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Monthly CBTTC Advisory Council Report-Out
May 2020

To our patient partners,

We hope this finds you all safe and healthy. Thank you for your feedback on this report. Feel free to continue sending comments so we can improve these monthly reports and provide information most valuable to you. We want to be mindful of your busy schedules, and have chosen to include the latest and most important updates below. Later this month, CBTTC will be releasing the next edition of our quarterly newsletter, where we’ll share even more updates. Let us know if there are any specific areas you’d like us to highlight further, and we’ll be sure to do so later this month. And 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.

We are continuing to meet with each of you to discuss CBTTC and the Advisory Council, expecting to  complete by the end of May. Recommendations and next steps for the Advisory Council structure and purpose will follow. 

As you know, CBTTC remains “open” despite a lot of research being halted or slowed during COVID-19. Our data resources including genomic and clinical data are still available to be requested. Additionally, we are approaching a milestone of 100 CBTTC data projects. Our target is to hit this milestone during the month of May, Brain Tumor Awareness Month. You can assist us by sharing our social media posts and calling out the ability to access our data resources, free of charge without embargo. 

During these trying times please know how important BTAM is, and that #wewontstop until every child has a cure.

Please find the report for May below. 

-CBTTC Executive Committee
Operations Report:

CBTTC Site Status:

Hospitals can apply to join the CBTTC at any time. There are 2 levels of membership, Primary Member and Satellite member. The goal is to have as many hospitals around the world join but due to funding restrictions we can not accept new primary member sites unless funding is identified for the hospital or the hospital self-funds their participation. Please contact us if you identify a hospital that would like to join the CBTTC.

Total Number of Sites: 18

a listing of all sites can be found at:

  • New Site Applications: 0

  • New Sites Recently Approved and On-boarding: 3

    • NYU/Langone Children’s Hospital - Core Member

    • Arnold Palmer Children’s Hospital - Data Satellite

    • University of Michigan/CS Mott Children’s Hospital - Data Satellite

  • If you have interest in assisting one of the above sites with finding local funding to support their efforts, please let  us know. Many sites have informed us of what they expect to be upcoming funding shortfalls for research due to COVID-19.

CBTTC Enrollment:

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.

  • Total Enrolled: 4,639

    • Children: 3,537 

    • Mothers: 620

    • Fathers: 482 

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 - 97
  • DMG - 133
  • Medulloblastoma - 431
  • Low Grade Glioma - 976
  • High Grade Glioma - 372
  • Ependymoma - 313
  • Craniopharyngioma - 128
  • Dysembryoplastic Neuroeithelial Tumor (DNET)
  • Ganglioglioma - 171


Fiscal year July 1, 2020 to June 30, 2021

  • CBTTC Leadership is reviewing mitigation strategies for what will likely be one of the most financially challenging fiscal years for the CBTTC to date as nearly all fundraising efforts and philanthropy has been impacted.  As hospital systems come “back on line” and we regroup around our research infrastructure’s re-engagement, we will share updates with the Advisory Council.

Current CBTTC Grant Submissions:

Pediatric Brain Tumor SPORE - on track for submission in Sept 2020. Presentation scheduled late May to the National Cancer Institute (NCI). 123 letters of support (LOS) received to date. This would provide funding for four preclinical research projects, CBTN operations, and PNOC operations if awarded in late 2021 or early 2022. However, the grant does not provide funding for the clinical trials that will result from these efforts. If you are interested in support a clinical trial for the SPORE grant please let us know.

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, M.D., Ph.D.,  Johns Hopkins School of Medicine.
Proteogenomically determined neoantigen-specific T cell therapy for infant embryonal tumors
  • Co Lead: Brian R. Rood, M.D.Children's National Health System
  • Co Lead:  Eugene I. Hwang M.D. Children's National Health System
  • Co Lead: Catherine M. Bollard M.B.Ch.B., M.D 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. 53 are CBTTC investigators and 61 are non-CBTTC investigators. This includes two collaborations (one in review)  with the PNOC disease working groups for the pre-clinical work required for the development of a new clinical trial.

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

Disease Working Groups:
There are 6 initial CBTTC/PNOC working groups: Medulloblastoma, DMG, ATRT, LGG, Ependymoma and Craniopharygimona formed with the goal of launching clinical trials that build on the CBTTC’s research and data resources. This partnership allows the CBTTC resources, clinical data, biospecimens, preclinical models and genomic and proteomic data to be used to develop new data-driven  clinical trials.  We are identifying opportunities to provide progress updates , stay tuned. For any questions regarding the Working Groups 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, M.D., Ph.D.,  Johns Hopkins School of Medicine.

