New Treatments & Clinical Trials for Glioblastoma

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.

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.