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Pediatric Brain Tumor Atlas, Neonatal Research Network, Grant Awards, Global Health

Published on June 9, 2023 in Cornerstone Blog · Last updated 2 months 2 weeks ago
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In the News

 

As the weather heats up on the East Coast, this week brings hot-off-the-press research news from Children's Hospital of Philadelphia. Read about a new collaborative, open framework resource platform for childhood pediatric brain cancer and the reestablishment of CHOP's Division of Neonatology as a Neonatal Research Network clinical trial site. Congratulations go out to the postdoctoral diversity fellow who received a Burroughs Wellcome Fund award, and the Hartwell Foundation recognized two CHOP researchers for their contributions to biomedical research. Finally, learn how CHOP experts work for global health equity through data science.

Open Pediatric Brain Tumor Atlas Provides a First-of-its-Kind Collaborative Resource Platform

The rare nature of pediatric brain tumors means researchers have limited access to tissue samples and patient-derived cell lines that may be useful in establishing a diagnosis and developing a treatment plan. To address this issue, CHOP scientists collaborated with Alex's Lemonade Stand Foundation Childhood Cancer Data Lab, the Children's Brain Tumor Network, the Pacific Pediatric Neuro-Oncology Consortium, and more than 20 other institutions to create the Open Pediatric Brain Tumor Atlas (OpenPBTA).

The first-of-its-kind platform offers an open, comprehensive genomic analysis of more than 1,000 tumors and 22 cell lines, which is crucial for researchers who are seeking new therapeutic targets or for finding new ways to translate research into clinical practice. With the help of thousands of samples analyzed by genomic sequencing, researchers used this platform to identify initial findings about genetic variants associated with poorer outcomes that could help guide future diagnostic and therapeutic advances. Cell Genomics published the details of the platform and those initial findings.

The researchers found that the loss of TP53, a tumor suppressor gene, is a significant marker for poor overall survival in fast-growing brain and spinal cord tumors called ependymomas and certain diffuse midline gliomas. Dysregulation of this gene also results in hypermutant high-grade gliomas.

"While there have been many proponents of an open-source model for scientific research, nothing like this existed for pediatric cancer," said Jo Lynne Rokita, PhD, supervisory bioinformatics scientist leading OpenPBTA at the Center for Data-Driven Discovery in Biomedicine and one of the study's corresponding authors. "We designed OpenPBTA so that anyone could access the data, contribute to its analysis, and/or use it in their own research."

Read more in this CHOP news release.

Division of Neonatology Again Named NRN Clinical Trial Center

Sara B. DeMauro, MD, MSCE
Sara DeMauro, MD, MSCE

The Division of Neonatology at CHOP and the University of Pennsylvania successfully competed to remain part of the Eunice Kennedy Shriver National Institute of Child Health and Human Development-funded Neonatal Research Network (NRN) for the next seven years. This is their third cycle as a network center location, with Sara DeMauro, MD, MSCE, attending neonatologist in the Division of Neonatology, as the lead follow-up investigator and alternate principal investigator for the CHOP-Penn NRN center.

As an NRN member, the Division of Neonatology is at the forefront of academic centers in clinical and epidemiological research in the United States and is a national leader in the development and conduct of multi- and single-center clinical trials centers. The division's research focuses on understanding the fundamental biology of preterm birth and its complications, testing new therapeutics in newborns through clinical trials, and learning about risk factors for poor outcomes in sick neonates through observational studies.

The NRN strives to address sepsis prevention, intraventricular hemorrhage, pulmonary hypertension, surfactant administration, and outcome and resource requirements for very low birth weight infants by maintaining high standards for staff at each clinical trial site and developing consistent study protocols across all center locations.

Lorianna Colon, PhD, Selected for Postdoctoral Diversity Enrichment Program

The Burroughs Wellcome Fund selected CHOP Diversity Fellow Lorianna Colon, PhD, for the 2023 Postdoctoral Diversity Enrichment Program. The program fosters diversity and inclusivity in scientific research by providing support to outstanding postdoctoral fellows from underrepresented backgrounds. The awardees' training and professional development will be guided by mentors committed to helping them advance in their biomedical or medical research.

Dr. Colon's research examines sex differences in behavior and neural development in brain health, injury, and disease. Neurodevelopmental and neuropsychiatric disorders have a distinct sex bias in their age of onset and prevalence. Adverse environmental factors such as stress and drug abuse also contribute to the final outcomes of neural development. Dr. Colon studies the ways in which these factors work together to determine possible psychiatric interventions and treatment, and even early diagnosis.

