Childhood Cancer

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Phase I trial of multilamellar mRNA lipid particles for recurrent pediatric high grade glioma

University of Florida
Elias Sayour, MD/PhD
Grant Type: 
R Accelerated Award Grants
Year Awarded: 
Type of Childhood Cancer: 
Brain Tumors
Project Description: 

Due to the killing capacity of the immune system, immunotherapy targeting recurrent pediatric high grade glioma holds significant potential to meet the need for more effective and less toxic treatment options for patients with this disease. We have developed a new mRNA vaccine called RNA-nanoparticles. RNA-nanoparticles are small enough to communicate with the immune system redirecting it against a patient's specific cancer. These nanoparticles will be personalized against a patient's specific brain tumor and the technology is amenable to "off the shelf" manufacturing.

Project Goals

By employing RNA-nanoparticles encoding for tumor specific targets as an innovative and versatile platform, we can reprogram the intratumoral environment of pediatric high-grade glioma into an immune activated state. These particles have been active in mouse brain cancer and in pet dogs with glioma. Based on these data and promising safety data generated to date, we intend to conduct a phase I trial evaluating the safety and activity of this novel mRNA vaccine platform in children with recurrent high-grade gliomas.