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Monthly CBTTC Advisory Council Report-Out
September 2020

To our CBTTC partners,

Exciting times! We look forward to all of you meeting with Brian Rood our new co-executive chair on September 30th at 5pm to learn more about his vision for CBTN. On this call you will have the opportunity to hear from Brian and, in addition, provide your recommendations and ask questions regarding the FY 21 goals, the FY20 goal reports, FY21 budget plan, FY21 budget report, FY 21 communications plan, and FY 20 communication report. The reports and plans will be sent out prior to the meeting for your review. Please feel free to submit questions prior to the call. 

The new brand is coming! Please feel free to provide feedback on the brand strategy that was circulated. In the Communications Section of this report, you will find a link to our roll-out campaign, for your review and comment. We are making great strides on the website and for those that want a walk through of the new website with us and provide feedback we will be having a call on September 3rd at 5pm, please let us know if you would like to be included. We will send out the beta site to you all after the call where you can provide feedback right on the site.

As we start to make the transition from the Advisory Council to the new CBTN membership categories we will be reaching out to you to discuss how you would like to participate. We will also begin scheduling the new quarterly calls for the Executive Committee, financial subcommittee, and advocacy subcommittee for 1 month following the end of each fiscal quarter, with scheduled calls at end of October and January, breakout meetings for the EC and subcommittees during the May Investigator meeting, and an end-of-year call in late June. We are thrilled to begin planning the recognition program as well and will have that as a topic for our fall meeting.

Thank you for your ongoing feedback through private messages, during our new CBTN Community Information Session, during committee calls, and in listening sessions. We are always open to feedback to improve, and hope you find this monthly report-out helpful.

As a reminder we encourage you to share the newsletter subscription link with your personal networks so we can continue to inform the community of the work being done by investigators and patient advocates on behalf of children with brain tumors.

Please find the report for this past month below. 

-CBTTC Executive Committee
CBTTC Research Core Updates:

CBTTC Site Status:

Goal: is to increase membership to create the largest pediatric brain tumor resource, with representation across all histologies.

CBTTC now has 24 member sites with the US, Australia, Europe, and China represented globally! 6 of those 24 sites are all in varying stages of on-boarding. On-boarding includes execution of our master agreement, regulatory approval, training, and the collection of certifications. Our policy is to officially announce a new site once they have executed the agreement and have approved regulatory documents. 

As noted previously, if you identify a hospital that would like to join CBTTC, please contact us. We can reach out, provide a background of the consortium and then steps to apply. 

Total Number of Sites (internationally): 24* 
listing of all sites can be found at:
* 18 sites have fully completed regulatory review and site agreement process

New Site Applications in Progress (not yet submitted): 0
New Site Applications: 1

  • Doernbecher Children's Hospital
    • On-boarding Status: Application Review by EB in Progress
New Sites Recently Approved and Onboarding: 6
  • NYU/Langone Children’s Hospital - Core Member
    • On-boarding Status: Agreement executed, regulatory review in progress
  •  Arnold Palmer Children’s Hospital - Data Satellite
    • On-boarding Status: Agreement executed, regulatory review in progress
  • University of Michigan/CS Mott Children’s Hospital - Data Satellite
    • On-boarding Status: Agreement executed, regulatory review in progress
  • Sydney Children’s - Specimen Satellite
    • On-boarding Status: Welcome email was sent and on-boarding begins!
  • University Children's Zürich: Specimen Satellite
    • On-boarding Status: Welcome email was sent and on-boarding begins!
  • St. Louis Children’s - Data Satellite
    • On-boarding Status: Welcome email was sent and on-boarding begins!

If you have interest in assisting one of our already existing sites or those now on-boarding, please let us know! Many sites have informed us of what they expect to be upcoming shortfalls for research funding due to COVID-19. We thank all that have donated to CBTTC COVID relief needs to ensure all of the CBTTC sites continue to move forward full steam this year.

CBTTC Enrollment:
Goal: is to increase membership to create the largest pediatric brain tumor resource, with representation across all histologies.

CBTTC consents children and biological parents for participation. For patients, medical information about their brain tumor is collected, as well as biospecimens. For biological parents, only a biospecimen is collected; normally saliva. This is a sample collection that can be done at home and mailed in. Having the family participate is extremely important for research efforts.

