GEOapplication/xmlftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE327nnn/GSE327062/suppl/filelist.txtftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE327nnn/GSE327062/suppl/GSE327062_RAW.tarftp://ftp.ncbi.nlm.nih.gov/geo/series/GSE327nnn/GSE327062/primaryOK200OtherHomo sapiensOtherhttps://www.ncbi.nlm.nih.gov/geo/query/acc.cgi?acc=GSE327062GEOGSEfalseAdjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase I trialGlioblastoma is a fatal disease with a median prognosis of 12-18 months. Recent studies have shown encouraging results using neoantigen-based vaccines to stimulate glioblastoma-directed immune responses, but overall immunogenicity has been low. Here, we report the results of an open-label, single-arm, phase I clinical trial (GT-20; NCT04015700) to evaluate safety and feasibility (primary endpoints), as well as immunogenicity and preliminary clinical activity (secondary endpoints) of GNOS-PV01 monotherapy, a DNA-based personalized therapeutic cancer vaccine administered following surgical resection and radiation for patients with MGMT unmethylated glioblastoma. The GT-20 study vaccinated 9 patients utilizing up to 40 neoantigens per patient (range 17- 40) without causing any serious adverse events, unexpected toxicities, or dose limiting toxicities. The vaccine induced activation and expansion of circulating peripheral T cells in all evaluated patients, except one being treated with dexamethasone. The secondary endpoint was to evaluate six-month progression-free survival and twelve-month overall survival; each observed in 66.7% of patients. Median progression-free survival was 8.5 months, median overall survival was 16.3 months, and survival at 24 months was 33%, including one long-term survivor still alive four years from the time of initial surgery. This study met the pre-specified endpoints and supports the use of GNOS-PV01 as a potentially impactful component of glioblastoma immunotherapy.2026/04/06GSE327062GSM9647460GSM9647461GSM9647450GSM9647451GSM9647462GSM9647463GSM9647452GSM9647464GSM9647453GSM9647454GSM9647465GSM9647466GSM9647455GSM9647467GSM9647456GSM9647457GSM9647468GSM9647458GSM964745921697327062Homo sapiens