FAQs

CBTN turns donated tissue and biospecimens into a powerful, global research engine. Instead of limiting access, we make the world’s largest collection of pediatric brain tumor samples, preclinical models, and paired clinical and molecular data freely available to researchers everywhere.

Our open-access, cloud-based platform housed within the Gabriella Miller Kids First Data Resource Center (link) delivers harmonized, richly annotated data—removing traditional barriers and accelerating scientific discovery.

“Samples” refers to the biospecimens donated from patients and their families, including tumor tissue, blood, and saliva. These can be processed into DNA, RNA, and protein, so investigators can request exactly what they need for their research.

Pediatric brain tumors are rare and remarkably complex, with more than 125 known types. Tissue samples help researchers understand tumor biology, uncover treatment targets, and identify therapies that may already exist.

Paired with blood or saliva and linked to molecular and clinical data, each sample becomes a reusable data source rather than a one-time resource. The more we collect, the more answers researchers can pursue.

These samples help researchers distinguish between genetic changes caused by the tumor and those that are inherited or naturally occurring. They also fuel biomarker discovery, advancing efforts to diagnose and guide treatment using blood or spinal fluid instead of invasive procedures.

CBTN collects samples and clinical information across a child’s entire care journey, not just at a single surgery. If a tumor recurs or progresses, new tissue is collected. We also collect associated blood and spinal fluid samples and follow up on clinical outcomes at set intervals from 6 months through 10 years. This creates a rich, time-based view of how tumors evolve, how treatments change, and how children’s health progresses over time.

At every CBTN member institution, a dedicated study team, including neurosurgeons, neuro-oncologists, neuropathologists, and clinical research coordinators, oversees patient enrollment and data collection. Clinical research coordinators consent families, explain the project, and extract key clinical information from electronic health records, entering it into the CBTN Pediatric Brain Tumor Atlas (PBTA) database.

Because this work spans a child’s entire care journey, it is highly detailed and largely manual in nature. To scale responsibly, CBTN is piloting secure, automated methods for pulling clinical data directly from hospital systems, improving accuracy, consistency, and sustainability.

CBTN collects the most meaningful data points for research: dozens of fields chosen by clinicians, scientists, and the CBTN Clinical Data Working Group.

These include demographics, medical and family history, tumor pathology, diagnosis, tumor location, metastases, testing results, treatments, imaging, and any specimens shared with CBTN.

CBTN also tracks each patient throughout their care journey, capturing data at progression, recurrence, and when appropriate, the end of life.Fields are continuously reviewed and updated as clinical best practices evolve. To request a blank case report form, contact CBTN at research@cbtn.org.

Longitudinal clinical data, paired with molecular, imaging, and biospecimen data, create a time-based view of disease. This comprehensive perspective helps researchers:

+See how tumors progress or become treatment-resistant
+Identify differences between patients with the same diagnosis
+Develop predictive models and algorithms that guide precision medicine

One snapshot can’t tell the whole story. Longitudinal data reveals the full picture.

MRI scans are among the most important tools used to monitor a child’s brain tumor over time. These images track growth, response to treatment, and signs of progression.

When paired with molecular and clinical data, MRIs become even more powerful, fueling AI and machine-learning tools that can detect patterns, improve diagnoses, and inform future treatments.

CBTN prioritizes the collection of longitudinal imaging from multiple medical centers to accelerate the development of imaging-based insights and precision medicine approaches.

Molecular and genomic data reveal the mutations and biological changes driving a child’s tumor. Understanding these changes helps researchers identify targets for existing or new therapies and repurpose treatments from other cancers that share similar mutations. This data also helps researchers understand connections between rare developmental disorders and cancer predisposition.

By pinpointing what’s happening at the molecular level, researchers can pursue smarter, more precise treatment strategies.

Multimodal data combines multiple types of information—genomic, molecular, imaging, pathology, and clinical data—to give a more complete view of a tumor and its behavior. Analyzing these data together helps researchers understand how tumors develop, grow, and resist treatment. It also aids in the discovery of biomarkers for diagnosis or monitoring and is used to build personalized treatment models. CBTN platforms, including CAVATICA, Flywheel, and cloud-based portals, support this complex analysis and enable collaboration across disciplines and institutions.

