Several studies have shown that neoantigen-based vaccines can stimulate immune responses against glioblastoma, although their overall immunogenicity has remained low. To improve the efficacy of glioblastoma vaccines, a phase 1 clinical trial evaluated GNOS-PV01 monotherapy, a DNA-based personalized vaccine, in nine patients with newly diagnosed MGMT (O6-methylguanine methyltransferase)-unmethylated glioblastoma following surgical resection and radiation therapy. The vaccine induced activation and expansion of circulating peripheral T cells in all patients except one who was receiving dexamethasone treatment.

Notably, 6-month progression-free survival (PFS) and 12-month overall survival (OS) were each observed in 66.7% of patients. Median PFS was 8.5 months, while median OS was 16.3 months. Importantly, 33% of patients survived at 24 months, including one long-term survivor who remained…