Childhood Cancer Data Initiative Annual Symposium (Abstract Registration)

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# Created User IP address Abstract Title: Abstract: Abstract: Authors: Presenting Author: Institution: Email Address: Operations
45 Tue, 08/19/2025 - 12:15 Anonymous 10.208.28.152 Enrollment in Children’s Oncology Group’s Clinical Trials: Population-Based Linkage with the National Childhood Cancer Registry Background: Improvements in outcomes among children and adolescents diagnosed with cancer are attributable to many factors, including clinical trials such as those administered through the Children’s Oncology Group (COG), as well as population-based resources like the National Childhood Cancer Registry (NCCR). The objective of this study was to link COG trial data with the NCCR to evaluate overall enrollment patterns.

Methods: Data were received from the NCCR and COG, which were linked using an array of variables and then compared to evaluate enrollment patterns in COG studies from 2007-2018. Multivariable logistic regression was used to identify characteristics associated with not being enrolled in a COG study.

Results: Among 134,696 NCCR cancer patients, 51,062 matched with COG study enrollees. There were several differences in demographic and clinical characteristics between those enrolled and not enrolled in COG studies. Enrollment was higher among children aged 0-4 years compared to adolescents aged 15-19 years (53.7% vs 20.1%). Differences by race and ethnicity were also observed; for example, those who identified as non-Hispanic White were more likely to be enrolled than those who identified as non-Hispanic Asian/Pacific Islander (38.8% vs 32.9%). In a multivariable logistic regression model, several characteristics were significantly associated with not being enrolled in a COG study, including age at diagnosis, year of diagnosis, race and ethnicity, and cancer type.

