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Personalized DNA Vaccine Shows Early Promise in Glioblastoma Trial

The approach targets many tumor-specific mutations at once.

Overview

  • The Nature Cancer paper reports a nine-patient phase 1 study of GNOS-PV01 that found the vaccine was safe and activated the immune system in glioblastoma.
  • Researchers at Washington University and Mass General Brigham ran the single-arm trial at Siteman Cancer Center and saw increased immune-cell activity in all participants except one on an immune-suppressing steroid.
  • The DNA vaccine encodes up to about 40 patient-specific neoantigens chosen by a Washington University algorithm, aiming to make tumors visible to immune attack.
  • Clinical signals were encouraging in this small cohort, with two-thirds progression-free at six months, two-thirds alive at one year, two-thirds alive at two years, and one patient recurrence-free nearly five years after diagnosis.
  • Investigators emphasize that larger, controlled studies are needed and say combination-therapy trials using this personalized platform are underway to test whether outcomes can improve.