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R33M is privileged to contribute to advancing the incredible work of researchers, scientists, physicians and all practitioners in the field of pediatric brain cancer research. The tireless work of thousands of individuals dedicating their academic and professional lives to solving the mysteries of childhood brain cancer in order to give diagnosed children the longest, most healthy lives possible is an admirable calling. Unfortunately, their work is not given the support that it needs to accomplish everything required to stop the devastating outcomes so many children realize. This is why R33M is tirelessly devoted to raising awareness and money for pediatric brain cancer research. It is the only way outcomes will change. 

Highest-Risk Medulloblastoma


William A. Weiss, MD, PhD

University of California, San Francisco

R33M funded this 3-year research to generate a humanized mouse model and establish a preclinical basis for a clinical trial in children with this incurable form of medulloblastoma. This study was based on the hypothesis that MYCN cooperates with gain of GLI2 and with loss of P53 to drive medulloblastoma resistant to current standard therapies, and that modeling this highest-risk subtype of MB offers opportunities to identify effective therapies.

The goal of this research is to establish a human medulloblastoma tumor model that will provide an in-vivo platform that will rapidly identify efficacious targeted clinical drugs for children with this diagnosis.

The therapeutic progress report shared insights of proceeding long term study to identify which genes can be targeted to kill the tumor cells, while sparing a non-tumor cell. This is believed to be the most exciting aspect of the project to study date.

Phase 1 Vaccine Trial for high-grade glioma and ependymoma


Jason Fangusaro, MD, Attending Physician

Ann & Robert H. Lurie Children’s Hospital of Chicago, IL

R33M supported this Trial for a vaccine to boost the body's immune system to fight against tumor growth or recurrence for children. The vaccine was produced individually for each patient using his/her own resected tumor tissue. The Phase I and Feasibility Trial was to ensure the safely and feasibility of the vaccine. A total of 11 patients, ranging in age from 9 to 22 years of age,  were enrolled, and the treatment showed limited toxicity with evidence of extended disease stability. 

The final analysis showed that the vaccine was not able to maintain an immune response in the patients who received it and the study was suspended at the end of 2019. Although this trial ultimately did not bring benefit to those patients, it was able to provide valuable insight about the safety and feasibility of this immunotherapy treatment. This knowledge helped inform a new Phase I vaccine trial for DIPG. R33M also supported that trial.

Children's Brain Tumor Network

Children's Hospital of Philadelphia

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The mission of CBTN is to accelerate childhood brain tumor research to faster cures by working together to collect and share resources, to save more children. CBTN has over 30 types of pediatric brain and spinal cord tumor clinical and molecular data, biospecimens, and cell-lines available at no cost to academic researchers. Their open science model has shortened research time by up to 20 years.

Since 2017, R33M has been a partner to CBTN and its vision and work. We are a member of its Executive Council and work closely to understand the current and future needs in order to contribute with impact. R33M has contributed financially to the operations of CBTN directly to support the infrastructure of the critical day to day work that must be kept on pace.

In addition, R33M has played a key role in financing the onboarding of institutions into the CBTN membership process of collecting tumor tissue and sending that to CBTN for central storage and universal access, as well as the data management. Our support has included work with Texas Children's, Houston, Lucile Packard Children's Hospital, Stanford, and Children's Healthcare of Atlanta. 

The CBTN has 33 institutions with 8 more in the process of onboarding, representing 5 countries thus far. The benefits of sharing knowledge across this many points of expertise, with so many experts working daily to solve these problems is finally lifting the curtain of time off of the normally slow pace of research. CBTN's process is getting to answers and R33M is so honored to be a part of that work.

DIPG Vaccine Clinical Trial


Ashley Plant, MD

Ann & Robert H. Lurie Children’s Hospital of Chicago, IL

Following the Phase I HSPPC-96 Vaccine therapy trial, the knowledge gained from that study helped inform a new Phase I vaccine trial for DIPG. R33M supported this trial to continue the learnings gained to date and to further the benefits to our children. 

This study sought to test a heat shock protein vaccine made specifically for DIPG. The vaccine will be given to patients in combination with two additional drugs to help overcome the resistance that the DIPG tumor cells may have to the immunotherapy. 

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