Glioblastoma is the most aggressive brain tumor, with median survival around 15 months despite surgery, radiation, and chemotherapy with temozolomide. Standard treatment hasn't changed substantially in two decades, and the tumor almost always returns. Research is testing approaches that cross the blood-brain barrier, target tumor-specific mutations, and harness the immune system.
What's actually going on in research
Trials are testing CAR-T cell therapies engineered to attack glioblastoma antigens, tumor-treating fields combined with new drugs, vaccines targeting tumor mutations, and drugs that exploit IDH mutations or EGFR amplification. Researchers are also studying convection-enhanced delivery that pumps drugs directly into the tumor, bypassing the blood-brain barrier that blocks most chemotherapy.
CAR-T cell therapy
Modified immune cells are being tested against proteins on glioblastoma's surface, including EGFRvIII and IL13Rα2. Early trials show these cells can reach brain tumors and attack them, though durability remains challenging.
Tumor vaccines
Personalized vaccines made from each patient's tumor mutations aim to train the immune system to recognize cancer cells. Several trials combine these vaccines with checkpoint inhibitors to boost the immune response.
Targeted mutation drugs
About 12% of glioblastomas have IDH mutations, and drugs like vorasidenib are being tested specifically for these tumors. Other trials target EGFR amplification, present in about 40% of cases.
What to know before you search
Eligibility typically depends on tumor genetics (IDH status, MGMT methylation, specific mutations), whether this is newly diagnosed or recurrent disease, performance status, and prior treatments received.
What types of trials are currently open
- Immunotherapy trials — Testing CAR-T cells, vaccines, and checkpoint inhibitors to activate the immune system against glioblastoma. Many combine multiple immune approaches.
- Targeted therapy trials — Testing drugs against specific mutations like IDH, EGFR, or BRAF found in tumor testing. Eligibility requires the matching mutation.
- Drug delivery trials — Testing new ways to get drugs past the blood-brain barrier, including wafers placed during surgery or catheters that pump drugs into the tumor.
- Combination trials — Adding experimental drugs to standard radiation and temozolomide to see if outcomes improve beyond current treatment.
- Recurrent glioblastoma trials — Testing new treatments specifically for tumors that have returned after initial surgery and radiation.
Recently added Glioblastoma trials
Observational Connectomics in Adults Undergoing Resection of WHO II-IV Glioma
This observational study will create a large scale database (registry) of glioma patients' data across multiple participating institutions (hospitals) to study a range of clinical research questions.
A Phase I Clinical Study of YN003 in the Treatment of Glioma
This is a Phase 1, open-label, single-arm, dose-escalation and dose-expansion study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of YN003 Injection in patients with recurrent or progressive glioma. The study consists of Part A (dose escalation using a Bayesian Optimal Interval design) and Part B (dose expansion at the recommended Phase 2 dose).
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