Conclusion: Our results suggest that several groups are underrepresented in COG clinical trials. This information can help guide the prioritization of population groups for engagement in future studies.
  1. First Name: Philip
    Middle Initial: J
    Last Name: Lupo
    Degree(s): Ph.D.
    Organization: Emory University
  2. First Name: David
    Middle Initial: A
    Last Name: Siegel
    Degree(s): M.D., MPH
    Organization: Centers for Disease Control and Prevention
  3. First Name: Nicola
    Middle Initial: C
    Last Name: Schussler
    Degree(s): BS
    Organization: Information Management Services, Inc
  4. First Name: Todd
    Middle Initial: A
    Last Name: Alonzo
    Degree(s): Ph.D.
    Organization: University of Southern California
  5. First Name: Suzanne
    Last Name: Adams
    Degree(s): ODS-C, BS
    Organization: Information Management Services, Inc
  6. First Name: David
    Last Name: Angelaszek
    Degree(s): MS
    Organization: Information Management Services, Inc
  7. First Name: Shanthala
    Last Name: Basavappa
    Degree(s): MS
    Organization: Essex, an Emmes Company
  8. First Name: Tiffany
    Middle Initial: M
    Last Name: Chambers
    Degree(s): MPH
    Organization: Baylor College of Medicine
  9. First Name: Linda
    Last Name: Colye
    Degree(s): BS
    Organization: Information Management Services, Inc
  10. First Name: Eric
    Last Name: Durbin
    Degree(s): Dr.PH., MS
    Organization: University of Kentucky
  11. First Name: Johanna
    Middle Initial: L
    Last Name: Goderre
    Degree(s): MPH
    Organization: National Cancer Institute
  12. First Name: Tiffany
    Last Name: Hayes
    Degree(s): MPH
    Organization: Essex, an Emmes Company
  13. First Name: Will
    Last Name: Howe
    Degree(s): BS
    Organization: Information Management Services, Inc
  14. First Name: Elizabeth
    Last Name: Hsu
    Degree(s): Ph.D.
    Organization: National Cancer Institute
  15. First Name: Richard
    Last Name: Lee
    Degree(s): BS
    Organization: Information Management Services, Inc
  16. First Name: Denise
    Middle Initial: R
    Last Name: Lewis
    Degree(s): Ph.D., MPH
    Organization: National Cancer Institute
  17. First Name: Angela
    Middle Initial: B
    Last Name: Mariotto
    Degree(s): Ph.D.
    Organization: National Cancer Institute
  18. First Name: Brad
    Middle Initial: H
    Last Name: Pollock
    Degree(s): Ph.D., MPH
    Organization: University of California Davis
  19. First Name: Anca
    Last Name: Preda
    Degree(s): M.D., MPH
    Organization: National Cancer Institute
  20. First Name: Michael
    Middle Initial: E
    Last Name: Roth
    Degree(s): M.D.
    Organization: The University of Texas MD Anderson Cancer Center
  21. First Name: Jennifer
    Last Name: Stevens
    Degree(s): BS
    Organization: Information Management Services, Inc
  22. First Name: Tina
    Last Name: Terranova
    Degree(s): MSc
    Organization: ICF
  23. First Name: Sarah
    Middle Initial: L
    Last Name: Vargas
    Degree(s): Ph.D.
    Organization: Children's Oncology Group
  24. First Name: Douglas
    Middle Initial: S
    Last Name: Hawkins
    Degree(s): M.D.
    Organization: University of Washington
  25. First Name: Lynne
    Last Name: Penberthy
    Degree(s): M.D., MPH
    Organization: National Cancer Institute
 David Siegel Centers for Disease Control and Prevention irn3@cdc.gov
43 Fri, 08/08/2025 - 15:52 Anonymous 10.208.24.35 Extrachromosomal DNA associates with poor survival across a broad spectrum of childhood solid tumors Circular extrachromosomal DNA (ecDNA) is a common form of oncogene amplification in aggressive cancers. The frequency and diversity of ecDNA has been catalogued in adult and some childhood cancers; however, its role in most pediatric cancers is not well-understood. To address this gap, we accessed large pediatric cancer genomics data repositories and identified ecDNA from whole genome sequencing data using cloud computing. This retrospective cohort comprises 3,631 solid tumor biopsies from 2,968 patients covering all major childhood solid tumor types. Aggressive tumor types had particularly high incidences of ecDNA. Pediatric patients whose tumors harbored extrachromosomal DNA had significantly poorer five-year overall survival than children whose tumors contained only chromosomal amplifications. We catalogue known and potentially novel oncogenes recurrently amplified on ecDNA and show that ecDNA often evolves during disease progression. These results highlight patient populations that could potentially benefit from future ecDNA-directed therapies. To facilitate discovery, we developed an interactive catalogue of ecDNA in childhood cancer at https://ccdi-ecdna.org/.
  1. First Name: Owen
    Middle Initial: S
    Last Name: Chapman
    Degree(s): PhD
    Organization: Nagoya City University
  2. First Name: Sunita
    Last Name: Sridhar
    Degree(s): MD
    Organization: Rady Children's Hospital San Diego
  3. First Name: Eugene
    Last Name: Yui-Ching
    Degree(s): PhD
    Organization: SBP Medical Discovery Institute
  4. First Name: Rishaan
    Last Name: Kenkre
    Organization: SBP Medical Discovery Institute
  5. First Name: Jonathan
    Last Name: Kirkland
    Organization: UC San Diego
  6. First Name: Aditi
    Last Name: Dutta
    Organization: UC San Diego
  7. First Name: Shanqing
    Last Name: Wang
    Organization: UC San Diego
  8. First Name: Wenshu
    Last Name: Zhang
    Organization: UC San Diego
  9. First Name: Miguel
    Last Name: Brown
    Degree(s): PhD
    Organization: Children's Hospital of Philadelphia
  10. First Name: Jens
    Last Name: Luebeck