Ependymoma leads: 

  • Eugene Hwang, MD; Children’s National Health System
  • Mariella Filbin, MD, PhD; Dana-Farber Cancer Institute

DMG leads:

  • Sabine Mueller, MD, PhD; UCSF Benioff Children’s Hospital
  • Javad Nazarian PhD University Children’s Hospital of Zurich

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:

From April 1 to May 1, we have received 4 biospecimen requests. 

All-time total number of requests: 71

  • 58 total requests approved
    • 44 sample requests approved
    • 14 cell line requests approved
  • 4 requests changed to data access requests (data was already generated)
  • 3 requests were withdrawn from consideration by the investigator
  • 6 requests are under review

Data Requests:
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 April we have exceeded this goal with 38. Additionally, “processed data” is available anytime without request has been utilized thousands of times. You can find more information on the data projects here

From April 1 to May 1, we have received 6 data requests.
All-time total number of requests: 99

  • 92 total requests approved
  • 6 requests were withdrawn from consideration
  • 1 request is under review

Data Types                                FY19   |   FY20 Goal     |   FY20 Actual
Raw Genomic Data Users:         14     |         28            |           38
Open Access Data Users:        1,080  |   1,620 (50%)   |       1,261

Map of current CBTTC Data Users

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 the journals Cell and Cancer Cell.

