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Phase 1b Clinical Trial for Genetically Modified Poliovirus Therapy Enrolling Children and Teens at Duke University


At a presentation given today at the International Symposium on Pediatric Neuro-Oncology (ISPNO), David M. Ashley, PhD, FRACP, MBBS of Duke University, shared that a genetically modified poliovirus therapy developed at Duke Cancer Institute is now being tested to treat pediatric brain tumors.

Phase 1b Study PVSRIPO for Recurrent Malignant Glioma in Children is currently enrolling children and teens ages 12-18 after showing significantly improved long-term survival for adults with recurrent glioblastoma.

The Phase 1 study in adults showed a three-year survival rate of 21 percent. This compares to a three-year survival rate of four percent in adult patients with the same type of recurring brain tumors who underwent the previously available standard treatment. Those results were presented June 26 at the 22nd International Conference on Brain Tumor Research and Therapy in Norway and 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, in Duke's press release on the study's results. “There is a tremendous need for fundamentally different approaches. 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.”

Ashley, who was a co-senior author on the Phase 1 study in adults and is one of the principal investigators in the pediatric study, echoed Bigner's quote during his ISPNO presentation and indicated there’s potential for this therapy to work even better for pediatric patients.

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 Matthias Gromeier, MD in his lab at Duke, the modified virus preferentially zeroes in on tumor cells, igniting a targeted immune response. The purpose of the Phase 1b study is to confirm the safety of the selected dose and potential toxicity of oncolytic poliovirus (PV) immunotherapy with PVSRIPO for pediatric patients.

The Pediatric Brain Tumor Foundation has long been a supporter of Duke's pediatric brain tumor research through our Institute Grant program, and we are excited by the promise immunotherapy research holds for children and teens battling brain tumors, the deadliest form of pediatric cancer.

Learn more about Duke's Phase 1b study here, and read more about the PBTF's new Immunotherapy Research Initiative here.