  • Total Number of Sites: 24*
    • *18 have been fully on-boarded
  • Overall Total Enrolled
    • All-Time: 4,729
    • FY21: 81
  • Total Children Enrolled
    • All-Time: 3,585
    • FY21: 24
  • Total Mothers Enrolled
    • All-Time: 644
    • FY21: 24
  • Total Fathers Enrolled
    • All-Time: 500
    • FY21: 18
CBTTC Research Resources:

The CBTTC collects, manages, and distributes longitudinal clinical data, biospecimens, pre-clinical models, genomic, proteomic, and other data generated from the collected biospecimens. These resources are available for any investigator to request. 

The following is a breakdown of some of the major histological groups (however, more is available). As you all talk with researchers, please let them know these resources are available for their research. All of these have longitudinal clinical data, and as funds become available we generate genomic data, proteomic data, and pre-clinical models. If you have an interest in supporting data generation or pre-clinical model development and drug testing for a specific histology, please let us know. Generation of data and pre-clinical models are critical as they are non-consumptive (unlike biospecimens) and can be used by anyone as many times as needed.

You can query the available data here

Histology    |   Number of Subjects Enrolled with this Diagnosis
  • ATRT - 99
  • DMG - 135
  • Medulloblastoma - 441
  • Low Grade Glioma - 996
  • High Grade Glioma - 346
  • Ependymoma - 317
  • Craniopharyngioma - 128
  • Dysembryoplastic Neuroeithelial Tumor (DNET) - 76
  • Ganglioglioma - 185

Financial Updates:

NIH Grant Submission
Pediatric Brain Tumor SPORE: Last chance if interested to provide a letter of philanthropic support for any of the areas of the SPORE and have those funds directed including CBTTC, the research projects listed below or the Development Research Program and Career Enhancement Program. As discussed the NIH provides 1.4 million dollars but much more is needed. These are great investment opportunities as your funds will be in addition to NIH funding and show the NIH our commitment to the Pediatric Brain Tumor SPORE success.  

  • This would provide funding for four preclinical research projects, CBTTC operations, and PNOC operations if awarded in late 2021 or early 2022. 
  • 123 letters of support (LOS) received to date.
  • Four translational research projects were selected to participate:
Mechanism, Function, and Expression of ERVs Across H3K27me3 Depleted Brain Tumors
  • Co-lead: Stephen C. Mack, PhD; Texas Children’s Hospital
  • Co-lead: Donald W. Parsons, MD, PhD; Texas Children’s Hospital
A multipronged approach for targeting tumor cell energy metabolism and activation of the integrative stress response pathway
  • Co-lead: Sabine Muller MD, PhD; UCSF Benioff Children’s Hospital, University Children’s Hospital of Zurich
  • Co-lead: Javad Nazarian, PhD; University Children’s Hospital of Zurich
  • Co-lead: Eric Raabe, MD, PhD; Johns Hopkins School of Medicine.
Proteogenomically determined neoantigen-specific T cell therapy for infant embryonal tumors
  • Co-lead: Brian R. Rood, MD; Children's National Health System
  • Co-lead: Eugene I. Hwang, MD; Children's National Health System
  • Co-lead: Catherine M. Bollard, MBChB, MD; Children's National Health System
Optimization of CAR T cell therapy for DIPG
  • Project lead: Nick Vitanza, MD; Seattle Children’s Hospital
  • Co-lead: Jessica Foster, MD; Children’s Hospital of Philadelphia
  • Genomic lead: Mike Berens, PhD; Translational Genomics Research Institute 
  • CSF lead: Jeffrey Iliff, PhD; UW Medicine
  • Clinical oversight: Julie Park, MD; Seattle Children’s Hospital
  • Scientific oversight: Rimas Orentas, PhD; Seattle Children’s Hospital
CBTTC Research

Key Highlights: CBTTC has collaborated on 160 research projects with over 100 investigators across 9 countries. 56 are CBTTC investigators and 66 are non-CBTTC investigators. This includes three collaborations with the PNOC disease working groups for the pre-clinical work required for the development of a new clinical trial.