    Degree(s): PhD
    Organization: UC San Diego
  11. First Name: Yan
    Middle Initial: Yuen
    Last Name: Lo
    Organization: SBP Medical Discovery Institute
  12. First Name: Elias
    Last Name: Rodrigez-Fos
    Degree(s): PhD
    Organization: Experimental and Clinical Research Center of the Max Delbrück Center and Charite Berlin
  13. First Name: Anton
    Middle Initial: G
    Last Name: Henssen
    Degree(s): MD, PhD
    Organization: Experimental and Clinical Research Center of the Max Delbrück Center and Charite Berlin
  14. First Name: Konstantin
    Last Name: Okonechnikov
    Degree(s): PhD
    Organization: Hopp Children’s Cancer Center Heidelberg (KiTZ)
  15. First Name: David
    Middle Initial: R
    Last Name: Ghasemi
    Degree(s): MD, PhD
    Organization: Hopp Children’s Cancer Center Heidelberg (KiTZ)
  16. First Name: Kristian
    Middle Initial: W
    Last Name: Pajtler
    Degree(s): MD PhD
    Organization: Hopp Children’s Cancer Center Heidelberg (KiTZ)
  17. First Name: Daisuke
    Last Name: Kawauchi
    Degree(s): PhD
    Organization: Nagoya City University
  18. First Name: Vineet
    Last Name: Bafna
    Degree(s): PhD
    Organization: UC San Diego
  19. First Name: Megan
    Last Name: Pau;
    Degree(s): MD
    Organization: Rady Children's Hospital San Diego
  20. First Name: Kevin
    Last Name: Yip
    Degree(s): PhD
    Organization: SBP Medical Discovery Institute
  21. First Name: Jill
    Last Name: Mesirov
    Degree(s): PhD
    Organization: UC San Diego
  22. First Name: Lukas
    Last Name: Chavez
    Degree(s): PhD
    Organization: SBP Medical Discovery Institute
Lukas Chavez SBP Medical Discovery Institite lchavez@sbpdiscovery.org
42 Thu, 08/07/2025 - 14:53 Anonymous 10.208.28.64 Childhood Cancer Data Initiative Federation API: Integrating Data Across Pediatric Cancer Institutions While advances have been made in pediatric cancer treatments, an ongoing challenge is improving outcomes for patients with rare cancer subtypes as well as patients with refractory or recurrent disease. A continuing barrier is the siloing of data across institutions, preventing researchers from assembling the large cohorts necessary to study cancer mechanisms and develop novel therapies. To address this issue, the NCI’s Childhood Cancer Data Initiative (CCDI) developed the CCDI Federation API in 2022, creating a platform that aggregates and integrates clinical and research data from multiple pediatric cancer institutions. Developed in collaboration with Children’s Hospital of Philadelphia, Pediatric Cancer Data Commons, St. Jude Children’s Research Hospital, and Treehouse Childhood Cancer Initiative, the Federation API was designed to be easily integrated into other NCI resources and includes detailed implementation guides for new participating institutions. The Federation API empowers users to discover, access, and visualize data on hundreds of thousands of samples from thousands of patients, accelerating novel scientific discoveries and enhancing data access for researchers, clinicians, patients, and families.
  1. First Name: Subhashini
    Last Name: Jagu
    Degree(s): Ph.D.
    Organization: National Cancer Institute
  2. First Name: Holly
    Middle Initial: C
    Last Name: Beale
    Degree(s): Ph.D.
    Organization: University of California, Santa Cruz
  3. First Name: Ellen
    Middle Initial: T
    Last Name: Kephart
    Degree(s): B.S.
    Organization: University of California, Santa Cruz
  4. First Name: Olena
    Middle Initial: M
    Last Name: Vaske
    Degree(s): Ph.D.
    Organization: University of California, Santa Cruz
  5. First Name: Melissa
    Middle Initial: S
    Last Name: Cline
    Degree(s): Ph.D.
    Organization: University of California, Santa Cruz
  6. First Name: Jingchun
    Last Name: Zhu
    Degree(s): Ph.D.
    Organization: University of California, Santa Cruz
  7. First Name: Mary
    Middle Initial: J
    Last Name: Goldman
    Organization: University of California, Santa Cruz
  8. First Name: Brian
    Last Name: Craft
    Organization: University of California, Santa Cruz
  9. First Name: Allison
    Middle Initial: P
    Last Name: Heath
    Degree(s): Ph.D.
    Organization: Children's Hospital of Philadelphia
  10. First Name: Hannah
    Middle Initial: R
    Last Name: Calkins
    Degree(s): M.S.
    Organization: Children's Hospital of Philadelphia
  11. First Name: William
    Middle Initial: N
    Last Name: Khan
    Degree(s): B.S.
    Organization: Children's Hospital of Philadelphia
  12. First Name: Xu
    Last Name: Zhu
    Degree(s): M.S.
    Organization: Children's Hospital of Philadelphia
  13. First Name: Christopher
    Middle Initial: M
    Last Name: Nemarich
    Degree(s): M.A.
    Organization: Children's Hospital of Philadelphia
  14. First Name: Jinghui
    Last Name: Zhang
    Degree(s): Ph.D.
    Organization: St. Jude Children's Research Hospital
  15. First Name: Michael
    Last Name: Rusch
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  16. First Name: Clay
    Last Name: McLeod
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  17. First Name: Stephanie
    Last Name: Sandor
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  18. First Name: Jobin
    Middle Initial: Jacob
    Last Name: Sunny
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  19. First Name: Delaram
    Last Name: Rahbarinia
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  20. First Name: Kevin
    Last Name: Benton
    Degree(s): M.S.
    Organization: St. Jude Children's Research Hospital
  21. First Name: Jake
    Last Name: Noble
    Degree(s): B.A.
    Organization: St. Jude Children's Research Hospital
