In two early trials, blood cancer treatment appears promising for deadly brain tumor

Recent trials have demonstrated promise in treating glioblastoma, an aggressive brain cancer with a bleak prognosis and no cure. Two small-scale trials showcased the potential of personalized immunotherapy, specifically CAR-T therapy, in patients with recurrent glioblastoma.

Both trials focused on modifying CAR-T therapy to target multiple proteins commonly found in glioblastoma tumors. While initial results were encouraging, including significant tumor shrinkage, researchers emphasize the need for further investigation to determine long-term efficacy.

One trial, led by Dr. Marcela Maus, employed CAR-T cells targeting two proteins, EGRF and its variant, delivered directly into the cerebrospinal fluid. This localized approach aimed to enhance therapeutic efficacy while minimizing systemic side effects.

In another trial, led by Dr. Donald O’Rourke, CAR-T cells targeted EGFR and IL13Rα2, injected directly into the cerebrospinal fluid following tumor-shrinking radiation. Patients experienced tumor reduction and increased CAR-T cell presence in spinal fluid, suggesting therapeutic potential.

Despite promising initial responses, durability remains a key question. Neurological side effects, while manageable, underscore the complexity of CAR-T therapy administration.

These trials underscore the evolving landscape of glioblastoma treatment, with ongoing efforts to optimize CAR-T therapy combinations and explore adjunctive treatments like radiation and chemotherapy. Collaboration among researchers fuels optimism for advancing glioblastoma therapies, although challenges persist in achieving lasting outcomes.