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Monthly CBTTC Advisory Council Report-Out
June 2020

To our patient partners,

We hope this finds you all safe and healthy. Thank you for your continued feedback on this report. We want to be mindful of your busy schedules, and have chosen to include the latest and most important updates below. Feel free to continue sending comments so we can improve these monthly reports and provide information most valuable to you. 

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 have finished meeting with CBTTC Leadership and the Advisory Council, and are compiling the data to share with you all in June for discussion, and hope to launch new strategies starting July 2020.

As you know, CBTTC remains “open” despite a lot of research being halted or slowed during COVID-19. During Brain Tumor Awareness Month, the CBTTC was proud to surpass our 100th approved scientific data project! Many thanks to you for helping to raise awareness of our goal to reach this milestone in May, and for encouraging our scientific community to submit data requests this month. Our data resources, including genomic and clinical data, continue to be available by request, and our lab is beginning to reopen in June!

During these trying times, please know that we are committed to doing what we can to join you all in raising awareness about pediatric brain tumors - this month and every month. #WeWontStop until every child has a cure.

Please find the report for May below. 


-CBTTC Executive Committee
Operations Report:

CBTTC Site Status:

As previously reported, CBTTC/PNOC Leadership sent invitations to PNOC sites that aren’t CBTTC sites already. This will facilitate and accelerate the transfer of data and specimens through our Master agreements, decreasing timelines and increasing efficiency. We will provide additional updates as soon as applications are received. 

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): 18
a listing of all sites can be found at:

  • New Site Applications (completed): 0
  • New Site Applications (in progress): 1
  • 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: 4,647

    • Children: 3,545 

    • 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) - 71
  • Ganglioglioma - 171
*Note: Suspension of lab activities due to COVID-19 social distancing measures have prevented the enrollment of new subjects during the month of May. As lab spaces begin to reopen at the CBTTC operations center and our member institutions beginning in June, we will resume biospecimen collection and sharing to the greatest extent possible.

Financial Updates:
As expected, philanthropic donations are down due in part to the current pandemic. As we continue to work with the PIs, their institutions, and donors, we will be reaching out to you for recommendations. Jena Lilly ( will act as the point of contact for questions and will be reaching out to discuss the situation. 

We're working rapidly to utilize new fundraising platforms in an effort to reach a broader audience. Early details are being developed through the rest of this week. Please keep watch for an additional eblast to all Advisory Council members, which will outline this effort and ways in which we could use your support!

Current CBTTC Grant Submissions:

Pediatric Brain Tumor SPORE - Mike Prados and Adam Resnick presented to the NCI SPORE program on May 27 and received approval to move forward with the submission in September 2020 as all requirements were met.
  • This would provide funding for four preclinical research projects, CBTN operations, and PNOC operations if awarded in late 2021 or early 2022. 
  • 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.
  • 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. 53 are CBTTC investigators and 66 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 a paper in 2020. All data are available and a request was sent out for anyone to contribute to the manuscript and analysis.


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: 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 May we have exceeded this goal with 47. Additionally, “processed data” is available anytime without request has been utilized thousands of times. You can find more information on the data projects here

  • 100 total requests approved

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 8 different scientific journals.

Accepted: 10 (one new since last report)

  1. Bobyn, A., Zarrei, M., Zhu, Y., Hoffman, M., Brenner, D., Resnick, A. C., Scherer, S. W., & Gallo, M. (2020). Ancestry and frequency of genetic variants in the general population are confounders in the characterization of germline variants linked to cancer. BMC medical genetics, 21(1), 92.

  2. 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.
  3. 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.
  4. 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
  5. 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.
  6. 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.
  7. 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.
  8. 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.
  9. 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.
  10. 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: 3
  1. 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).
  2. The Immunogenomic Landscape of Primary Pediatric Solid Tumours- (Submitted to Cancer Cell)
  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)

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:

2 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 will be presenting to the AACR meeting (June 22-24). Her abstract is #6746: Novel BRAF gene fusions in pediatric histiocytic neoplasms respond differently 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. Please reach out to Jena Lilly ( if you would like to sponsor.

