H I G H G R A D E G L I O M A 2 0 2 4



H I G H G R A D E G L I O M A 2 0 2 4
The Pediatric Neuro-Oncology Consortium (PNOC) is leading innovative research and treatment efforts for pediatric brain cancers PNOC020 is an ambitious initiative aimed at developing and testing a novel mRNA vaccine tailored specifically to combat high-grade glioma, a highly aggressive and lethal form of brain cancer in children. This trial is vital because of the severe challenges and limited efficacy associated with current treatment options for this devastating disease. High-grade gliomas are responsible for over 40% of childhood brain tumor deaths.
PNOC020 aims to revolutionize the treatment of high-grade glioma by using an mRNA vaccine that teaches the immune system to recognize and combat cancer cells. Unlike traditional treatments, this vaccine utilizes clusters of mRNA particles administered intravenously, eliciting a strong immune response. Preliminary trials in adults and dogs have highlighted the potential efficacy of this innovative approach, making it a promising alternative for treating high-grade glioma in children.
Our previous trials have shown that using dendritic cell (DC) vaccinations for brain tumors in both adults and children is safe and feasible. However, these vaccines require scale-up synthesis, making it hard to get them to patients quickly. To address these challenges, we are now exploring a new method to more rapidly activate the immune response (within hours) against a patient’s individual cancer.
PNOC FOUNDATION
PNOC020 seeks to transform the treatment landscape for high-grade glioma by offering a highly personalized and effective mRNA vaccine therapy. The trial will be conducted in two phases:
Phase 1: Launching in Q4 of this year, this phase will focus on evaluating the safety and initial efficacy of the mRNA vaccine in pediatric patients.
Phase 2: Building on the findings from Phase 1, this phase will further assess the vaccine's effectiveness and optimize the treatment protocols.
The impact of PNOC020 extends beyond immediate treatment outcomes. By developing a robust and scalable mRNA vaccine platform, this trial aims to set new standards in pediatric oncology, potentially improving survival rates and quality of life for children diagnosed with high-grade glioma. The success of PNOC020 could pave the way for similar personalized therapies for other types of pediatric cancers, thereby revolutionizing the field and offering new hope to countless families.
throughout the body. Localized treatments often fail to address microscopic disease that has spread beyond the primary tumor site, resulting in recurrence. Traditional immunotherapies may target general cancer markers and can be less effective due to their broader approach In contrast, PNOC020’s mRNA vaccine provides a more potent immune response by teaching the immune system to recognize and attack cancer cells, reducing collateral damage to healthy cells and offering a comprehensive approach to eliminating the disease.
The success of PNOC020 is not only dependent on the vaccine itself but also on the establishment of a comprehensive infrastructure to support the trial. This includes setting up manufacturing capabilities for the personalized mRNA vaccines across children’s hospitals, both nationally and internationally. The infrastructure will serve as a template for future trials, ensuring that innovative treatments can be delivered efficiently and effectively. The vaccine production process involves several key steps, including tumor sample collection, RNA extraction, mRNA synthesis, and encapsulation in lipid nanoparticles, ensuring a tailored treatment for each patient.
PNOC is uniquely positioned to lead this effort, with plans already underway to establish manufacturing hubs at leading institutions such as CNMC These hubs will ensure that vaccines are produced to the highest standards and that patients receive the necessary monitoring and care throughout the trial.
cover these expenses, with half of this amount already secured. The funding will be used for:
Manufacturing: Setting up and operating facilities to produce the personalized mRNA vaccines
Clinical Trials: Conducting the trial at multiple sites, including patient enrollment, monitoring, and data collection.
Support Infrastructure: Establishing the necessary infrastructure at participating hospitals to ensure the safe and effective administration of the vaccine.
By supporting PNOC020, donors can play a pivotal role in advancing a groundbreaking treatment for highgrade glioma. This trial not only has the potential to improve outcomes for children with this devastating disease but also paves the way for future advancements in personalized cancer therapies Your investment in PNOC020 will help us bring hope to families and children affected by high-grade glioma and contribute to the broader fight against pediatric cancer.
We extend our heartfelt gratitude to our partners and donors, whose commitment is crucial to our continued success. Your support helps us move closer to our goal of providing innovative and effective treatments for children battling high-grade glioma. CureSearch is the lead funder of this trial.