Dr. Elias Sayour – RNA-NANOPARTICLE VACCINES FOR PEDIATRIC HIGH-GRADE GLIOMA

Elias Sayour, MD, PhD

University of Florida

CureSearch Catapult Award: 2020-2022

Focus: High-grade glioma

Project Title: RNA-nanoparticle vaccines for pediatric high-grade glioma

About Dr. Sayour

Elias Sayour, MD, PhD, is an Associate Professor of Neurosurgery, and Pediatrics, as well as Principal Investigator of the RNA Engineering Laboratory within the Preston A. Wells, Jr. Center for Brain Tumor Therapy and UF Brain Tumor Immunotherapy Program, and Co-Leader of the UFHCC Cancer Therapeutics & Host Response. He completed undergraduate training at Fordham University, received his medical degree from the University at Buffalo, and his doctorate from Duke University.

After finishing his pediatric residency at the Cohen’s Children’s Medical Center of New York at North-Shore-LIJ, he completed a hematology-oncology fellowship at Duke University Medical Center before accepting a faculty position at the University of Florida. Dr. Sayour is currently an NIH funded investigator, a University of Florida Term Professor, and a board-certified pediatrician and oncologist. He is working to develop lipid-nanoparticles to train the immune system to fight cancer, and has received many awards for his work including the U.S. Department of Defense Cancer Research Award, the American Brain Tumor Association Discovery Award, and Hannah’s Hereos St. Baldrick’s Scholar Award. Dr. Sayour has spearheaded new paradigm treatments currently being tested in canine (pet dog) patients with terminal brain cancer, and also serves as Principal Investigator/Study Chair on first-in human immunotherapy studies for children with aggressive brain cancers.

Project Overview: Primary high-grade gliomas (pHGGs) in children are almost uniformly lethal. While there is promise for immunotherapy, gliomas are overwhelmingly immunosuppressive, posing an access and activity challenge for the immune activation required for immunotherapy. Dr. Sayour and colleagues have developed a novel treatment platform which leverages the use of nanoparticles (NPs) combined with messenger RNA (mRNA) taken from the patient’s tumor to make a product that functions as both a vaccine and an agent that is capable of increasing activity of the immune system. To facilitate translation of this promising technology into first-in-human studies for pHGG, Dr. Sayour and colleagues have partnered with the Pacific Pediatric Neuro-Oncology Consortium (PNOC), where their clinical trial concept was accepted for multi-institutional trial development.

Final Research Update:

Dr. Sayour’s Catapult Award supported the clinical translation of a novel, personalized immunotherapy designed to overcome the powerful immune suppression seen in pediatric high-grade gliomas. His team developed an innovative RNA vaccine platform that uses messenger RNA derived from a patient’s own tumor, packaged with specialized nanoparticles, to both activate the immune system and reprogram the tumor microenvironment so immune cells can function more effectively. With support from CureSearch, this approach advanced to first-in-human testing, demonstrating that a single vaccine dose can rapidly trigger tumor-specific immune response in patients within hours. The project also established the manufacturing, quality control, and distribution infrastructure needed to deliver these personalized vaccines across multiple institutions. As a result, the pediatric arm of this multi-center Phase I/II clinical trial is now positioned to open nationally through PNOC, marking a critical step toward making this promising therapy available to children with otherwise lethal brain tumors.

September 2024 Research Update:

The team treated a fourth patient, a young adult, on the expanded phase I trial and observed a significant immune response after the vaccine was administered. The pediatric portion of the clinical trial has been approved by the IRB and it is open to enrollment at the University of Florida, prior to opening at various PNOC sites.

September 2022 Research Update:

In early September 2022, Dr. Sayour validated a starting phase 1 dose level for pediatric patients, and is now seeking to enroll pediatric glioma patients at the University of Florida. The project is on track to then enroll pediatric patients throughout the country shortly thereafter in collaboration with the Pacific Pediatric Neuro-Oncology Consortium (PNOC).

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