Accepted: 9

  1. Connors, M., W. Paden, P. B. Storm, A. J. Waanders, and S. S. Lang. 2019. 'Low-grade astrocytoma in the setting of a developmental venous anomaly', Childs Nerv Syst.
  2. Dang, Mai T, Michael Gonzalez, Krutika S Gaonkar, Komal S Rathi, Patricia Young, Sherjeel Arif, Li Zhai, Md Zahidul Alam, Samir Devalaraja, and Tsun Ki To. 2020. 'Single-cell transcriptomic profile reveals macrophage heterogeneity in medulloblastoma and their treatment-dependent recruitment', BioRxiv.
  3. Gaonkar, Krutika S., Komal S. Rathi, Payal Jain, Yuankun Zhu, Miguel A. Brown, Bo Zhang, Pichai Raman, Phillip B. Storm, John M. Maris, Adam C. Resnick, Jaclyn N. Taroni, and Jo Lynne Rokita. 2019. 'annoFuse: an R Package to annotate and prioritize putative oncogenic RNA fusions', BioRxiv: 839738
  4. Hoffman, M., A. H. Gillmor, D. J. Kunz, M. J. Johnston, A. Nikolic, K. Narta, M. Zarrei, J. King, K. Ellestad, N. H. Dang, F. M. G. Cavalli, M. M. Kushida, F. J. Coutinho, Y. Zhu, B. Luu, Y. Ma, A. J. Mungall, R. Moore, M. A. Marra, M. D. Taylor, T. J. Pugh, P. B. Dirks, D. Strother, L. Lafay-Cousin, A. C. Resnick, S. Scherer, D. L. Senger, B. D. Simons, J. A. Chan, A. S. Morrissy, and M. Gallo. 2019. 'Intratumoral Genetic and Functional Heterogeneity in Pediatric Glioblastoma', Cancer Res, 79: 2111-23.
  5. Ijaz, H., M. Koptyra, K. S. Gaonkar, J. L. Rokita, V. P. Baubet, L. Tauhid, Y. Zhu, M. Brown, G. Lopez, B. Zhang, S. J. Diskin, Z. Vaksman, J. L. Mason, E. Appert, J. Lilly, R. Lulla, T. De Raedt, A. P. Heath, A. Felmeister, P. Raman, J. Nazarian, M. R. Santi, P. B. Storm, A. Resnick, A. J. Waanders, and K. A. Cole. 2020. ‘Pediatric High Grade Glioma Resources From the Children’s Brain Tumor Tissue Consortium (CBTTC) and Pediatric Brain Tumor Atlas (PBTA)’ , Neuro Oncol, 22: 163-65.
  6. Ijaz, Heba, Mateusz Koptyra, Krutika S. Gaonkar, Jo Lynne Rokita, Valerie P. Baubet, Lamiya Tauhid, Yankun Zhu, Miguel Brown, Gonzalo Lopez, Bo Zhang, Sharon J. Diskin, Zalman Vaksman, Jennifer L. Mason, Elizabeth Appert, Jena Lilly, Rishi Lulla, Thomas De Raedt, Allison P. Heath, Alex Felmeister, Pichai Raman, Javad Nazarian, Maria Rita Santi, Phillip B. Storm, Adam Resnick, Angela J. Waanders, and Kristina A. Cole. 2019. 'Pediatric High Grade Glioma Resources From the Children’s Brain Tumor Tissue Consortium (CBTTC) and Pediatric Brain Tumor Atlas (PBTA)', BioRxiv: 656587.
  7. Rivero-Hinojosa S, Kinney N, Garner HR, Rood BR. ‘Germline microsatellite genotypes differentiate children with medulloblastoma’, Neuro-Oncology. 2020 Jan 11;22(1):152-62.
  8. Steidle, E. A., V. A. Morrissette, K. Fujimaki, L. Chong, A. C. Resnick, A. P. Capaldi, and R. J. Rolfes. 2020. 'The InsP7 phosphatase Siw14 regulates inositol pyrophosphate levels to control localization of the general stress response transcription factor Msn2', J Biol Chem, 295: 2043-56.
  9. Surrey, L. F., P. Jain, B. Zhang, J. Straka, X. Zhao, B. N. Harding, A. C. Resnick, P. B. Storm, A. M. Buccoliero, L. Genitori, M. M. Li, A. J. Waanders, and M. Santi. 2019. 'Genomic Analysis of Dysembryoplastic Neuroepithelial Tumor Spectrum Reveals a Diversity of Molecular Alterations Dysregulating the MAPK and PI3K/mTOR Pathways', J Neuropathol Exp Neurol, 78: 1100-11.
Submitted: 4
  1. Molecular mechanisms and functional and therapeutic impact of aberrant splicing in diffuse intrinsic pontine glioma (to be expanded to all high-grade gliomas) (in progress) 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
  2. Francesca Petralia, Nicole Tignor, Boris Reva, Mateusz Koptyra, Shrabanti Chowdhury, Dmitry Rykunov, Azra Krek, Weiping Ma, Jiayi Ji, Xiaoyu Song,  Pichai Raman, Yuankun Zhu, Jeffrey R. Whiteaker, Antonio Colaprico, Anna Calinawan, Selim Kalayci, Zeynep H. Gümüş, Yiran Guo, Miguel A. Brown, Richard G. Ivey, Gonzalo Lopez, Seungyeul Yoo, Lizabeth Katsnelson, Ying Wang,  Jacob J. Kennedy, Uliana J. Voytovich, Lei Zhao, Felipe da Veiga Leprevost, Hui-Yin Chang, Krutika Satish Gaonkar, Brian M. Ennis, Bo Zhang, Valerie Baubet, Lamiya Tauhib, Jena V. Lilly, Jennifer L. Mason, Bailey Farrow, Nathan Young, John Spytz, Sanjukta Thakurta, Javad Nazarian, Nithin D. Addapa, James N. Palmer,  Robert M. Lober, Samuel Rivero-Hinojosa, Liang-bo Wang, Joshua Wang, Matilda Broberg, Xi Steven Chen, Jun Zhu, Eric E. Schadt, Mehdi Mesri, Emily Boja, Tara Hiltke, Henry Rodriguez, Bing Zhang, Li Ding, Antonio Iavarone, Maciej Wiznerowicz, Allison P. Heath, Jo Lynne Rokita,  Alexey I. Nesvizhskii, David Fenyo, Steven Gygi, Amanda G. Paulovich, Adam C Resnick, Phillip B. Storm, Brian R. Rood, Pei Wang, Children’s Brain Tumor Tissue Consortium and Clinical Proteomic Tumor Analysis Consortium. Integrated Proteogenomic Characterization across Major Histological types of Pediatric Brain Cancer. (Submitted to Cell, December 2019).
  3. The Immunogenomic Landscape of Primary Pediatric Solid Tumours- (Submitted to Cancer Cell)
  4. 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)

Pending Submission: 4

  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.

