Vaccine triggers fierce response to fight brain tumor

By Michelle Koidin Jaffee

In a first-ever human clinical trial of four adult patients, an mRNA cancer vaccine developed at the University of Florida quickly reprogrammed the immune system to attack glioblastoma, the most aggressive and lethal brain tumor.

Dr. Elias Sayour and colleagues in the lab
(From left) Dr. Elias Sayour, Chong Zhao and Arnav Barpujari discuss the mRNA cancer vaccine developed at the University of Florida.

The results mirror those in 10 pet dog patients suffering from naturally occurring brain tumors whose owners approved of their participation, as they had no other treatment options, as well as results from preclinical mouse models. The breakthrough now will be tested in a Phase 1 pediatric clinical trial for brain cancer.

Reported May 1 in the journal Cell, the discovery represents a potential new way to recruit the immune system to fight notoriously treatment-resistant cancers using an iteration of mRNA technology and lipid nanoparticles, similar to COVID-19 vaccines, but with two key differences: use of a patient’s own tumor cells to create a personalized vaccine, and a newly engineered complex delivery mechanism within the vaccine.

“Instead of us injecting single particles, we’re injecting clusters of particles that are wrapping around each other like onions, like a bag full of onions. And the reason we’ve done that in the context of cancer is these clusters alert the immune system in a much more profound way than single particles would,” said senior author and MBI researcher Elias Sayour, M.D., Ph.D., a UF Health pediatric oncologist who pioneered the new vaccine, which like other immunotherapies attempts to “educate” the immune system that a tumor is foreign.

Read the full UF Health news release.

Read an article about the study in The Conversation.

Read the paper in Cell.

Researchers collaborating in Dr. Sayour's lab
Group of researchers in Sayour lab collaborating
Researcher in Sayour lab doing research
Researchers in Sayour lab point at screen
Researcher examines liquid
researchers reviewing brain scans in a lab setting
Doctor Sayour discussing the findings with his lab
doctor Sayour presenting the work of his lab
Group photo of everyone in the lab