Biospecimen Requests:
In an attempt to increase the utilization of biospecimens, CBTTC has recruited additional scientific experts to review proposals and formed two committees. You can find information on these projects here

All-time total number of requests: 75

  • 63 total requests approved
    • 46 sample requests approved
    • 17 cell line requests approved
  • 4 requests changed to data access requests (data was already generated)
  • 4 requests were withdrawn from consideration by the investigator
  • 4 requests are with the investigator for revisions or under further review

Data Requests for raw genomic data: Our goal is to increase the number of requests and utilization of data. Our goal was to double request from last year 14, to 28 and as of July we have exceeded this goal with 55. Additionally, “processed data” is available anytime without request has been utilized thousands of times. You can find more information on the data projects here

  • 110 total requests approved

Map of current CBTTC Data Users

OPEN PBTA landscape analysis phase one is complete and the goal is to finalize a paper in 2020. All data are available and a request was sent out for anyone to contribute to the manuscript and analysis.

Research Opportunities:
We are looking for opportunities to empower and accelerate brain tumor research. Please let us know if there any other researchers you’ve connected with that you would like us to reach out to?

Research Publications:

CBTTC has several key publications occurring now, with new submission to 12 different scientific journals.

Total Accepted: 17
Accepted this month: 2

  1. Aubin, Rachael, Emma C. Troisi, Adam N. Alghalith, Maclean P. Nasrallah, Mariarita Santi, and Pablo G. Camara. 2020. “Cell Ecosystem and Signaling Pathways of Primary and Metastatic Pediatric Posterior Fossa Ependymoma.”
  2. Petralia, Francesca, Nicole Tignor, Boris Reva, Pichai Raman, Shrabanti Chowdhury, Dmitry Rykunov, Azra Krek, et al. 2020. “Abstract 445: Integrated Proteogenomic Characterization across Seven Histological Types of Pediatric Brain Tumors.” Cancer Research 80 (16 Supplement): 445–445.
Total Currently Submitted: 4
  1. Submitted an abstract entitled “Discovering genetic interactions in the CBTTC dataset” for BioSB2020, a Dutch conference with focus on bioinformatics and systems biology. Abstract selected for a poster presentation. Conference originally scheduled for 21-22 April 2020, but now postponed to 27-28 October 2020. More information:"
  2. Submitted an abstract titled “Reconstructing the Gene Regulatory Landscape of Pediatric BrainTumors” to the 2020 ISMB meeting (Regulatory and Systems Genomics track)
  3. Payal Jain, Lea F. Surrey, Joshua Straka, Pierre Russo, Richard Womer, Marilyn M. Li, Phillip B. Storm, Angela Waanders, Michael D. Hogarty, Adam Resnick, Jennifer Picarsic. ‘Novel BRAF fusions in pediatric histiocytic neoplasms define distinct therapeutic responsiveness to RAF paradox breakers’. (Submitted to Cancer Research, May 2020)
  4. The Immunogenomic Landscape of Primary Pediatric Solid Tumours- (Submitted to Cancer Cell)
Pending Submission: 5
  1. High-grade glioma cell line models for pediatric cancer. Mateusz Koptyra, Komal S. Rathi, Valerie Baubet, Jo Lynne Rokita
  2. OPEN PBTA Manuscript is on track, goal pre-print Sept 2020
  3. CBTTC Resource Paper being reviewed submission to J Neuro-Oncology and Neurosurgery goal Sept 2020
  4. A Transcriptome-based Classifier to Determine Molecular Subtypes in Medulloblastoma.
  5. Ammar S. Naqvi, Brian Ennis, Krutika S. Gaonkar, Michael Koldobskiy, Yuankun Zhu, Miguel A. Brown, Bo Zhang, Phillip B. Storm, Adam C. Resnick, Jo Lynne Rokita (2020). “Molecular mechanisms and functional impact of aberrant splicing in diffuse intrinsic pontine gliomas”.

Research Presentations at Conferences:

