A Targeted Approach to Medulloblastoma Treatment

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Children's Brain Tumor Network


Through its #cureMEdullo initiative, the Carson Leslie Foundation (CLF) will fund two new projects in collaboration with the Children’s Brain Tumor Network (CBTN) in pursuit of better outcomes for children with medulloblastoma.

The first is a precision medicine clinical trial that will bring together three innovative methods to inform care, never-before used in concert to treat aggressive forms of medulloblastoma. Patients will benefit from genomic and expression analysis at the time of diagnosis. Data specific to the patient’s tumor will be analyzed against CBTN’s Pediatric Brain Tumor Atlas, the world’s largest clinically annotated data resource for pediatric brain tumors. This is a precision strategy that has not before been leveraged for medulloblastoma patients. The analysis will be paired with an innovative new method for monitoring and adjusting drug treatment strategies in real-time.

Strengthening this project is the incorporation of the CNS-TAP technology, which employs a data-matching algorithm to analyze up-to-the minute pre-clinical and clinical data against the latest published findings of available drug therapies, down to the specific conditions in which each therapy was used. This three-pronged approach will allow doctors an unprecedented level of clarity in following the best therapy strategy tailored to each child. All data generated through this process will be added to the Pediatric Brain Tumor Atlas, providing crucial data for future patients.

A second project supported by CLF, in partnership with Wylie’s Day Foundation, will support pre-clinical medulloblastoma research involving specialized xenograph models. CBTN’s pre-clinical program focuses on the development and characterization of tumor “avatars,” or models, that can be utilized both in vitro and in vivo. Models are generated directly from live tumor tissue such that they can recreate part of the tumor’s biology. In addition to using the models to study tumor biology, this program provides an opportunity for drug testing and the development of clinical trials. To secure FDA approval for a clinical trial, therapies need to be tested to determine if they target and kill the specific subtype of tumor while ensuring safety for use in children. The best way to test drugs for efficacy and safety is to use tumor samples that are “alive,” which are grown from tumor tissue samples and behave the way a tumor might inside the body. These tools are a valuable resource and are incredibly labor intensive to develop and maintain.

Cultivating and characterizing these specially designed mouse models will allow researchers to generate precious new data that will inform individualized treatment strategies for every patient, every time.

The Carson Leslie Foundation is an invaluable partner for the Children’s Brain Tumor Network and draws on a fierce commitment to supporting research that will deliver better and less toxic treatments for our children – as swiftly as possible. Through this partnership, CLF and CBTN build upon a shared vision of a world where no child suffers from these devastating illnesses.

About Carson Leslie Foundation and #cureMEdullo

The Carson Leslie Foundation is dedicated to honor & fulfill Carson’s wish to support research leading to a cure for pediatric cancer and enrich the lives of teens in the battle. CLF’s #cureMEdullo initiative is designed to catalyze the Medulloblastoma community by working across research consortia network, pediatric brain tumor foundations, treating clinicians and their institutions, industry, and most importantly- Medulloblastoma patients and their families as the “one place” reflect the state-of-the-art and needs of the disease.

carsonlesliefoundation.org | curemedullo.org

Collaborative Labs:

Wechsler-Reya Lab at Sanford Burnham Prebys

Led by Dr. Robert Wechsler-Reya, The Wechsler-Reya La, studies the signals that control cell growth and differentiation in the nervous system and how these signals are dysregulated in brain tumors. They focus on medulloblastoma, the most common malignant brain tumor in children, and use models to understand the disease and to develop novel approaches to therapy. Their current areas of interest include: discovering oncogenic drivers and creating new models; elucidating the molecular mechanisms of metastasis; identifying new therapeutics and approaches to drug delivery; harnessing the immune system to target tumors. They also work closely with physicians at Rady Children’s Hospital and elsewhere to translate our findings into trials that can benefit patients. Their goal is to develop safer and more effective therapies for children with brain tumors.


Koschmann Lab at University of Michigan

Led by Dr. Carl Koschmann, the Koschmann lab is studying the molecular mechanisms by which mutations in pediatric High-Grade Glioma (HGG) and Diffuse Intrinsic Pontine Glioma (DIPG) can be therapeutically targeted. The Koschmann Lab’s work in precision medicine pushes the boundaries of targeted therapies and precision diagnostics for children diagnosed with these brain tumors. Through highly collaborative basic, translational, and clinical research, the Koschmann Lab strives towards improving outcomes and providing hope for children fighting these devastating diseases.


Center for Data Driven Discovery in Biomedicine (D3b) at Children’s Hospital of Philadelphia

The Center for Data Driven Discovery in Biomedicine (D3b), led by Dr. Adam Resnick, Dr. Jay Storm, and Jena Lilly, is a translational biomedical research Center of Emphasis at the Children’s Hospital of Philadelphia Research Institute that creates large scale data and specimen resources, tools, and services that can be used to inform treatment and accelerate research activities on a global scale. D3b’s multi-disciplinary expertise is accelerating bench-to-bedside research on behalf of children diagnosed with cancer and other rare conditions. D3b’s seven collaborative units bring together experts in oncology and basic research, genomics, data science, bioinformatics, neurosurgery, and other research-related disciplines to discover breakthroughs for every child, every time, everywhere.


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