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Monthly CBTTC Advisory Council Report-Out
August 2020

To our CBTTC partners,

Continuing through August we hope you all are healthy and enjoying the last month of summer. As discussed we will be rolling out our new membership structure in the coming months in line with the recruitment of our new Director of Partnership and Outreach Project Manager.  This month we would like to bring to you our new Brand Strategy document created by Bobby Moulder and begin working with those interested in the development of the new website. Our plan is to launch the new CBTN brand in September with our new website. If you would like to to assist in the design and content review of the website and associated launch materials please email Jonathan Waller (wallerj@email.chop.edu). 

In the Communications section, you will find links to view the Brand Strategy document and a new CBTN campaign poster for which we're seeking your feedback! 


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 last 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 21 member sites with the US, Australia, Italy, and China represented globally! 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 final 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. Please note additional primary sites receive annual funding so we are precluded from bringing on new primary sites currently, unless the site fund’s their participation they are sponsored.


Total Number of Sites (internationally): 21 
listing of all sites can be found at: https://cbttc.org/about-cbttc/member-institutions/
New Site Applications in Progress (not yet submitted): 1
New Site Applications: 3

  • Sydney Children’s
    • Onboarding Status: Application review by EB in progress
  • University Children's Zürich
    • Onboarding Status: Application review by EB in progress
  • St. Louis Children’s
    • Onboarding Status: Application review by EB in progress

New Sites Recently Approved and Onboarding: 3

  • NYU/Langone Children’s Hospital - Core Member
    • Onboarding Status: Agreement executed, regulatory review in progress
  •  Arnold Palmer Children’s Hospital - Data Satellite
    • Onboarding Status: Agreement executed, regulatory review in progress
  • University of Michigan/CS Mott Children’s Hospital - Data Satellite
    • Onboarding Status: Agreement executed, regulatory review in progress

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: 21
  • Overall Total Enrolled
    • All-Time: 4,672
    • FY21: 24
  • Total Children Enrolled
    • All-Time: 3,558
    • FY21: 12
  • Total Mothers Enrolled
    • All-Time: 629
    • FY21: 9
  • Total Fathers Enrolled
    • All-Time: 485
    • FY21: 3
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 https://portal.kidsfirstdrc.org/dashboard.

Histology    |   Number of Subjects Enrolled with this Diagnosis
  • ATRT - 98
  • DMG - 133
  • Medulloblastoma - 436
  • Low Grade Glioma - 993
  • High Grade Glioma - 346
  • Ependymoma - 316
  • Craniopharyngioma - 128
  • Dysembryoplastic Neuroeithelial Tumor (DNET) - 76
  • Ganglioglioma - 181

Financial Updates:

NIH Grant Submission
Pediatric Brain Tumor SPORE: At this time there is a new opportunity 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. 55 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 https://cbttc.org/research/scientific-projects-biospecimen/

All-time total number of requests: 74

  • 61 total requests approved
    • 44 sample requests approved
    • 17 cell line requests approved
  • 4 requests changed to data access requests (data was already generated)
  • 3 requests were withdrawn from consideration by the investigator
  • 1 request is now under review by the Scientific Committee for approval
  • 1 requests are in primary review
  • 4 request is with the investigator for revisions


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 https://cbttc.org/scientific-projects-data/

  • 109 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 11 different scientific journals.

Total Accepted: 15
Accepted this month: 5

  1. Bjork, I., Peralez, J., Haussler, D., Spunt, S.L., Vaske, O.M. 2019. Data sharing for clinical utility. Cold Spring Harbor Molecular Case Studies . Oct 23;5(5), pii: a004689
     
  2. Pfeil, J., Sanders, L.M., Anastopoulos, I., et al. 2020. Hydra: A mixture modeling framework for subtyping pediatric cancer cohorts using multimodal gene expression signatures. PLoS Comput Biol,16(4):e1007753. doi:10.1371/journal.pcbi.1007753
     
  3. Sanders, L.M., Rangaswami, A., Bjork, I., Lam, D.L., Beale, H.C., Kephart E.T., Durbin, A., Learned, K., Currie, R. A., Lyle, G., Pfeil, J., Shah, A.T., Lee, A.G., Leung, S.G., Behroozfard, I.H, Breese, M.R., Peralez, J., Hazard, F.K, Lacayo, N., Spunt, S.L., Haussler, D., Salama, S.R., Sweet-Cordero, E.A., and Vaske, O.M. 2019. Comparative RNA-seq analysis aids in diagnosis of a rare pediatric tumor. Molecular Case Studies.Oct 23;5(5), pii: a004317
     
  4. Vaske, O.M., Bjork, I., Salama, S.R., et al. 2019. Comparative Tumor RNA Sequencing Analysis for Difficult-to-Treat Pediatric and Young Adult Patients With Cancer. JAMA Netw Open, 2(10):e1913968. doi:10.1001/jamanetworkopen.2019.13968
     
  5. Vaske, O.M. and Haussler, D. 2019. Data sharing for pediatric cancers. Science, 363(6432):1125
Total Currently Submitted: 5
Submitted this month: 2
  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: https://www.aanmelder.nl/biosb2020"
     
  2. Submitted an abstract titled “Reconstructing the Gene Regulatory Landscape of Pediatric BrainTumors” to the 2020 ISMB meeting (Regulatory and Systems Genomics track)
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 (lillyj@gmail.com) 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:


Events/Communications:

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




Brand Development Updates:
The CBTN website will soon be ready for your comments and feedback - we’ll be sharing a link with you in the next few weeks and will provide you with additional opportunities to review as we continue to develop this new resource. The website will have a larger focus on the CBTN’s discovery projects and make it simpler for users to find the information they are looking for. 

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 (moulderr@email.chop.edu) or communications@cbttc.org. 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 communications@cbttc.org.


Social Media Metrics - July 2020:

Facebook

  • Audience Reach: 740 users (860 impressions)
  • Users Directly Engaged: 37
  • Engagement Rate: 5% (1-3%: industry avg.)
  • Followers Gained: 2
Current Followers: 574

Twitter

  • Audience Reach: 6,040 users
  • Users Directly Engaged: 77
  • Engagement Rate: 1.27% (0.33% industry avg.)
  • Followers Gained: 13
Current Followers: 606


CBTTC Website Traffic - July 2020:

  • 719 Unique Users (83.6% New Users)
  • 948 Sessions
  • 1,735 Pageviews
Past Reports
 
Did you miss one of our last CBTTC Advisory Council Reports? You can access them here!
Follow us on Twitter
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Visit our Website
Copyright © 2020 Children's Hospital of Philadelphia/Center for Data-Driven Discovery in Biomedicine, All rights reserved.


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