Researchers from Duke University are reporting promising early results from a clinical trial testing a poliovirus-based therapy for glioblastoma, an aggressive form of brain cancer. The findings, published in NEJM Evidence, come from a Phase 1 trial evaluating the safety and efficacy of the experimental treatment known as PVSRIPO.
The trial involved 61 patients with glioblastoma that had returned after initial treatment. All had previously undergone surgery, radiation and chemotherapy. Of these, 61 received a single dose of PVSRIPO infused directly into their tumors.
Although the median overall survival was similar to historical rates at 12.5 months, long-term results showed that 21% of patients survived for three years or more. In comparison, only 4% survival is usually expected at that point, according to the researchers. Notably, all long-term survivors had evidence of a strong immune response triggered by the treatment.
“We are encouraged by these outcomes,” said principal investigator Dr. Darell D. Bigner of the Duke Cancer Institute. “We are not claiming a cure, but the survival benefits for a subset of patients are significant and meaningful.”
The PVSRIPO therapy uses a genetically modified poliovirus engineered to target and kill cancer cells while sparing normal cells. It also stimulates the body’s immune system to recognize and attack the tumor.
Side effects were generally manageable, with inflammation and brain swelling being the most commonly reported issues. The researchers say these findings support further studies, including current Phase 2 trials and immunotherapy combination approaches.
