FORT LAUDERDALE, Fla. (NewsNation) — In a first-ever human clinical trial, researchers at the University of Florida developed an mRNA vaccine that reprograms the immune system to fight the most aggressive and lethal brain tumor.
Glioblastoma has a median survival rate of around 15 months, according to the University of Florida Health, with treatment including surgery, radiation, and some combination of chemotherapy.
Researchers used mRNA technology and lipid nanoparticles, similar to COVID-19 vaccines during the pandemic, to fight “notoriously treatment-resistant cancers.”
However, there were two differences — researchers used patients’ tumor cells to create a personalized vaccine and a “newly engineered complex delivery mechanism within the vaccine,” the University of Florida said in a news release.
Clinical trials show promising results
Scientists and researchers at the University of Florida reported their findings Friday in the journal Cell, a culmination of more than seven years of studies.
The team started their research with pre-clinical mouse models that then turned into a clinical trial of 10 pet dog patients with terminal brain tumors. They lived a median of 139 days, compared to a median survival of 30 to 60 days for dogs with the condition, researchers said.
Following promising results with the dogs, the team advanced the research to a small U.S. Food and Drug Administration-approved clinical trial that involved four human patients.
Researchers said patients lived longer than expected.
“We extended survival in these patients to nine months, eight months, 10 months — they all eventually succumbed. But I have to mention when we started this trial, we started at very low dosing,” Dr. Elias Sayour, an associate professor of neurosurgery and pediatrics at the University of Florida, told NewsNation. “We only gave a few vaccines — two patients got two vaccines; the other two patients got four vaccines. So, we still have a lot left to learn.”
What’s next?
Scientists and researchers said their next step is to test a Phase 1 clinical trial for brain cancer, including up to 24 adults and pediatric patients, to validate their findings.
Researchers said once an “optimal and safe dose” is confirmed, an estimated 25 children would participate in Phase 2.
“This is a breakthrough for two important reasons — we talk about the mRNA vaccine technology … which has been in the making for 30-plus years; this is not new stuff,” said NewsNation medical contributor Dr. Dave Montgomery. “The second part of this, which is really fascinating and very different from COVID, is that they used the tumor biology of each of these patients to make the vaccine more specific to that particular tumor.”