Her project, recognized by the Burroughs Wellcome Fund, "Sex Differences in Opioid Relapse: Targeting Dentate Gyrus to Prevent Relapse to Drug Taking," seeks to offer sex-specific novel interventions for the treatment of drug relapse. Even after long periods of abstinence, re-exposure to an opioid-linked context can provoke craving and relapse to opioid use. The goal of her research is to break the addiction cycle of drug relapse by manipulating key brain regions that contribute to relapsing when individuals with Opiate Use Disorder encounter environments that remind them of their opioid use.

"I am honored to be a recipient of this prestigious award. It is an investment in my professional development and advancement to research independence," Dr. Colon said. "It's a unique opportunity to gain access to an extraordinary cohort of esteemed scientists across the country and to connect with program alumni who have become successful independent scientists."

Two CHOP Investigators Receive Prestigious 2022 Hartwell Award

Christopher S. Thom
Christopher Thom, MD, PhD

Christopher Thom, MD, PhD, and Sarah Henrickson, MD, PhD, are among the 10 recipients of the 2022 Hartwell Individual Biomedical Research Awards. The Hartwell Foundation provide funding to individuals for early stage, innovative, and cutting-edge biomedical applied research that will potentially benefit children but whose projects have not yet qualified for funding from traditional sources.

Dr. Thom is a principal investigator in the Division of Neonatology at CHOP and assistant professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine. His research focuses on computational and cellular approaches to understand genetic determinants of hematopoiesis — the production of blood cells and platelets. He is recognized for his work using platelet proteomics to improve neonatal transfusion practices.

Photo of Sarah E. Henrickson
Sarah Henrickson, MD, PhD

Dr. Henrickson, leader of the Henrickson Lab at CHOP and assistant professor of Pediatrics at Penn, studies the impact of pediatric asthma and obesity on immune function, including response to vaccines. The award recognizes her work leveraging patterns of immune dysfunction to target primary immune regulatory disorders.

"The Hartwell Foundation's focus on improving children's health through mechanistic biomedical research is directly aligned with my lab's goals to improve our understanding and treatment of rare primary immune deficiencies," Dr. Henrickson said. "I am honored to join Dr. Thom and the other CHOP and Penn Investigators as members of the Hartwell Investigator community of basic and translational researchers."

See the Hartwell Foundation's announcement to learn more about the winners.

Global Partnerships Aim to Improve Health Information Systems

Sansanee Craig
Sansanee Craig, MD

CHOP's Global Health Informatics Program, part of the Department of Biomedical and Health Informatics, participated in collaborative workshops to support those who work in healthcare and data science, bringing leading voices from CHOP and the University of Pennsylvania together with scientists from the University of Botswana and the Open Medical Institute (OMI) MEX initiative to share their expertise.

In collaboration with CHOP, the University of Botswana's eHealth Research Unit hosted a workshop on data science for healthcare professionals that covered basic data science, medical informatics concepts, and Python for analyzing, reporting, and displaying healthcare data. Lecturers included experts from the Botswana-UPenn partnership: Audrey Masizana, PhD, senior lecturer for Computer Science, and Kagiso Ndlovu, eHealth Research Unit coordinator from the University of Botswana; Badisa Mosesane, computer science scholar and program manager of CHOP Botswana; Joy Payton, supervision of data education at CHOP; and Grey Faulkenberry, MD, MPH, co-chair of the Global Health Informatics Program.

Additionally, Sansanee Craig, MD, co-chair of CHOP Global Health Informatics, facilitated lectures and workshops in conjunction with OMI MEX, a joint initiative between the OMI and Alianza Médica para la Salud in Mexico City at the end of May. Bimal Desai, MD, MBI, FAAP, Robert Grundmeier, MD, and Eli Lourie, MD, MBI, FAAP, joined Dr. Craig in directing workshops focused on important aspects such as workforce development, digital health readiness assessments, ethical considerations in private security, clinical decision support, and an overview of electronic health records.

ICYMI

Catch up on our headlines from our May 26 In the News:

  • S. News & World Report Ranks No. 1 Pediatrics Program
  • CHOP Selected as Lead Study Site for Gene Therapy Clinical Trial for Rare Disease
  • Philadelphia County Medical Society Honors Scientist's Contributions to Healing
  • Melanoma Research Alliance Awards Young Investigator Grant to Leyuan Ma, PhD
  • American Thoracic Society Recognizes Ariel Williamson, PhD, DBSM, for Outstanding Achievements in Sleep Health
  • Novel Computational Tool Identifies More Targets for Cancer Immunotherapy

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