invasive treatment that combines low-intensity ultrasound with a sonosensitiser drug (typically 5-ALA, already used in brain tumour surgery) taken orally. When the ultrasound activates the drug in tumour tissue, it generates reactive oxygen species that kill cancer cells, without affecting healthy brain tissue.
Current evidence: A first-in-human Phase 1/2 trial (Alpheus Medical / SonALAsense) in recurrent high-grade glioma showed median OS of 15.7 months (historical benchmark: 6 – 8 months) and median PFS of 5.5 months (historical: 1.8 months), with no treatment-related deaths or serious adverse events. A Phase II trial in newly diagnosed GBM (SDT combined with standard chemoradiotherapy) is underway in China. A Mayo Clinic trial using MR-guided focused ultrasound with 5-ALA in recurrent GBM opened in 2025.
Why it matters for patients: The treatment is non-invasive, uses an existing drug already approved for intraoperative use in brain tumour surgery (5-ALA), and can be administered at day-case appointments. It can also transiently open the blood-brain barrier, potentially improving delivery of chemotherapy.
Availability: Investigational – clinical trials in the US, Europe, and China. Not yet available via NHS. A multi-centre randomised trial is planned.
MR-Guided Adaptive Radiotherapy
What it is: Real-time MRI is used during each radiotherapy session to adapt the treatment plan to the current shape and position of the tumour. Dose can be escalated to high-risk, treatment-resistant regions.
Current evidence: A Phase II/III trial (University of Michigan) is investigating whether this approach improves overall survival in newly diagnosed GBM compared to conventional chemoradiotherapy. Estimated completion 2027.
Availability: Specialist centres only. Limited UK availability.
Dietary and metabolic approaches -Ketogenic Diet
There is growing interest in whether dietary interventions can influence the tumour environment alongside standard treatment.
What is Keto: A high-fat, very low-carbohydrate diet. The hypothesis is that cancer cells are more dependent on glucose than normal cells and reducing glucose availability may slow tumour growth.
Current evidence: A randomised multi-site trial is comparing a ketogenic diet versus a standard anti-cancer diet in newly diagnosed GBM alongside standard of care. This is the first properly powered randomised study; evidence to date is preliminary.
Important: Please discuss any significant dietary change during treatment with your oncologist and a registered dietitian. Nutritional adequacy and calorie intake are critical. Some foods can interfere with the efficacy of chemotherapy.
Antivirals - Valganciclovir
Some research has explored whether cytomegalovirus (CMV), found in some GBM tumours, may play a role in tumour biology.
What it Valganciclovir: An antiviral drug licensed for CMV infection, explored as an adjunct in GBM based on the hypothesis that CMV may support tumour growth.
Evidence: Observational data suggested benefit, but the VIGAS Phase II randomised trial did not show a significant difference in outcome. This remains an active area of debate.
Availability: A prescription drug. Off-label use in GBM is not recommended outside a trial context.
If you have treatments to suggest for this list, please email hello@brainstrust.org.uk
Last reviewed: June 2026. brainstrust – the brain cancer people. Registered charity 1114634.