CAR-T shows tumor control in phase 1 glioblastoma trial

A catheter carried modified immune cells directly into brain tumors in a phase 1 trial involving 15 patients with recurrent glioblastoma. The B7-H3-targeting CAR-T treatment was associated with tumor control in eight participants, including one complete remission, while the researchers focused primarily on safety.

CAR-T — short for chimeric antigen receptor T-cell therapy — uses a patient’s own T cells, a type of immune cell. Researchers enriched those cells from a blood sample, introduced a gene that helps them bind to B7-H3, and returned them to the patient. B7-H3 is found at high levels in most glioblastomas, while the blood-brain barrier has limited the reach of some other emerging cancer treatments.

The standard approach to glioblastoma combines surgery and chemotherapy, but the cancer returns in most cases, often in a more aggressive and treatment-resistant form. For recurrent disease, median life expectancy is less than a year, leaving many patients with few options. The trial’s tumor responses lasted as long as three years in some participants.

The treatment was generally tolerated, with side effects such as fever, vomiting and itchy skin. Three participants experienced more serious effects, including increased intracranial pressure and epilepsy; the researchers say these events were manageable with clinical intervention. Because severe side effects were more often linked to the highest dose, the team identified the second dose level for future research.

So what changes for patients now? Not standard care yet, but the treatment is moving from an initial human study into a larger clinical trial for people whose recurrent tumors have resisted available therapies. The researchers are pursuing a phase 2 trial to examine efficacy in more patients. The evidence remains limited: these results come from a small phase 1 cohort.