CBTN’s database includes a wide range of molecular and genomic data, although not every patient has data for every type. Current datasets include:

• Whole Genome Sequencing (WGS)
• Whole Exome Sequencing (WES)
• RNA Sequencing (RNA-seq)
• Single-cell and single-nucleus RNA-seq
• Proteomics and phosphoproteomics
• DNA methylation

Not yet. Generating deep molecular datasets is costly and not covered by insurance, so CBTN relies on partnerships and philanthropy to produce large-scale multimodal data.

Working with private donors and the National Institutes of Health (NIH) and National Cancer Institute (NCI), we have generated multimodal data for thousands of specimens and continue to expand these efforts.

Our goal is comprehensive, holistic data for every patient. Philanthropic support accelerates this work; more information is available on our donation page (LINK).

Having a child who is facing an incurable brain tumor is a devastating reality no family should endure. Choosing to donate brain tissue at the time of a child’s passing is a deeply personal decision.

CBTN partners with Gift From a Child (GFAC), a national initiative that helps families explore tissue donation and guides them through a simple, respectful process.

For more information, including how to connect with a GFAC Tissue Navigator, visit Gift from a Child here.

Post-mortem tissue offers insights that cannot be gained in any other way. It enables scientists to understand how a tumor has evolved after all available treatments and study tumor cells at recurrence or during treatment failure. Tissue donation also provides access to normal brain tissue samples that cannot be collected during life, as well as from inoperable tumor tissue where no surgical sample was possible.

Collecting tissue across the full course of a child’s illness, from diagnosis to progression and post-mortem, creates the most complete picture of the disease. These donations help researchers uncover how tumors evolve and support the development of more effective, targeted therapies for children yet to be diagnosed.

Children and young adults diagnosed with a brain tumor, as well as their parents, are eligible to participate in CBTN. These participants come from diverse backgrounds and communities across the globe.

The majority of CBTN participants are treated at CBTN member institutions.

If your child has been diagnosed with a brain tumor and is currently being treated at one of our institutions, they may be eligible to participate in the CBTN and ensure that their tissue is collected. Please ask your child’s doctor for more information. Your child’s physician can describe the donation process, obtain your consent to donate samples, and answer any additional questions.

If your child is not currently being treated at one of the CBTN member institutions, they may still be able to contribute samples to the CBTN. To request an out-of-network donation, please contact a member of our research team via email and we will follow up to clarify any questions and coordinate the donation.

We extend our heartfelt gratitude to the many generous and committed families participating in CBTN. Together, we are making pioneering progress in pediatric brain tumor research.

If you or your child is/was treated at one of the CBTN member institutions, you may have consented to participate in CBTN. Families of patients from CBTN member institutions can connect with their treating clinicians directly or reach out to the Operations Center to determine if their child is currently part of the study. If your hospital was not a member of CBTN when your child was treated but has since joined and you’d like your child’s data incorporated into the study, please reach out to the Operations Center to discuss next steps.

CBTN covers all costs associated with the donation of specimens or data by patient families. CBTN is funded primarily by philanthropy, receiving financial support from individuals, foundations, and other institutions to cover the operational expense of gathering biospecimens and data that is shared with researchers around the globe at no cost to them. To support us in this effort, visit our donation page.

The samples and information will be stored at CBTN’s operations center, located at the Center for Data Driven Discovery at the Children’s Hospital of Philadelphia (CHOP). All information that identifies the patient is removed. The deidentified samples and information are then used by researchers across the world.

Tissue submitted to CBTN is what remains after the tissue collected from a biopsy or resection is used for the child’s care. Many families give permission for these unused portions of tissue to be submitted to CBTN with the hope that new knowledge might help other patients in the future. Patient families can request tissue that might still be available from their submission by contacting  CBTN at research@cbtn.org.

Patient privacy is a core priority. Biospecimens and corresponding data donated to CBTN will never include information that can be linked to you or your family.

The pathology department where a patient is treated is responsible for determining the condition of each patient’s disease (whether a tumor is benign or malignant). Once a diagnosis is confirmed, that information is relayed to their doctor, who will review the results. CBTN research is not part of your child’s diagnosis process.

