Glioblastoma is the most aggressive brain tumor in adults, with about 12,000 new cases each year in the US. Standard treatment combines surgery, radiation, and temozolomide chemotherapy, but the tumor nearly always returns. Median survival remains around 15 months, making glioblastoma one of the most urgent unmet needs in oncology.
What's actually going on in research
Trials are testing tumor-treating fields combined with new drugs, vaccines that train the immune system to recognize tumor cells, viral therapies that infect and kill cancer cells, CAR-T cells engineered to attack glioblastoma, and drugs targeting IDH mutations in a subset of patients. Researchers are also studying tumor metabolism, the blood-brain barrier as an obstacle to treatment, and why glioblastoma becomes resistant to therapy.
Personalized vaccines
Several trials create custom vaccines from each patient's tumor, teaching their immune system to recognize cancer cells. Early studies show some patients living years longer than expected.
Oncolytic viruses
Engineered viruses are injected directly into tumors, where they infect and destroy cancer cells while activating immune responses. Multiple virus types are now in human trials.
CAR-T therapy
Immune cells from patients are modified to attack proteins on glioblastoma cells, then infused back. Early trials show the cells can reach brain tumors and trigger responses.
What to know before you search
Eligibility typically depends on whether the tumor is newly diagnosed or recurrent, molecular markers like IDH and MGMT status, prior treatments, and performance status.
What types of trials are currently open
- Immunotherapy trials — Testing vaccines, checkpoint inhibitors, or CAR-T cells that aim to activate the immune system against glioblastoma.
- Targeted therapy trials — Testing drugs aimed at specific mutations like IDH1, EGFR amplification, or other molecular changes found in tumor tissue.
- Combination trials — Testing whether adding new drugs to standard temozolomide and radiation improves outcomes or delays recurrence.
- Device trials — Studies of tumor-treating fields, implantable wafers, or convection-enhanced delivery systems that get drugs past the blood-brain barrier.
- Recurrent glioblastoma trials — Testing treatments specifically for tumors that have returned after initial therapy, where options are limited.
Recently added Glioblastoma trials
[177Lu]Lu-DOTA-EB-RGD2 Therapy in Patients With Recurrent High-grade Glioma
This is an investigator-initiated, Phase I clinical trial. It aims to evaluate the safety, tolerability, dosimetry, and preliminary anti-tumor activity of a novel radiopharmaceutical, \[177Lu\]Lu-DOTA-EB-RGD2, in patients with recurrent high-grade gliomas. Participants will receive the drug either via intravenous infusion or directly into the tumor cavity through a pre-implanted Ommaya reservoir (a subcutaneously placed device that allows direct access to the tumor cavity). The study employs a "3+3" dose-escalation design to determine the maximum tolerated dose (MTD). Adverse events, biodistribution, and tumor response (by MRI) will be assessed. Approximately 24 patients will be enrolled across two major Chinese medical centers: Beijing Tiantan Hospital and Peking Union Medical College Hospital.
Dosimetry, Safety, and Efficacy Study of [177Lu]Lu-XT771 in Patients With Recurrent Glioblastoma
The primary objective of this study is to evaluate the dosimetry, safety, and tolerability of the investigational radiopharmaceutical \[177Lu\]Lu-XT771 in patients with recurrent glioblastoma, an aggressive form of brain cancer. \[177Lu\]Lu-XT771 is designed to specifically target and deliver beta radiation directly to tumor cells that overexpress carbonic anhydrase IX and XII (CA IX and CA XII). This early-phase, investigator-initiated trial will enroll a small group of approximately 3-5 patients, each receiving a single dose of \[177Lu\]Lu-XT771. The drug will be administered locoregionally via an implanted Ommaya reservoir, directly into the tumor cavity. Following administration, patients will be closely monitored using single-photon emission computed tomography/computed tomography (SPECT/CT) to assess the biodistribution of the drug and to quantify the absorbed radiation dose to both the tumor and normal organs. The study will also document all adverse events to characterize the safety profile of the treatment and will provide a preliminary assessment of its anti-tumor activity, as measured by progression-free survival. The information gathered from this exploratory study will be used to determine the recommended safe starting dose for future Phase I clinical trials.
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