4 Abstracts accepted
  1. Ammar S. Naqvi, Brian Ennis, Krutika S. Gaonkar, Michael Koldobskiy, Yuankun Zhu, Miguel A. Brown, Bo Zhang, Phillip B. Storm, Adam C. Resnick, Jo Lynne Rokita (2020). “Molecular mechanisms and functional impact of aberrant splicing in diffuse intrinsic pontine gliomas”. ABSTRACT #446. RNA 2020 on-line meeting.
  2. Payal Jain presented to the AACR virtual meeting (June 22-24). Her abstract was #6746: Novel BRAF gene fusions in pediatric histiocytic neoplasms respond differently to RAF targeted therapies based on dimerization profiles.
  3. Lauren M. Sanders, A. Geoffrey Lyle, Holly C. Beale, Ellen Towle Kephart, Katrina Learned, Jacob Pfeil, Jennifer Peralez, Norman Lacayo, Arun Rangaswami, Sheri L. Spunt, Isabel Bjork, David Haussler, Sofie R. Salama, Olena M. Vaske, "Comparative gene expression analysis for identification and prioritization of therapeutic targets in a cohort of childhood cancers." Poster, AACR Advances in Pediatric Cancer Research Conference, Montreal, September 2019.
  4. Olena M. Vaske. Data Federation Approaches in Treehouse Childhood Cancer Initiative. Invited Talk. Childhood Cancer Data Initiative Symposium, Washington, DC, July 2019.
1 Abstract Pending
  1. Submitting an abstract to the AACR conference "Tumor heterogeneity: from single cells to clinical impact" to be held in Philadelphia on September 10-13. Preparing a manuscript entitled "Cell Ecosystem and Signaling Pathways of Posterior Fossa Childhood Ependymoma Revealed by Single-Cell Transcriptomic Profiling", - plan to submit for publication this fall.
Cell line Data Generation Efforts
Currently the CBTTC has 70+ cell lines that are available for pre-clinical testing. To enhance these pre-clinical models researchers perform genomic testing WGS and RNA Seq on the cell lines in addition to the tumor samples. Currently, this genomic characterization has begun for specific brain tumor types but is funding-dependent to continue. Please reach out to Jena Lilly ( if you would like to sponsor.

CBTTC/PNOC Working Groups:
There are seven CBTTC/PNOC working groups - Medulloblastoma, DMG, ATRT, LGG, Ependymoma, Craniopharyngioma and Imaging. Each working group has their own goals and they convene once every month and collaborate on preclinical and clinical research. The groups are facilitated with CBTTC by allowing for sharing of resources, clinical data, biospecimens, preclinical models and genomic and proteomic data that can be used to develop new data-driven clinical trials. If you would like to participate or learn more please contact Kamnaa Arya (ARYAK@EMAIL.CHOP.EDU

Medulloblastoma Leads: 

  • Robert Wechsler-Reya, PhD; Sanford Burnham Prebys
  • Tabitha Cooney, MD; UCSF Benioff Children’s Hospital

Craniopharyngioma  Leads: 

  • Cassie Kline, MD; UCSF Brain Tumor Center
  • Fatema Malbari, MD; Texas Children’s Hospital

LGG leads: 

  • Angela Waanders, MD, MPH; Ann & Robert H. Lurie Children’s Hospital of Chicago
  • Daphne Adele Haas-Kogan, MD; Dana-Farber Cancer Institute
  • Pratiti Bandopadhayay, MBBS, PhD; Dana-Farber Cancer Institute
  • Joanna Phillips, MD, PhD; UCSF Helen Diller Family Comprehensive Cancer Center

ATRT leads: 

  • Ashley Margol, MD, MS; Children’s Hospital Los Angeles
  • Annie Huang, MD, PhD, FRCP(C); The Hospital for Sick Kids
  • Eric Raabe, MD, PhD; Johns Hopkins School of Medicine.

Ependymoma leads: 

  • Eugene Hwang, MD; Children’s National Health System
  • Mariella Filbin, MD, PhD; Dana-Farber Cancer Institute
  • Steven Mack, PhD; Texas Children's Hospital
  • Derek Hanson, MD; Hackensack Meridian Health

DMG leads:

  • Sabine Mueller, MD, PhD; UCSF Benioff Children’s Hospital
  • Javad Nazarian, PhD; University Children’s Hospital of Zurich]
Imaging Working Group leads:
  • Josh Rubin, MD, PhD; Washington University School of Medicine, St. Louis
  • Javier Villaneuva-Meyer, MD; University of California, San Francisco
  • S. Ali Nabavizadeh, MD; Hospital of the University of Pennsylvania 
CBTTC Communications:


Do you need any resources, collateral materials, or participation from the CBTTC for any scheduled event or any other activity.

September is Childhood Cancer Awareness Month!

We know you share our eager anticipation for honoring Childhood Cancer Awareness Month (CCAM), and we hope to support your outreach, fundraising, and awareness efforts as much as possible. As with our previous awareness month campaigns, a big component of our messaging plans for the coming month will center around spotlighting the amazing efforts of your respective organizations in supporting and advancing childhood brain cancer research. We’ve already heard from a number of you regarding events and campaigns planned over the coming weeks, but as we work to firm up our messaging calendar for September, please send along any campaign hashtags, talking points, event details, or other information that we can share out further with our followers on social media, our newsletter subscribers, and our research partners!