Patient families should always consult with their doctors about participating in clinical trials. If any clinically significant results are found, your clinician will be contacted, and further testing on the sample will be performed if necessary. Our updated consent also allows for each patient to be contacted, through their clinicians, should we find any clinically significant results. Note that per federal regulations for research studies, information will be sent to the patient’s clinician to be shared directly with the patient and their family. To be reconsented on the newest consent or to confirm which consent you have in place, please contact CBTN at research@cbtn.org. Families can also learn about genetic links from the published findings of CBTN research projects.

CBTN is a multi-institutional research study with the goal of collecting and creating large-scale aggregate resources for researchers to access globally. CBTN’s platforms are built to support the integration and queriability of deidentified data in ways that harness “Big Data” technologies across as large a number of patients as possible. These platforms are being leveraged to support clinical decision making in the setting of clinical trials, but identifiable, patient-specific views of research data is not currently supported. Patients and their families interested in viewing their personal clinical data should request any clinical information directly from their physicians.

Historically, research-grade data was not permitted to be returned to patients and their families due to federal regulations and guidelines. The Operations Center has recently worked with the CHOP Institutional Review Board to update the consent language to allow for an enrolled patient to request and review data generated under the CBTN’s research protocol via approved workflows. This consent update is currently being disseminated for review and updating by CBTN site members.

Yes, the doctor who is leading the CBTN study at your hospital will review the CBTN research study and consent to participate in research.

Per patient consent, CBTN shares all deidentified data with researchers and commercial entities for the purposes of research and under terms that ensure the nonexclusive use of such data.

Scientists use tissue samples to study how a tumor works by looking at its genes, molecules, and proteins. These specimens play a critical role in our emerging understanding of how these tumors work. For example, studying samples from breast cancer patients helped researchers create a test to identify breast cancer types, create new treatments, and customize treatments. Samples can be used to create cellular-based models for testing of new and more effective treatments for childhood brain tumors.

Cell lines are essentially a version of the tumor that can grow outside of the body in the laboratory. They can be continually grown in culture, so they are considered a renewable source of a biological sample. Cell lines cannot be generated from all tumors. While cell lines do not recapitulate all aspects of a tumor, they can be used in many ways by researchers, including testing against existing and experimental drugs to determine if the drugs might be effective against that cancer.

The CBTN empowers scientists, researchers, and clinicians to request and share CBTN’s resources (specimen samples or data) for use in their discovery projects.

More than 85,000 specimen samples have been provided to researchers across 442+ projects to date (and counting), in addition to data (200+ projects), all at no cost to them. If you are a researcher interested in utilizing CBTN resources, our Research Resources page includes an overview of what is available, additional FAQs, and a link to submit a scientific project.

No. Resources are provided to academic researchers at no cost to them to accelerate the pace of research. This is made possible through generous philanthropic support. If you are interested in supporting CBTN’s ongoing effort to collate and distribute data, please consider donating here.

Updates about each research study can be easily accessed on our Scientific Projects page. Each project listing includes the researchers involved, the types of CBTN resources utilized, and any subsequent outcomes. Research outcomes can also be accessed on our Publications page.

Due to patient privacy rules, we are unable to reidentify the patients from whom specimens and data were used in each study.

All CBTN source data is required to remain open and available as a publicly available resource in ways that adhere to NIH policies. However, while researchers are encouraged to share discovery data with the broader community, all such discoveries will be credited to the investigators involved in the project, not to CBTN. CBTN does ask that researchers note in the publication that its resources were utilized, but we never require investigators to include our team as authors on publications.

While CBTN uses an internal naming convention for models, we closely track where each model was developed and any previous names, sharing information transparently with researchers, to ensure there is no duplication. Likewise, if a cell line name has been defined by a submitting investigator, CBTN maintains this identifier as the primary identifier.

CAVATICA is an online platform used by researchers to access and analyze raw sequencing data in the cloud. Traditionally, data scientists downloaded datasets and stored and analyzed data on their computers. The downloading process can take time and requires significant computational resources, and in essence, duplicates data in siloed research. CAVATICA not only stores the data in a cloud environment but also enables the use of its computer power for analysis, avoiding the need for data download and use of powerful computers and enabling any data scientist across the globe to perform analysis.