  22. First Name: Samuel
    Middle Initial: L
    Last Name: Volchenboum
    Degree(s): M.D., Ph.D.
    Organization: University of Chicago, Department of Pediatrics
  23. First Name: Luca
    Last Name: Graglia
    Degree(s): M.S.
    Organization: University of Chicago, Department of Pediatrics
  24. First Name: Michael
    Middle Initial: T
    Last Name: Watkins
    Degree(s): Ph.D.
    Organization: University of Chicago, Department of Pediatrics
  25. First Name: Brian
    Middle Initial: T
    Last Name: Furner
    Degree(s): M.S.
    Organization: University of Chicago, Department of Pediatrics
  26. First Name: Lydia
    Last Name: Rogers
    Degree(s): MBA
    Organization: Frederick National Laboratory for Cancer Research
  27. First Name: Natalia
    Last Name: Asafieva
    Degree(s): Ph.D.
    Organization: Frederick National Laboratory for Cancer Research
  28. First Name: Patrick
    Last Name: Dunn
    Degree(s): Ph.D.
    Organization: Frederick National Laboratory for Cancer Research
  29. First Name: Mark
    Last Name: Cunningham
    Degree(s): M.A.
    Organization: Frederick National Laboratory for Cancer Research
  30. First Name: Janisha
    Last Name: Patel
    Degree(s): M.S.
    Organization: Frederick National Laboratory for Cancer Research
 A. Geoffrey Lyle University of California, Santa Cruz aglyle@ucsc.edu
41 Thu, 08/07/2025 - 13:55 Anonymous 10.208.28.64 Pediatric PRISSMM model for real world medical record data to capture stage, treatment and cancer outcomes Background: Approaches to obtaining structured clinical oncology data from the medical record are not standardized. PRISSMM was developed to reliably and reproducibly capture real-world oncology clinical data for adult solid malignancies from the medical record. We adapted the PRISSMM method to pediatric solid tumors(pediPRISSM) and assessed the feasibility of using pediPRISSMM to determine stage at presentation and progression-free survival(PFS) in patients with Ewing sarcoma(EWS). Methods: Patients diagnosed with EWS who had tumor molecular profiling had medical records abstracted using pediPRISSMM at two institutions. Staging was derived from disease site information from imaging performed during staging (4 weeks from diagnosis). PFS from the diagnosis was based on disease status from radiology reports(PFSimaging) and chemotherapy drug regimen start and stop dates(PFSdrugs). Results: 158 patients with a confirmed EWS-associated fusion were included. Median age at diagnosis was 17 years (range of 1-75), 63% were male and 80% had primary bone disease. Currently, complete pediPRISSM data are available for 43 patients. It was possible to derive stage from pediPRISSMM radiology data for 63% of patients. PFSimaging and PFSdrugs were concordant in 74% of patients. Medical record review for patients with a difference of >6 weeks in PFS revealed multi-institutional care as the most common reason for discordance. Conclusions: Although further analysis is ongoing, these data support the universal implementation of accurate staging forms as part of routine clinical documentation. Concordant findings using PFSimaging and PFSdrugs were achieved in the majority of patients, with highest success seen in patients treated at single institutions.
  1. First Name: Sidney
    Middle Initial: N
    Last Name: Benich
    Degree(s): M.S.
    Organization: Dana-Farber Cancer Institute
  2. First Name: Stephanie
    Last Name: Suser
    Degree(s): B.A.
    Organization: Memorial Sloan Kettering Cancer Center
  3. First Name: Evelina
    Last Name: Ceca
    Degree(s): MBA
    Organization: Dana-Farber Cancer Institute
  4. First Name: Liliana
    Last Name: Rodriguez
    Organization: Memorial Sloan Kettering Cancer Center
  5. First Name: Madhumitha
    Last Name: Sridharan
    Degree(s): B.E.
    Organization: Dana-Farber Cancer Institute
  6. First Name: Julian
    Last Name: Schwartz
    Degree(s): B.S.
    Organization: Memorial Sloan Kettering Cancer Center
  7. First Name: Zachary
    Last Name: Kahn
    Degree(s): B.S.
    Organization: Dana-Farber Cancer Institute
  8. First Name: Anita
    Last Name: Bowman
    Degree(s): M.S.
    Organization: Memorial Sloan Kettering Cancer Center
  9. First Name: Joseph
    Last Name: White
    Degree(s): Ph.D.
    Organization: Dana-Farber Cancer Institute
  10. First Name: Rose
    Last Name: Brannon
    Degree(s): Ph.D.
    Organization: Memorial Sloan Kettering Cancer Center
  11. First Name: Wendy
    Middle Initial: B
    Last Name: London
    Degree(s): Ph.D.
    Organization: Dana-Farber Cancer Institute
  12. First Name: Neerav
    Middle Initial: N
    Last Name: Shukla
    Degree(s): M.D.
    Organization: Memorial Sloan Kettering Cancer Center
  13. First Name: Katherine
    Middle Initial: A
    Last Name: Janeway
    Degree(s): M.D., MMSc
    Organization: Dana-Farber Cancer Institute
 Sidney Benich Dana-Farber Cancer Institute sidneyn_benich@dfci.harvard.edu
40 Thu, 08/07/2025 - 10:37 Anonymous 10.208.28.64 Optimized Syngeneic Neuroblastoma Models to Advance Cell Therapies Adoptive cell therapies, including T and natural killer T (NKT) cells expressing GD2 specific chimeric antigen receptors (CAR), have shown promise in treating relapsed/refractory (r/r) neuroblastoma (NB) but remain non-curative. A significant barrier to improving these therapies is the reliance on xenogeneic models, which fail to capture the full spectrum of innate and adaptive immune responses. We recently reported that CAR NKTs have superior in vivo antitumor activity in several syngeneic tumor models (PMID: 39354225), but not in NB due to the lack of relevant models.