Research Presentations at Conferences:

Payal Jain will be presenting to the AACR meeting (June 22-24). Her abstract is #6746: Novel BRAF gene fusions in pediatric histiocytic neoplasms respond differentially to RAF targeted therapies based on dimerization profiles.

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.

Launch of Cavatica Australia
CBTTC has partnered with The Australian Research Data Commons (ARDC), Bioplatforms Australia, the Australian BioCommons, and Children’s Cancer Institute, and Seven Bridges to expand CAVATICA to utilize Amazon Web Services in Sydney and piloting new capabilities to support Australian initiatives such as Zero Childhood Cancer. The expansion has already enhanced international collaboration by providing easier and faster approaches to sharing data and analyzing rare childhood cancer data.
More information on this collaboration will be included in the May 2020 Quarterly Newsletter
CBTTC Communications:
Advisory Council Listening Tours:
Mike Prados has been leading discussions with each patient family/foundation that are either currently on the Advisory Council or once a member to further explore.  We will collate these and upcoming feedback and share with the group. These have been extremely helpful and thank you again for your time and recommendations.

  • Total complete: 8
  • Total scheduled: 6
  • Total to be scheduled: 3


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

Brain Tumor Awareness Month - Campaign Summary:

The spring of 2020 is a unique and challenging period for communities around the globe. In response to the global COVID-19 pandemic, virtually all facets of our society have been impacted. However, childhood brain cancer - a clear and present danger for thousands of children and their families around the world - has not slowed down due to this pandemic. Fortunately, the investigators, clinicians, patient advocates, foundations, and leaders who have dedicated their time to finding cures have not lost focus.  Despite any current or future challenges, #WeWontStop until cures and better treatments for pediatric brain tumors are discovered.

Throughout the month of May, the CBTTC will highlight the work being done right now to continue the fight against childhood brain tumors and to push public awareness of the devastating impact these diseases have on the lives of children and their families around the world. Additionally, We will continue to share the many ways researchers and patient communities alike can get involved; by sharing data and other resources, requesting CBTTC data to power new research efforts, utilizing our cloud-based platforms and analysis tools, or by providing philanthropic/awareness support.

Brand Development:
The CBTTC will become the Children’s Brain Tumor Network, effective September 2020. The CBTTC will develop new materials and branding for the CBTTC, both internally and through the support of external firms.

Final logo elements (minor icon design tweaks, color palettes, taglines and typeface) are being selected and will be determined by May 11, 2020. A survey for colors, taglines, etc. will be sent out this week to Advisory Council foundations for feedback. 

We sincerely thank each of you for taking the time to provide guidance and valuable feedback as we’ve worked through this rebranding process. With your key insights, the Executive Committee is confident that we will arrive at a final suite of brand materials that strongly convey the vision and mission of this consortium.



  • Vision & mission statements were finalized as of December 18, 2019
  • Brand pillars 
    • Pioneering Spirit | Global Inclusion | Open Science Model
  • Logo concept selected by Executive Committee on Monday, April 27th
  • Colorization and final typeface currently being selected and revised


Social Media Metrics - April 2020:


  • Audience Reach: 3,263 users (3,740 impressions)
  • Users Directly Engaged: 181
  • Engagement Rate: 4.83% (1-3%: industry avg.)
  • Followers Gained: 8
Facebook Audience Growth Goal:
50% growth from Sept. 2019 to end of FY2020 (455 followers to 682)
Current Followers: 533


  • Audience Reach: 7,057 users
  • Users Directly Engaged: 71
  • Engagement Rate: 1% (0.33% industry avg.)
  • Followers Gained: 5
Twitter Audience Growth Goal:
50% Growth from Sept. 2019 to end of FY 2020 (467 followers to 700)
Current Followers: 571

CBTTC Quarterly Newsletter:

Next Edition - Scheduled Thursday, May 21, 2020
Current Subscribers: 161
Growth Goals: 25% sustained growth in subscribers each issue
FY 2020 Subscriber Goal: 226 Subscribers

CBTTC Website Traffic - April 2020:

  • 620 Unique Users (80.8% New Users)
  • 807 Sessions
  • 1,497 Pageviews

Current Grant Submissions:

  • Pediatric Brain Tumor SPORE on track for submission in Sept 2020.
  • Presentation in May to NCI.
  • 123 letters of support (LOS) received to date.
Copyright © 2020 Children's Hospital of Philadelphia/Center for Data-Driven Discovery in Biomedicine, All rights reserved.

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