The platform is equipped with ready-to-use, open-source bioinformatic tools that can be applied to a user’s own data, enabling rapid harmonization of data and its integration with the large-scale data of CBTN. The data stored on the portal can be either already analyzed or raw/source data, depending on what the scientist needs. CAVATICA currently houses the data from many contributing sites where data was generated, starting with CBTN members. In addition, the platform holds not only tumor data but also data on other pediatric diseases, allowing scientists to look for similarities between diseases that could inform therapy development. Finally, CAVATICA has been developed to interoperate with and connect to other NIH platforms and data sources, providing a rapid way for researchers to connect to and bring data together from multiple sources.

CBTN’s master agreement with sites and researchers mandates that data or materials collected on the CBTN study cannot be sold and must remain an open resource for global use. Within that framework, CBTN is open to partnering with commercial entities to advance development and understanding of pediatric brain tumors. Areas of partnership include but are not limited to, the following:

• Data Science
  • Target/biomarker identification
  • Tumor subtyping; cancer mechanism studies
  • Predictive modeling
  • Blood-brain barrier research
  • Clinical outcome analysis
  • Algorithm development
  • Artificial intelligence
• Comparison Diagnostic Assay Development
  • Biospecimen available to support development
• Clinical Trial Support
  • Compassionate use data collection
• Drug Development
  • Preclinical models paired with data
  • Support drug indications
• Data Generation & Collaborative Projects
  • Molecular characterization is often resource-dependent
  • CBTN is open to collaborative projects to generate additional molecular characterization data

Commercial entities are required to cover the personnel costs and operational expenses associated with collaborative research initiatives, either through philanthropy, grant funds, or other mechanisms.

As with any investigation using CBTN resources, while the source data is required to remain open, any discoveries made by the effort are owned by the entity making those discoveries.

Health Care Information Exchanges are emerging systems that collate clinical data from electronic health records as they are submitted by participating institutions. Currently, not all hospitals participate, and the ones that do participate at various levels. CBTN is working with HIE to identify whether clinical data can be collected to support a path towards scalability and deepened efficiency.

Great! We can’t wait to connect with you. Contact CBTN at research@cbtn.org, and we’ll connect you to the right person on our team.

CBTN is strives to push the field forward through piloting new and innovative approaches to data aggregation, sharing, and analysis, all in service of improving patient outcomes. A few current such pilots include:

• Clinical decision support: In partnership with the Pacific Pediatric Neuro-oncology Consortium’s PNOC027 trial and building on efforts and lessons from PNOC003 and PNOC008, CBTN’s Precision Medicine Working Group, in partnership with researchers at CBTN’s Operations Center at CHOP, is analyzing deep genomic and molecular profiling patient data against the CBTN’s harmonized cohort data set to help determine how other patients with similar genetic profiles performed in treatment. They are then tasked with generating a report with potential therapeutic targets and information about relevant clinical trials to support clinical decision-making. Pediatric brain tumor patients have never had the benefit of a comparative pediatric data set and have instead been compared to adults. This new approach represents an exciting opportunity to use historical data to create personalized care plans for children based on data from other kids.
• Clinical trial development: CBTN is excited to partner with any clinical trialist or consortium interested in advancing treatments for pediatric brain tumors through data-informed trial development. Efforts are currently driven through the work of the CBTN/PNOC Working Groups.
• Clinical data abstraction/connections to electronic health record systems: CBTN envisions a treatment environment where clinical data connects directly to the research ecosystem to support longitudinal patient care. CBTN and the Center for Data Driven Discovery in Biomedicine at CHOP are currently piloting four projects to support scalable, streamlined clinical data abstraction directly with hospital systems. These include partnerships with Amazon Web Services and the NIH’s National Cancer Institute Childhood Cancer Data Initiative.
• Disease-specific registries: CBTN has ongoing partnerships with disease-specific initiatives, foundations, and registries, including OligoNation, the Chordoma Foundation, ETMR One, and the DIPG Registry. More recently, CBTN has partnered with the National Cancer Institute, Children’s Oncology Group, PNOC, and Children’s Hospital of Los Angeles to pilot a Rare Tumor Protocol. This effort will lay the groundwork for deepened disease-specific data collection to support the advancement of therapeutics research and clinical trial development. The first pilot will be in AT/RT.

If you have questions related to CBTN data or requesting CBTN research resources, please visit the Research Resources FAQ section on the CBTN website. You may also download the CBTN acronym dictionary of key research acronyms and terms.