To develop syngeneic NB models for evaluating CAR T and CAR NKT cell therapies, murine NB cell lines expressing GD2 and B7H3 were created via lentiviral transduction and optimized through orthotopic implantation into C57BL/6 mice. These cell lines were engineered for cyclophosphamide (Cy) resistance to model r/r NB and support lymphodepleting preconditioning. The tumor microenvironment (TME) was characterized using flow cytometry. Murine T and NKT cells were transduced with GD2/B7H3 CARs encoding CD28 or 41BB costimulatory domains.

Results showed that GD2 and B7H3 NB models exhibited high tumor inoculation rates and stable antigen expression. The TME featured diverse immune populations. In vitro, CAR T/NKTs eliminated antigen-specific NB cells. In vivo, both therapies reduced tumor burden post Cy preconditioning, mirroring clinical trial outcomes but without achieving complete tumor control.

To conclude, we developed Cy resistant syngeneic orthotopic NB models with stable GD2 and B7H3 expression. These models enable a detailed study of cell therapies in the context of an intact immune system and TME.
  1. First Name: Ying
    Last Name: Wang
    Organization: Baylor College of Medicine
  2. First Name: Linjie
    Last Name: Guo
    Degree(s): M.D. Ph.D.
    Organization: Baylor College of Medicine
  3. First Name: Akshaya
    Last Name: Adaikkalavan
    Organization: Baylor College of Medicine
  4. First Name: Amy
    Middle Initial: N.
    Last Name: Courtney
    Degree(s): Ph.D.
    Organization: Baylor College of Medicine
  5. First Name: Xavier
    Last Name: Rios
    Degree(s): M.D. Ph.D.
    Organization: Baylor College of Medicine
  6. First Name: Xin
    Last Name: Xu
    Degree(s): Ph.D.
    Organization: Baylor College of Medicine
  7. First Name: Michael
    Middle Initial: S.
    Last Name: Wood
    Organization: Baylor College of Medicine
  8. First Name: Erica
    Middle Initial: Di
    Last Name: Pierro
    Degree(s): Ph.D.
    Organization: Baylor College of Medicine
  9. First Name: Xin
    Last Name: Zhou
    Degree(s): Ph.D.
    Organization: University of North Carolina at Chapel Hill
  10. First Name: Gianpietro
    Last Name: Dotti
    Degree(s): M.D.
    Organization: University of North Carolina at Chapel Hill
  11. First Name: Leonid
    Middle Initial: S.
    Last Name: Metelitsa
    Degree(s): M.D. Ph.D.
    Organization: Baylor College of Medicine
 Ying Wang Baylor College of Medicine ying.wang3@bcm.edu
39 Wed, 08/06/2025 - 22:33 Anonymous 10.208.28.64 Developing neuroprotective nanotheranostic agents for the image-guided treatment of radiotherapy-induced brain injury Introduction: Radiotherapy-induced brain injury (RIBI) affects up to 90% of brain tumor survivors treated with radiotherapy. Thus, there is a need for RIBI therapeutic strategies.

Hypothesis: Oxidative stress and neuroinflammation are key contributors to RIBI. Thus, therapies that reduce oxidative stress and neuroinflammation could mitigate RIBI.

Objective: Two redox-responsive nanotheranostic agents were developed and evaluated in a preclinical mouse model of RIBI.

