A genetically modified poliovirus therapy developed at The Preston Robert Tisch Brain Tumor Center shows significantly improved long-term survival for patients with recurrent glioblastoma, with a three-year survival rate of 21 percent in a phase 1 clinical trial.
Comparatively, just four percent of patients at Duke with the same type of recurring brain tumors were alive at three years when undergoing the previously available standard treatment.
Phase 1 clinical trial results of the poliovirus therapy are being presented June 26 at the 22nd International Conference on Brain Tumor Research and Therapy in Norway and simultaneously published in The New England Journal of Medicine.
“Glioblastoma remains a lethal and devastating disease, despite advances in surgical and radiation therapies, as well as new chemotherapy and targeted agents,” said Darell D. Bigner, MD, PhD, emeritus director of The Preston Robert Tisch Brain Tumor Center at Duke and senior author of the study.
“There is a tremendous need for fundamentally different approaches,” Bigner said. “With the survival rates in this early phase of the poliovirus therapy, we are encouraged and eager to continue with the additional studies that are already underway or planned.”
Bigner and colleagues -- including co-senior author David Ashley, PhD, MBBS, and co-lead authors Annick Desjardins, MD, and Matthias Gromeier, MD. all in the Department of Neurosurgery – reported median follow-up of 27.6 months in the phase 1 trial, which was launched in 2012 with a young patient who was just entering nursing school. She has since married and works as a registered nurse.
The therapy includes a genetically modified form of the poliovirus vaccine, which is infused directly into the brain tumor via a surgically implanted catheter. Developed by Gromeier in his lab at Duke, the modified virus preferentially zeroes in on tumor cells, igniting a targeted immune response. Gromeier and co-authors recently published a study in Science Translational Medicine describing the mechanism of action for the poliovirus therapy.
An 18-year collaboration with the National Cancer Institute’s (NCI) Experimental Therapeutics (NExT) Program, NCI, part of the National Institutes of Health, and the U.S. Food and Drug Administration enabled the pre-clinical and translational phases of development of this novel therapy for glioblastoma.