RNA-nanoparticle vaccines for pediatric high-grade glioma

Dr. Elias Sayour – University of Florida Elias Sayour, MD, PhD

CureSearch Catapult Award: 2020-2022 

Focus: High-grade glioma  

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

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.  

Project Update: As of December 2021, Dr. Sayour and his team have created infrastructure for cGMP grade synthesis of mRNA vaccines and allowed creation of an immunomonitoring queue specific to mRNA cancer vaccines. This infrastructure can now be leveraged for conduct of new studies in pediatric cancer. In addition, as this is a first-in-human pediatric clinical trial, the FDA requires the therapy be tested first for safety in three or more adults treated in an ICU. Dr. Sayour has enrolled his first adult patient in the trial and anticipates treatment administration in the first quarter of 2022. 

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