Method: Two agents (P2a and P2b) with varying numbers of phenylboronic acid pinacol ester (BAPE) moieties to scavenge reactive oxygen species (ROS) were developed and characterized. The agents were next intravenously injected (IV) into mice at two weeks post-irradiation, as follows: Group 1 [phosphate buffered saline (PBS)]; Group 2 (P2a); Group 3 (P2b). The mice were then monitored with fluorescence imaging; magnetic resonance imaging (MRI) and immunohistochemistry.

Results: Both agents accumulated in the irradiated region of the mouse brain 4 h post-IV and were retained for 7 days. Contrast-enhanced T1W MRI, 1.5 months post-IV, showed significantly reduced disruption of the blood brain barrier in mice treated with P2b compared to PBS and P2a. T2W MRI also showed significantly reduced edema/astrogliosis in mice treated with P2b and P2a, compared to PBS. Immunohistochemistry confirmed significantly reduced microglial activation in mice treated with P2b and P2a, compared to PBS; and reduced infiltrative macrophages in mice treated with P2b compared to P2a and PBS.

Conclusion: These results show that redox-responsive nanotheranostic agents with a high number of ROS scavengers can mitigate RIBI-associated neuroinflammation and improve RIBI outcomes in brain tumor survivors.
  1. First Name: Saikat
    Last Name: Maiti
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  2. First Name: Maya
    Last Name: Teitz
    Degree(s): B.Sc.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  3. First Name: Esteban
    Last Name: Velarde
    Degree(s): B.Sc.
    Organization: Department of Radiation Oncology and Molecular Radiation Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21231
  4. First Name: Xiaoju
    Last Name: Yang
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  5. First Name: Shana
    Last Name: Lee
    Degree(s): B.SC.
    Organization: Department of Molecular and Comparative Pathobiology, Johns Hopkins University School of Medicine, Baltimore, MD 21205
  6. First Name: Kristen
    Last Name: Lecksell
    Degree(s): B.Sc.
    Organization: Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  7. First Name: Barbara
    Middle Initial: J.
    Last Name: Smith
    Degree(s): B.Sc.
    Organization: Department of Cell Biology, Baltimore, MD 21205
  8. First Name: Anupama
    Last Name: Kumari
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  9. First Name: Adnan
    Last Name: Bibic
    Degree(s): Ph.D.
    Organization: F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD 21205
  10. First Name: Ethel
    Middle Initial: J.
    Last Name: Ngen
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287; Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD 21287
Saikat Maiti, Ph.D. Johns Hopkins University School of Medicine smaiti1@jhmi.edu
38 Wed, 08/06/2025 - 16:23 Anonymous 10.208.24.36 Nanopore Sequencing for Pediatric CNS Tumor Classification Central nervous system (CNS) tumors are the most common solid tumors diagnosed in children, making up about 17% of diagnosed childhood cancer cases. Most pediatric CNS tumor cases require a combination of imaging, histopathological, and molecular diagnoses to determine the tumor type/subtype. My project utilizes an emerging molecular diagnostic technology -- nanopore sequencing -- to quickly and accurately generate high depth whole genome DNA methylation profiles for a diverse cohort of 150 pediatric CNS tumors, and this data will be used to train and validate a pediatric-specific deep learning classifier for intraoperative use. As part of a clinical research collaboration, I tested the previously-developed machine learning classifier Sturgeon on high depth nanopore DNA methylation data for 8 matched pairs of standard patient tissue and tumor aspirate (16 total samples). For each sample, I demonstrated between 4-34x genomic coverage, classified the sample’s tumor type/subtype, validated key subtype-defining features, and generated a digital karyotype to visualize large tumor-specific copy number variation(s). Through this initial analysis, I was able to identify key shortcomings in the existing software, including inappropriate labeling for pediatric samples, insufficient adjustment for low tumor purity samples, and confident misclassification of rare/unique tumors. After making adjustments to the classifier's data processing, all 16 samples were classified correctly with high confidence. Together, I have used nanopore DNA methylation profiling to demonstrate fast and accurate classification of CNS tumor aspirate, and in the future, I will generate a similar machine learning classifier for pediatric-specific CNS tumor classification.
  1. First Name: Allison
    Middle Initial: A
    Last Name: Murray
    Organization: University of North Carolina Chapel Hill
  2. First Name: Breanna
    Middle Initial: E
    Last Name: Mann
    Degree(s): Ph.D.
    Organization: University of North Carolina Chapel Hill
  3. First Name: Andrew
    Middle Initial: B
    Last Name: Satterlee
    Degree(s): Ph.D.
    Organization: University of North Carolina Chapel Hill
  4. First Name: David
    Last Name: Kram
    Degree(s): M.D., MCR
    Organization: University of North Carolina Chapel Hill
  5. First Name: Jeremy
    Middle Initial: R
    Last Name: Wang
    Degree(s): Ph.D.
    Organization: University of North Carolina Chapel Hill
 Allison A Murray University of North Carolina Chapel Hill amurray1@unc.edu
37 Wed, 08/06/2025 - 12:34 Anonymous 10.208.24.36 Developing predictive and translational magnetic resonance imaging biomarkers of neuroinflammation, cognitive impairment, and survival outcomes for radiotherapy-induced brain injury Introduction: Radiotherapy-induced brain injury (RIBI) affects up to 90% of brain tumor survivors treated with radiotherapy. Thus, there is a great need for imaging strategies capable of detecting RIBI early on, for its effective management.

Objective: Here, we used multi-parametric magnetic resonance imaging (MRI) to identify translational and predictive biomarkers of RIBI in a preclinical mouse model.