CBTN Brand Launch
In September, we’re also excited to carry out our official public launch of the new CBTN brand and associated materials! In this Google Document, you’ll find our general launch campaign goals and tactics (including several proposed opportunities to participate in this effort). Those of you who have the ability, we would greatly appreciate your review and feedback of these goals, as well as any suggestions for how we can partner with you on getting the word out to the broader patient and research community about the expanded vision and mission of the CBTN and our deepening partnership with the Pacific Pediatric Neuro-Oncology Consortium (PNOC) in clinical trial support/development and translational research efforts.
On social/web media, we plan to build our outreach campaign around the launch of our new website, The website will be loaded with an array of new features, new information, and new ways for visitors to engage with us. We are hard at work to finalize and import comprehensive information for our over 100 research projects, as well as working groups, external partnerships, funding opportunities and more. Our internal project managers and team members are working to complete the new site, and hope to nail down the date in September when we’ll be ready to unveil the site to the public. In the 2 weeks leading up to that date, we’ll be counting down to the launch with teasers of our new site, logo, brand elements, expanded mission, and other digital content.
We are eager to share it with you and will be keen to get your first impressions and valuable feedback!

Brand Development Updates:
Please click the link below to view the CBTN brand style guide. This style guide defines the CBTN’s brand standards and will be used as a reference in creating all CBTN branded handouts, infographics, videos and other collateral materials. This is intended for CBTN internal use as well as working to create materials with external vendors, agencies and member institutions. 

View the style guide

We’ve also begun creating a library of CBTN print and digital resources - more of these assets including one-pagers and infographics will be shared with you over the coming weeks and months and will form a toolkit for foundations and other groups to use to share about the CBTN’s research. 

Below is a poster (18x24”) that we created as an inspiration for our operations team - we would like to share a version of this poster with each of you. Please email your response to the questions below to Bobby Moulder ( or We really appreciate your feedback and want to make sure these resources are representing the needs of the various communities we serve in the CBTN, especially our patient families. 

View the poster

Questions about the poster (Please respond via email)

  1. In this initial version, we have listed the first names of each of the children featured. If you would prefer to not have a name listed by the photo of each child, please let us know. 
  2. We have selected photos of children from the Advisory Council and other selected patients - if you’d prefer to have a poster including photos of researchers or clinical team members, or other patients, please contact us. We can make a version with updated images. 
  3. If you would like more than 1 complimentary poster sent to you, please contact us with your interest. Pricing for each poster will depend on the quantity ordered/vendor selected. We’re also happy to share the digital files so that you’re able to print at your own convenience. 
  4. If you would like to receive a printed poster, please share the best mailing address to send to you! 

If you have suggestions or feedback for website features to include, perhaps from other websites you’ve especially liked, please contact us at

Social Media Metrics - August 2020:


  • Audience Reach: 2,535 users (2,831 impressions)
  • Users Directly Engaged: 128
  • Engagement Rate: 5.04% (1-3%: industry avg.)
  • Followers Gained: 6
Current Followers: 580


  • Audience Reach: 5,784 users
  • Users Directly Engaged: 162
  • Engagement Rate: 2.8% (0.33% industry avg.)
  • Followers Gained: 5
Current Followers: 609

CBTTC Website Traffic - August 2020:

  • 659 Unique Users (84.9% New Users)
  • 824 Sessions
  • 1,725 Pageviews
Q&A Spotlight

Question: How do researchers get access to specimens?

Answer: Any research from around the world can request CBTN specimens for their research. The first step is completing the specimen project request form. This form is then QC’d  by the CBTN Operational Team to see if there is a collaborative project already in place or data already available and any initial questions or errors are corrected. The project proposal is then assigned to a primary scientific reviewer that has expertise in the area in which the project is proposing to do research. The primary reviewer will provide a summary and either questions back to the investigator or provide provisional approval for the proposal to go to the full scientific committee. The scientific committee then reviews and either approves or provides additional questions. If approved the researcher is notified and the specimens are prepared for shipment. The caveat is that anything learned and raw data created must be shared back with the community.
Past Reports
Did you miss one of our last CBTTC Advisory Council Reports? You can access them here!
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Copyright © 2020 Children's Hospital of Philadelphia/Center for Data-Driven Discovery in Biomedicine, All rights reserved.

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