Thank you to everyone for making this year’s meeting a productive, interactive event. To ensure that we continue the momentum, I strongly encourage each of you to help me find the “champions” for the suggested working groups: 1) think tank for rapid translation into clinical trials, and 2) early stage investigator support. We need someone who is not only enthusiastic but also willing to help lead each of these working groups.
Now, on to the meeting summary.
On Sunday, March 10, we held an experiment – an interactive experience to engage CBTTC stakeholders in a discussion of “Positive core values” and “Envisioning what could be,” over three distinct sessions.
The first session kicked off the evening with a cocktail hour, and an opportunity for CBTTC stakeholders across multiple fronts to engage and get to know one another. Attendees were assigned to tables, and prompted to complete 1:1 interviews. There were lively discussions as foundation partners and institution members discussed their backgrounds, and shared vision and goals for CBTTC.
During the second session, participants shared 1:1 table discussions with their tables and the larger group. We found there was consensus that CBTTC’s positive core will accelerate discoveries for cures for all children with brain tumors, rare and common disease types alike. In fact, some of our stakeholders envisioned a future where CBTTC does not exist because it is no longer needed. In the interim, CBTTC’s strengths of anticipatory planning (of future needs), attention to detail (as outlined by our Master Agreements and Data Use Agreements), and efficient team at the Operations center provide structure and guidance to continued success of this consortium. By setting aside our individual egos to participate in a collaborative research model of shared biospecimens and clinical data, each institution is helping build CBTTC’s positive core.
In the third and final session, after dinner was served, table discussions focused on what needs to be done to achieve what we envisioned for the future of CBTTC. Each table approached this conversation somewhat differently; however, the group discussion involved many shared themes. In the future, the CBTTC data structure and research platforms could empower real-time clinical decision support by setting the standard for clinical data sharing. CBTTC could also harness existing computing power for discovery of new therapeutic targets. In order to achieve these goals, we would need to harness industry, both pharma and biotech, as well as continue to partner with clinical trial-based consortiums. As we continue to challenge ourselves and encourage scientists to develop research projects to address existing gaps in pediatric brain tumor research we also need to make sure we are maximizing use of existing data and contributing to the “de-siloing” of research. Some concrete “asks” from the Sunday evening group included:
1) Rebranding – CBTTC is really hard to say and doesn’t capture what we are doing (not a “passive” repository, instead an “active” research entity), and
2) improve education/outreach on website and information to families.
As the current CBTTC Executive Board Chair, I was impressed with what each table presented during sessions 2 and 3. I will take all of the recommendations back to CBTTC leadership to discuss, prioritize, and work into FY20 objectives.
On Monday morning, March 11, we started the official meeting with a session outlining how to access data generated by the Pediatric Brain Tumor Atlas (PBTA). This session will forever be known as the “Elephant Session”. This is one of those “you had to be there” types of topics. Dr. Adam Resnick, the CBTTC Scientific Co-Chair, reviewed the types of data generated to date, and Dr. Allison Heath performed a live demo of how to access the research platforms to review and analyze the different types of data available. Both stressed that the data is housed using NIH-Entrusted Partner guidelines; therefore, to access genomic raw data through the Kids First Data Resource Portal, an NIH eCommons logon/password is required for authentication. Action item post-meeting: Kids First DRC and the CBTTC need to develop education modules including YouTube type of videos on how/where to access data. For now, though, please request access to CBTTC generated data through www.CBTTC.org, go to the Research tab, and submit a Data Use Agreement.
The second session: CBTTC Scientific Project Updates. This started off with an overview of current approved biospecimen and data projects by Dr. Javad Nazarian, CBTTC Scientific Co-Chair. Four different investigators were asked to present on their projects to highlight the potential translational impact and pathway to the clinic. Dr. Brian Rood from Children’s National Medical Center presented on his work on proteomics, both the NIH funded work and his individual proteogenomic project. Both are exciting and contribute to the complexity of data empowered by CBTTC. Dr. Sameer Agnihotri from the UPMC Children’s Hospital of Pittsburgh presented on his two approved projects – an integrated genomic analysis looking at PIK3CA and 10q LOH in high-grade gliomas, and an elucidation of the somatic epigenetic landscape of meningiomas and schwannomas. The scientific session ended with information on two different NIH collaborative efforts involving high-grade gliomas and single cell sequencing, Pediatric Cell Tumor Atlas and Project HOPE. Dr. Kristina Cole from Children’s Hospital of Philadelphia presented on the newly awarded Center for Pediatric Cell Tumor Atlas, a U21 Cancer Moonshot funded study as part of the Human Tumor Atlas Network, with focus on longitudinal single cell sequencing (evaluating cells at different treatment time points). Dr. Michael Prados from University of California San Francisco presented on Project HOPE (High Grade Glioma -Omics in Pediatrics) focus on single cell sequencing analysis over multiple cell types from a single tumor specimen. The presentations stimulated conversation and ideas, and left us with an action item post-meeting: There is a need for formation of a CBTTC clinical trial collaborative working group to focus on bringing findings to clinical trials.
On Monday afternoon after an interactive lunch in which we learned unusual facts about different attendees, the meeting ended with an introduction of the new full member and satellite member sites, and opportunities for collaborations. The afternoon started off with Dr. Marilyn Li from Children’s Hospital of Philadelphia providing information on how CBTTC is directly contributing to the American Association of Cancer Research – Project GENIE. This is a national effort to catalyze precision oncology through the development of a regulatory-grade registry aggregating and linking clinical-grade cancer genomic data with clinical outcomes. The overall goal is to power translational research. Newest approved sites include Johns Hopkins University, All Children’s Hospital, Dayton Children’s Hospital, Wake Forest University, and Nemours/AI duPont Hospital for Children. The enthusiasm and the expertise that the new sites will bring was exciting to hear. Dr. Eric Raabe started off the introduction, and as the newest full member site will bring not 1, but 2 hospitals on board (Bloomberg Children’s Center in Baltimore, MD, and All Children’s Hospital in St Petersberg, FL). Dr. Robert Lober from Dayton Children’s Hospital highlighted how valuable it is to bring on smaller community hospitals. Dr. David Kram’s group from Brenner’s Children’s Hospital at Wake Forest will bring expertise in developing preclinical organoid tumor cell models. And, Dr. Andrew Walter from AI Dupont in Wilmington, DE, ended the introductions highlighting how valuable consortium based collaborations are to improving clinical outcomes.
The afternoon also included two inspiring talks and a panel forum discussion. Dr. Jie Ma, an invited speaker form Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of Medicine in Shanghai, China, described the newly formed Chinese Children’s Neuro-Oncology group bringing together 35 institutions across mainland China. His impressive background and 20+ year career in Neurosurgery is only outshone by his clear enthusiasm for this new endeavor. Dr. Michael Prados, an invited speaker who also presented earlier in the morning, ended the afternoon session with highlights from the Pacific Pediatric Neuro-Oncology Consortium (PNOC). CBTTC and PNOC working together can authentically accelerate the pace of translational research.
The meeting ended with a panel forum with CBTTC Leadership (Executive Board Chair, Scientific Co-Chairs) fielding questions from the audience and submitted earlier by foundations.
Thank you to everyone who participated on Sunday and/or Monday, over this next year, we will continue to build upon the momentum from this meeting.
Angela J Waanders, MD, MPH
Executive Board Chair, Children’s Brain Tumor Tissue Consortium
Director of Precision Medicine in Oncology, Ann & Robert H. Lurie Children’s Hospital of Chicago
Associate Professor of Pediatrics, Northwestern University Feinberg School of Medicine