Method: Mice were stereotactically irradiated with an X-ray beam at a dose of 80 Gy (1.7Gy/min), and longitudinally monitored using MRI, behavioral tests of learning and memory, positron emission tomography (PET), histology, and immunohistochemistry. The irradiated mice were studied in comparison to non-irradiated control mice.

Results: Contrast-enhanced T1-weighted MRI was best suited to detect the onset of injury, by detecting changes in the blood-brain barrier (BBB) permeability. Maximum BBB permeability was detected at one-month post-irradiation. This coincided with the detection of maximum neuroinflammation, using IBA1 and CD68 immunohistochemistry and 11C-CPPC and 11C-DPA PET radiotracers of neuroinflammation. This simultaneous maximum BBB permeability and neuroinflammation detection also coincided with the onset of transient cognitive impairment, detected using the fear-conditioning behavioral test of learning and memory. T2-weighted MRI was best suited to detect intermediate injury, while T2*-weighted MRI was best suited to detect late injury. This MRI biomarker of late injury preceded significant weight loss, severe cognitive impairment, and decreased survival outcomes in the irradiated mice compared to the non-irradiated mice.

Conclusion: We identified three translational and predictive MRI biomarkers of RIBI that could enable the better management of RIBI in brain tumor survivors.
  1. First Name: Maya
    Last Name: Teitz
    Degree(s): B.Sc.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  2. First Name: Saikat
    Last Name: Maiti
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  3. First Name: Santosh
    Last Name: Yadav
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  4. First Name: Esteban
    Last Name: Velarde
    Degree(s): B.Sc.
    Organization: Department of Radiation Oncology and Molecular Radiation Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21231
  5. First Name: Xiaoju
    Last Name: Yang
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  6. First Name: Shana
    Last Name: Lee
    Degree(s): B.Sc.
    Organization: Department of Molecular and Comparative Pathobiology, Johns Hopkins University School of Medicine, Baltimore, MD 21205
  7. First Name: Kristen
    Last Name: Lecksell
    Degree(s): B.Sc.
    Organization: Department of Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287
  8. First Name: Chantelle
    Last Name: Terrilion
    Degree(s): Ph.D.
    Organization: Department of Psychiatry and Behavioral Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21287.
  9. First Name: Adnan
    Last Name: Bibic
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287; F.M. Kirby Research Center for Functional Brain Imaging, Kennedy Krieger Institute, Baltimore, MD 21205
  10. First Name: Ethel
    Middle Initial: J.
    Last Name: Ngen
    Degree(s): Ph.D.
    Organization: Russell H. Morgan Department of Radiology and Radiological Science, Johns Hopkins University School of Medicine, Baltimore, MD 21287; Johns Hopkins Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD 21287
 Ethel J. Ngen, Ph.D. Johns Hopkins University School of Medicine engen1@jhmi.edu
36 Wed, 08/06/2025 - 11:51 Anonymous 10.208.24.36 Real-World Molecularly Targeted Treatment Registry (MaTTeR): a Pilot Study to Enrich CCDI Data Utilizing Directed EMR Extraction The incorporation of genomic profiling into the care of pediatric, adolescent and young adult (AYA) cancer patients has resulted in identification of targetable alterations and use of molecularly targeted therapy (MTT), necessitating collection and reporting of outcomes of real-world use of MTT. Using genomic data contributed to the CCDI and our institutional cohorts, we identified a cohort of patients who received MTT outside of clinical trials. We included patients with cancer seen at the Dana-Farber/Boston Children’s, who enrolled in a clinical sequencing/banking study and had next-generation sequencing (NGS) performed (OncoPanel, Rapid Heme and Fusion Panels). Sequencing results were used to identify patients with potentially targetable alterations in a list of 66 genes. Patients who received MTT were identified, and data including dosing, toxicity and response were extracted from the medical record. Between 2013 and 2024, 2163 patients had tumor profiling via targeted NGS. 35% (760/2163) had an actionable alteration detected for which MTT could have been administered. 114 patients received at least one regimen that included an MTT (72 patients with brain tumors, 28 with solid tumors, 14 with hematologic malignancies). Alterations in BRAF, NF1, ALK, FLT3, and PIK3CA led to the most MTT use. Of the 114 patients who received MTT, 79% (90/114) received at least one targeted therapy regimen outside of a clinical trial. This project will create a registry of MTT use within the CCDI that can be expanded to include additional patients and be a resource for MTT use for the pediatric and AYA cancer community.
  1. First Name: Suzanne
    Middle Initial: J
    Last Name: Forrest
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  2. First Name: Kee Kat (Aaron)
    Last Name: Yeo
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  3. First Name: Evelina
    Last Name: Ceca
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  4. First Name: Maeve
    Last Name: Smart
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  5. First Name: Olivia
    Last Name: Puopolo
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  6. First Name: Sabrina
    Last Name: Testa
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  7. First Name: Elizabeth
    Middle Initial: F
    Last Name: Cohen-Sacks
    Organization: Dana-Farber Cancer Institute
  8. First Name: Ledia
    Middle Initial: A
    Last Name: Gebremedhin
    Organization: Dan-Farber Cancer Institute
  9. First Name: Katherine
    Middle Initial: A
    Last Name: Janeway
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
  10. First Name: Yana
    Last Name: Pikman
    Degree(s): M.D.
    Organization: Dana-Farber/Boston Children's Cancer and Blood Disorder Center
 Suzanne J. Forrest Dana-Farber/Boston Children's Cancer and Blood Disorder Center Suzanne_forrest@dfci.harvard.edu
35 Mon, 08/04/2025 - 15:11 Anonymous 10.208.24.148 Analyzing the Impact of CCDI-COG Molecular Characterization Initiative (MCI) on Rare Tumors Background: The Childhood Cancer Data Initiative (CCDI) in collaboration with the Children’s Oncology Group (COG) offers paired germline and tissue sequencing for newly diagnosed pediatric rare tumors.
Methods: Within 6 months of diagnosis of a rare tumor, as defined by the Children’s Oncology Group, individuals 25 years old or younger are eligible for paired germline and tumor tissue exome enhanced sequencing of cancer-associated genes and targeted RNA fusion analysis.
Results: From 9/22/2022 through 06/30/2025, 697 individuals were enrolled. The most common diagnosis subgroups were thyroid carcinoma (n=168), neuroendocrine tumors (n=71), sex cord-stromal tumors (n=58), and other carcinomas (n=136). As of 03/31/2025, 448 paired samples were received (73.4%). Clinically actionable (Tier I/II) germline single nucleotide variants (SNV) and/or copy number variants (CNV) were identified in 108 (24.1%) of patients.The most prevalent germline alterations included SNVs in DICER1 (n=23, 5.1%), TP53 (n=10, 2.2%), RB1 (n=7,1.5%), CHEK2 (n=7, 1.5%), and VHL (n=6, 1.3%). In addition, 49.6% (n=222) of individuals demonstrated a Tier I/II SNV somatic variant and 51.8% (n=232) had a somatic CNV. Targeted RNA fusion analysis identified oncogenic fusions in 23.6% of tumors, most commonly associated with thyroid cancer or desmoplastic small round cell tumors.
Conclusions: The MCI has improved access to tumor genomic profiling for a wide range of rare tumors across COG centers. Germline cancer predisposition was identified in approximately a quarter of these individuals, highlighting the essential nature of this type of molecular profiling for genetic counseling and improving our understanding and treatment of rare tumors.
  1. First Name: Lauren
    Middle Initial: M
    Last Name: Vasta
    Degree(s): MD
    Organization: Walter Reed National Military Medical Center
  2. First Name: Jin
    Last Name: Piao
    Degree(s): PhD
    Organization: University of Southern California Keck School of Medicine
  3. First Name: Kenneth
    Middle Initial: S
    Last Name: Chen
    Degree(s): MD
    Organization: University of Texas Southwestern
  4. First Name: John
    Last Name: Hicks
    Degree(s): MD, PhD
    Organization: Baylor College of Medicine
  5. First Name: Erin
    Middle Initial: R
    Last Name: Rudzinski
    Degree(s): MD
    Organization: Indiana University
  6. First Name: Kareesma
    Last Name: Parbhoo
    Organization: Institute for Genomic Medicine at Nationwide Children's Hospital
  7. First Name: John
    Last Name: Shern
    Degree(s): MD, PhD
    Organization: National Cancer Institute
  8. First Name: Subhashini
    Last Name: Jagu
    Degree(s): PhD
    Organization: National Cancer Institute
  9. First Name: Gregory
    Last Name: Reaman
    Degree(s): MD
    Organization: National Cancer Institute
  10. First Name: Malcolm
    Last Name: Smith
    Degree(s): MD
    Organization: National Cancer Institute
  11. First Name: Catherine
    Last Name: Cottrell
    Degree(s): PhD
    Organization: Institute for Genomic Medicine at Nationwide Children's Hospital
  12. First Name: Douglas
    Middle Initial: S
    Last Name: Hawkins
    Degree(s): MD
    Organization: Seattle Children's Hospital and the University of Washington School of Medicine
  13. First Name: Kris Ann
    Middle Initial: P
    Last Name: Schultz
    Degree(s): MD
    Organization: Children's Minnesota
  14. First Name: Theodore
    Middle Initial: W
    Last Name: Laetsch
    Degree(s): MD
    Organization: Children's Hospital of Philadelphia
 Lauren M. Vasta Walter Reed National Military Medical Center lauren.vasta@gmail.com
34 Mon, 08/04/2025 - 14:12 Anonymous 10.208.28.82 The Open Single-cell Pediatric Cancer Atlas project: Collaborative analysis of pediatric tumor data The Open Single-cell Pediatric Cancer Atlas (OpenScPCA) project is an open, collaborative initiative to analyze publicly available data from the Single-cell Pediatric Cancer Atlas (ScPCA) Portal. The goal of OpenScPCA is to enhance the utility of single-cell pediatric cancer data and facilitate deeper insights into pediatric cancer biology.

