A new experimental cell therapy is showing early promise for patients facing glioblastoma, one of the most aggressive and difficult-to-treat brain cancers. In recent phase 1 clinical trial presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, researchers from Penn Medicine reported that nearly two-thirds of patients treated with a novel dual-target CAR T-cell therapy saw their tumors shrink after treatment.
Researchers tested a next-generation cell therapy called CART-EGFR-IL13Rα2 in patients with recurrent GBM whose tumors had certain genetic markers (EGFR amplification). This approach uses highly specialized immune cells, engineered to attack two different proteins on the tumor, and delivers them directly into the fluid-filled spaces of the brain.
Eighteen patients participated in the trial. Results showed that:
- The treatment was generally safe: while just over half experienced manageable grade 3 neurotoxicity (such as headaches or confusion), there were no life-threatening side effects.
- Among patients with measurable tumors, 62% saw their tumors shrink after the cell therapy, with one patient experiencing a significant, ongoing period of stable disease exceeding 16 months.
- For many, the cancer stopped growing for a short time, with the median progression-free survival being nearly two months.
- At the time of reporting, the average overall survival had not yet been reached, with the study ongoing.
These early findings suggest this new type of CAR T-cell therapy is both feasible and potentially effective for patients with very limited treatment options. More research and longer follow-up are needed, but there is real optimism as clinical trials continue to advance.
Reference:
Bagley SJ, Desai AS, Fraietta JA, et al. Intracerebroventricular bivalent CAR T cells targeting EGFR and IL-13Rα2 in recurrent glioblastoma: a phase 1 trial. Nat Med. 2025 Jun 1.
