When facing a brain cancer diagnosis, many patients and families find themselves asking the same question: Is there anything more effective than just chemotherapy or radiation? The good news? Medical science has taken major leaps forward. Two powerful options—targeted therapy and immunotherapy—are offering new hope. But how do they work, and who can benefit? Let’s break it all down in a simple, human way.
What Is Targeted Therapy?
Imagine a smart missile that seeks out the enemy without damaging the nearby town. That’s what targeted therapy does to cancer cells.
Unlike traditional chemotherapy, which attacks both cancerous and healthy cells, targeted therapy zooms in on specific mutations or proteins found in tumour cells. It disrupts the very systems that cancer depends on to grow and spread. Think of it as hacking the cancer’s operating system.
These therapies usually come in pill or infusion form. But they don’t work for every tumour—only those with certain molecular markers. That’s why genetic testing on the tumour often comes first.
How Immunotherapy Works Against Brain Tumors
Your immune system is incredibly powerful. It fights viruses, bacteria, and even rogue cells—until cancer finds clever ways to hide.
This is where immunotherapy comes in. It acts like a spotlight, helping your immune system see what it’s been missing. There are a few ways it does this:
- Checkpoint inhibitors remove the “brakes” that cancer uses to suppress immune response.
- Cancer vaccines train your body to recognise tumour markers.
- CAR T-cell therapy reprograms your immune cells to hunt down cancer like a heat-seeking missile.
Pretty sci-fi, right? But it’s very real—and researchers are refining it every day.
Why Brain Cancer Is So Hard to Treat
So, if we’ve got all these high-tech options… why is brain cancer still so deadly?
One word: barrier. The blood-brain barrier is like a super-strict security guard. It protects the brain from harmful substances—but also blocks many cancer drugs.
Add to that the brain’s delicate structure and the sheer variety of brain tumours (like glioblastoma, astrocytoma, medulloblastoma), and you’ve got a tough challenge. Some tumours mutate quickly, making it hard for treatments to keep up.
Common Targets in Brain Cancer Therapy
To use targeted therapy, doctors first need to identify what to target. Here are a few usual suspects:
- EGFR (Epidermal Growth Factor Receptor) – often altered in glioblastoma.
- IDH1 and IDH2 mutations – common in lower-grade gliomas.
- MGMT methylation – not a mutation, but a marker that predicts how well chemo might work.
These markers act like fingerprints, helping doctors decide which treatments may work best.
FDA-Approved Targeted Therapies for Brain Tumors
Here’s where things get practical. Some targeted drugs have already made their way into clinics:
- Bevacizumab (Avastin) – blocks blood vessel growth in glioblastoma.
- Tovorafenib – for pediatric brain tumours with specific BRAF alterations.
- Everolimus – approved for subependymal giant cell astrocytoma (SEGA) in tuberous sclerosis patients.
While these drugs aren’t miracle cures, they often slow tumour growth and help manage symptoms.
Promising Immunotherapy Trials in Brain Cancer
Clinical trials are like the testing ground for tomorrow’s breakthroughs. And right now, brain cancer is a hot focus:
- DCVax®-L: A personalised vaccine for glioblastoma that showed improved survival in a 2022 study.
- CAR T-cell therapies: Still experimental, but early results in glioma patients are encouraging.
- Checkpoint inhibitors: Like nivolumab (Opdivo), tested in combination with radiation and surgery.
These are not yet standard treatments—but they’re paving the way forward.
Side Effects: What to Expect
Let’s be honest—no treatment is side-effect free.
Targeted therapies often cause:
- Skin rash
- Fatigue
- Diarrhoea
- Elevated liver enzymes
Immunotherapies can stir up:
- Inflammation in the lungs, skin, or gut
- Autoimmune-like reactions
- Fever and fatigue
But here’s the good news: these effects are often milder than those of chemotherapy, and doctors are getting better at managing them.
Who Is a Candidate for These Treatments?
Not everyone can receive targeted therapy or immunotherapy. These treatments depend heavily on:
- Tumour genetics – Specific mutations or markers must be present.
- Patient health – Some treatments require a strong immune system.
- Previous treatments – Immunotherapy often works better in newly diagnosed or recurrent cases, not after multiple rounds of chemo.
Doctors will usually do a biopsy or advanced imaging tests before recommending these therapies.
Combining Immunotherapy with Other Treatments
Can you combine forces? Absolutely.
Some of the most exciting studies involve combo therapies:
- Radiation + checkpoint inhibitors
- Targeted drugs + chemo
- Surgery + cancer vaccine
The idea is simple: hit the tumour from multiple angles so it has less chance to fight back.
Think of it like a heist movie—you want the tech expert, the muscle, and the inside man. One approach alone isn’t always enough.
The Future of Precision Oncology in Brain Tumors
The horizon looks brighter than ever.
- AI and machine learning are helping doctors match treatments to tumour types with greater precision.
- CRISPR gene editing might someday let us fix the mutations before they cause harm.
- Personalised vaccines are being developed from a patient’s own tumour cells.
What was once science fiction is now becoming science fact.
Challenges and Limitations
But let’s not sugar-coat it—there are real hurdles too.
- Cost: These therapies can be expensive, and not all are covered by insurance.
- Access: Rural or low-resource settings may lack the tech or expertise.
- Resistance: Tumours can evolve and become resistant to targeted drugs.
- Limited pediatric data: Most trials are done on adults.
The race is on—not just to discover treatments, but to make them available to everyone who needs them.
Final Thoughts
So, is targeted therapy or immunotherapy the magic bullet for brain cancer?
Not quite—not yet.
But they’re a huge leap in the right direction. They offer precision, personalisation, and in many cases, longer life with better quality. If you or a loved one is facing brain cancer, talk to your doctor about these options. Ask about genetic testing. Inquire about clinical trials.
The future of brain cancer care is not just about killing tumours. It’s about targeting smarter, fighting cleaner, and living longer.
References
- Bevacizumab in the Treatment of Glioblastoma – National Cancer Institute, 2022. https://www.cancer.gov/about-cancer/treatment/drugs/bevacizumab
- DCVax-L Phase 3 Trial in Glioblastoma – Journal of Clinical Oncology, 2022. https://ascopubs.org/doi/full/10.1200/JCO.22.00352
- Immunotherapy for Brain Tumors: Current Landscape and Future Directions – Frontiers in Oncology, 2023. https://www.frontiersin.org/articles/10.3389/fonc.2023.1134901/full
- IDH Mutations in Glioma – Clinical Impact – The Lancet Oncology, 2021. https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(21)00071-3/fulltext
- Checkpoint Inhibitors in Brain Cancer – Nature Reviews Cancer, 2022. https://www.nature.com/articles/s41568-022-00499-z