The ScPCA Portal (https://scpca.alexslemonade.org/), developed and maintained by Alex’s Lemonade Stand Foundation (ALSF), is a data resource for single-cell and single-nuclei RNA sequencing data of pediatric tumors. The ScPCA Portal currently contains summarized gene expression data for over 700 samples from a diverse range of over 50 cancer types, all uniformly processed with an open-source workflow. All data on the portal is publicly available and ready for download in formats compatible with popular single-cell data analysis frameworks.

To coordinate further analysis of the ScPCA data, we launched the OpenScPCA project in April 2024. The project aims to engage a broad community of researchers analyzing genomic data from pediatric tumors, building off the previous success of the Open Pediatric Brain Tumor Atlas project (Shapiro et al. 2023). As part of the OpenScPCA project, ALSF offers contributors technical guidance, comprehensive documentation, and access to computational resources.

We invite researchers with expertise in pediatric cancer gene expression and single-cell RNA-seq analysis to join the OpenScPCA project. We hope those participating will discover new datasets to advance their research, gain experience with cutting-edge technologies, build their research portfolios, and join a supportive community. Get started at https://openscpca.readthedocs.io/en/latest/.
  1. First Name: Joshua
    Middle Initial: A
    Last Name: Shapiro
    Organization: Alex's Lemonade Stand Foundation
  2. First Name: Stephanie
    Middle Initial: J
    Last Name: Spielman
    Organization: Alex's Lemonade Stand Foundation
  3. First Name: Allegra
    Middle Initial: G
    Last Name: Hawkins
    Organization: Alex's Lemonade Stand Foundation
  4. First Name: Deepashree
    Last Name: Venkatesh Prasad
    Organization: Alex's Lemonade Stand Foundation
  5. First Name: Jennifer
    Last Name: O'Malley
    Organization: Alex's Lemonade Stand Foundation
  6. First Name: David
    Middle Initial: S
    Last Name: Mejia
    Organization: Alex's Lemonade Stand Foundation
  7. First Name: Jaclyn
    Middle Initial: N
    Last Name: Taroni
    Organization: Alex's Lemonade Stand Foundation
 Jaclyn N. Taroni Childhood Cancer Data Lab, Alex's Lemonade Stand Foundation jaclyn.taroni@ccdatalab.org
33 Thu, 07/17/2025 - 13:41 Anonymous 10.208.24.104 Aqueous Humor Liquid Biopsy Enables Molecular Diagnosis and Monitoring in Retinoblastoma Background: Retinoblastoma (RB), the most common intraocular malignancy in children, laid the foundation for cancer genetics through the discovery of the RB1 tumor suppressor gene and the "two-hit" hypothesis. However, precision oncology has largely bypassed RB due to the risks associated with intraocular tumor biopsy. To overcome this, we developed an aqueous humor (AH) liquid biopsy platform for genomic profiling using tumor-derived cell-free DNA (cfDNA).

Methods: We established LBSeq4Kids, a CLIA-certified laboratory-developed test combining low-pass whole genome sequencing (LP-WGS) for detecting somatic copy number alterations (CNAs) and a custom pediatric targeted sequencing panel (TSP) for single nucleotide variants and fusions. A total of 147 AH samples from 60 patients (75 eyes) with suspected intraocular malignancy were analyzed.

Results: In 41 RB patients, LP-WGS detected CNAs in 94% of diagnostic samples and 100% of recurrent cases, achieving an overall sensitivity of 97%. No CNAs or pathogenic variants were detected in 14 patients with benign mimickers, demonstrating 100% specificity. Serial testing showed CNA clearance in 61% of eyes after two treatment cycles, aligning with clinical remission. TSP identified RB1 mutations in 92% of germline variant cases and was valuable in CNA-negative tumors.

Conclusions: LBSeq4Kids enables safe, repeatable molecular profiling in RB, transforming it into a genomically accessible cancer. This approach offers precise diagnosis, risk assessment, treatment monitoring, and recurrence detection, marking a significant advance in pediatric precision oncology.
  1. First Name: Liya
    Last Name: Xu
    Degree(s): PhD
    Organization: Children's Hospital Los Angeles
  2. First Name: Laura
    Last Name: Kagami
    Degree(s): MD
    Organization: Children's Hospital Los Angeles
  3. First Name: Venkata
    Last Name: Yellapnatula
    Degree(s): PhD
    Organization: Children's Hospital Los Angeles
  4. First Name: Nerea
    Last Name: Goni
    Degree(s): Master
    Organization: University of Southern California
  5. First Name: Jaclyn
    Last Name: Biegel
    Degree(s): MD
    Organization: Children's Hospital Los Angeles
  6. First Name: Jesse
    Last Name: Berry
    Degree(s): MD
    Organization: Children's Hospital Los Angeles
 Liya Xu Children's Hospital Los Angeles liyaxu@usc.edu
31 Thu, 07/10/2025 - 05:54 Anonymous 10.208.24.72 The Need for Data Integration and Collaboration for Improved Pediatric Cancer Registry: Lessons Learned from the National Cancer Control Programme Integrating Children, Adolescents, and Young Adults (NCCP ICAYA) Background
Data integration remains a critical challenge in pediatric cancer care in Nigeria, where
fragmented information systems and limited collaboration hinder effective policymaking
and resource allocation. The WHO St. Jude NCCP ICAYA initiative highlighted the need for
coordinated data systems to support better outcomes for children, adolescents, and young
adults (CAYA) with cancer.

Aim
This study explores lessons from the NCCP ICAYA program and its influence on Nigeria’s
efforts to establish a comprehensive pediatric cancer registry, with a focus on ongoing
work in Nigeria

Methods
We conducted a qualitative review of sessions and stakeholder engagements from the
NCCP ICAYA program, focusing on themes of data integration, institutional collaboration,
and applicability to national registry development.

Results
The NCCP ICAYA initiative significantly deepened awareness of the importance of a unified
pediatric cancer registry. It underscored the value of cross-sector collaboration and real-
time data sharing across healthcare institutions. These insights have informed the ongoing
development of a national, population-based pediatric cancer registry capturing incidence,
treatment, and outcomes. Collaboration among the Federal Ministry of Health, Lagos
University Teaching Hospital, and NGOs such as The Dorcas Cancer Foundation has been
notably strengthened through the initiative.

Conclusion
The NCCP ICAYA program has played a pivotal role in advancing data integration and
stakeholder collaboration in Nigeria’s pediatric oncology landscape. Lessons from the
initiative have shaped the foundation for the Pediatric Cancer Registry and offer a scalable
model for national implementation to improve childhood cancer care outcomes in Nigeria.
  1. First Name: Korede
    Last Name: Akindele
    Organization: The Dorcas Cancer Foundation
 Korede Akindele The Dorcas Cancer Foundation korede@tdcf.ng