HGG: 38
DMG: 12
ATRT: 11
Medulloblastoma: 7
Ependymoma: 7
LGG: 13
Ganglioglioma: 1
Chordoma: 3
Neurofibroma/Plexiform: 1
Oligodendroglioma: 2
Meningioma: 2


Disease Working Groups:
There is a new Imaging working group in addition to the working groups focused on Medulloblastoma, DMG, ATRT, LGG, Ependymoma, and Craniopharyngioma. 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. 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

DMG leads:

  • Sabine Mueller, MD, PhD; UCSF Benioff Children’s Hospital
  • Javad Nazarian, PhD; University Children’s Hospital of Zurich
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 new strategies. These have been extremely helpful and we thank you again for your time and recommendations. As noted above, we will be reaching out with the data collated in June for additional discussion.


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

Brain Tumor Awareness Month:

Throughout the month of May, the CBTTC has highlighted the work being done right now to continue the fight against childhood brain tumors, including 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.

Carried out over social media, the campaign included video messaging, blog content, graphics and childhood brain tumor facts, and the sharing of news stories, resources, new research, and the many actions taking place across our many community partnerships to raise funds and awareness for brain tumor research.

The campaign culminated on June 2 with our first-ever CBTTC Facebook Live panel discussion, where we welcomed researchers, clinicians, and patient families to discuss their involvement in the PNOC/CBTTC Clinical Trial Working Groups and the need for increased collaboration to tackle childhood brain cancer in the US and worldwide.

The information shared by our panelists beautifully conveyed the importance of this work, and has been viewed by over 1,800 users on Facebook as of this reporting. We'd like to thank not only Kristen Gillette, Al Gustafson, and Amanda Haddock for joining the panel, but also each of the AC member foundations who shared, commented, and/or helped to promote this event on social media. We hope you were able to watch live, but we also have archived a recording of the panel discussion on Facebook, YouTube, and our website. You can watch here:

Brand Development:
A new logo has been selected to represent the Children’s Brain Tumor Network! We are extremely grateful for all of your feedback and suggestions in the development of this logo and believe it represents the core mission of collaboration, connectivity and accelerated pace of discovery.


The CBTTC is continuing to develop materials and plans for the launch of the new Children’s Brain Tumor Network (CBTN) brand. Development of a new CBTN website is underway, with the communications team working to identify key features to include in the new website. If you have suggestions or feedback for website features to include, please contact us at

Until every child is cured - this tagline best aligns with the marketing/fundraising campaign to support CBTN operations. “We Won’t Stop...until every child is cured.”



  • Vision & mission statements were finalized as of December 18, 2019
  • Brand pillars 
    • Pioneering Spirit | Global Inclusion | Open Science Model
  • Final Logo concept selected by Executive Committee on May 13th
  • Brand style guide currently being developed
  • Website development underway


Social Media Metrics - May 2020:


  • Audience Reach: 8,135 users (9,006 impressions)
  • Users Directly Engaged: 531
  • Engagement Rate: 6.52% (1-3%: industry avg.)
  • Followers Gained: 27
Facebook Audience Growth Goal:
50% growth from Sept. 2019 to end of FY2020 (455 followers to 682)
Current Followers: 561


  • Audience Reach: 33,207 users
  • Users Directly Engaged: 397
  • Engagement Rate: 1.19% (0.33% industry avg.)
  • Followers Gained: 15
Twitter Audience Growth Goal:
50% Growth from Sept. 2019 to end of FY 2020 (467 followers to 700)
Current Followers: 585

CBTTC Website Traffic - May 2020:

  • 740 Unique Users (81.9% New Users)
  • 933 Sessions
  • 1,856 Pageviews


Copyright © 2020 Children's Hospital of Philadelphia/Center for Data-Driven Discovery in Biomedicine, All rights reserved.

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