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Top Pediatrics Program for Medical Schools, First-of-Its-Kind Gene Therapy Trial, Novel Computational Tool

Published on May 26, 2023 in Cornerstone Blog · Last updated 6 months 1 week ago


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In the News


In this week's news roundup, we're celebrating the numerous awards and achievements of our researchers and physicians. The Perelman School of Medicine at the University of Pennsylvania and its Department of Pediatrics at Children's Hospital of Philadelphia ranked No. 1 in the United States for medical schools; a public-private partnership selected CHOP to lead a first-of-its-kind gene therapy clinical trial for multiple sulfatase deficiency; researchers received recognition for their contributions to surgical healing and sleep health; and scientists developed a novel tool to identify new cancer immunotherapy targets.

U.S. News & World Report Ranks No. 1 Pediatrics Program

U.S. News & World Report released its "Best Medical School" rankings for the 2023-2024 year, and the Perelman School of Medicine at the University of Pennsylvania's Department of Pediatrics located at CHOP is No. 1 in "Best Pediatrics Programs."

The ranking evaluations consider an institution's faculty resources and academic achievements of entering students. New this year, U.S. News refined its data down into the "Best Medical Schools for Research," calculated from the number of faculty currently involved in research and the number of grants received from the National Institutes of Health, and the "Best Medical Schools for Primary Care," determined by the number of graduates practicing in primary care specialties or residencies.

The Department of Pediatrics at CHOP and the Perelman School of Medicine at the University of Pennsylvania strive to lead the world in advancing healthcare for children and adolescents. In collaboration with CHOP Research Institute, they offer a supportive environment for clinical and academic work.

Visit the U.S. News & World Report to learn more about this year's rankings.

CHOP Selected as Lead Study Site for Gene Therapy Clinical Trial for Rare Disease

Rebecca C. Ahrens-Nicklas, MD, PhD
Rebecca Ahrens-Nicklas, MD, PhD

The Foundation for the National Institutes of Health (FNIH) Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) selected CHOP as the lead study site for clinical trials involving multiple sulfatase deficiency, a neurologic genetic disorder that affects the SUMF1 gene.

Leading the research is Principal Investigator Rebecca Ahrens-Nicklas, MD, PhD, research scientist in the Division of Genetics, whose work uses gene discovery to understand why inherited biochemical disorders often result in severe, untreatable neurologic symptoms, and Laura Adang, MD, PhD, attending physician in the Division of Neurology, who specializes in the care of neuro-inflammatory diseases.

"This is an unprecedented opportunity to meet the needs of families affected by rare diseases," Dr. Adang said. "We are honored to represent our leukodystrophy and lysosomal storage disorder families who have worked so hard to reach this incredible milestone. The impact of these rare diseases on individuals and their families cannot be overstated. The opportunity to represent our families and offer them hope by collaborating on innovative solutions is an incredible honor."

AMP BGTC is a public-private partnership between the NIH, U.S. Food and Drug Administration, biopharmaceutical and life science companies, and nonprofit and other organizations to encourage the development and delivery of personalized gene therapies that could treat individuals affected by rare genetic diseases.

Learn more about the Bespoke Gene Therapy Consortium.

Philadelphia County Medical Society Honors Scientist's Contributions to Healing

Holly Hedrick, MD, attending pediatric and fetal surgeon in the Division of Pediatric General, Thoracic and Fetal Surgery at CHOP, received the Strittmatter Award, one of The Philadelphia County Medical Society's (PCMS) most prestigious honors, bestowed upon a PCMS physician who has made valuable contributions to surgical and medical healing practices.

"I am honored to receive this award from the Philadelphia County Medical Society and am so grateful to the patients and families who have put their trust in me over the years to improve the trajectory of their lives," said Dr. Hedrick, who also is a professor of Surgery at the University of Pennsylvania and an investigator in the Center for Fetal Research at CHOP. "I am lucky to be supported by a brilliant team at CHOP, and together we will continue working to improve the lives of children in Philadelphia and around the world."

Dr. Hedrick's research focuses on the surgical management of birth defects, such as congenital diaphragmatic hernia (CDH), a condition in which a hole in the diaphragm allows abdominal organs to migrate into the chest, restricting lung development. Dr. Hedrick's Congenital Diaphragmatic Hernia program received Frontier status in 2020 to work toward the elimination of CDH-related mortality and morbidity through new research and the development of novel devices. She is also the co-director of the Neonatal Surgical Team, director of the Pulmonary Hypoplasia Program, and holds the Louise Schnaufer Endowed Chair in Pediatric Surgery.

The Strittmatter Award is named after Isidore P. Strittmatter, MD, the 63rd president of PCMS, who established the award in 1923. Dr. Hedrick will receive her award June 3, during PCMS' 162nd President's Installation and Annual Awards Night Presentations.

Melanoma Research Alliance Awards Young Investigator Grant to Leyuan Ma, PhD

Leyuan Ma
Leyuan Ma, PhD

Leyuan Ma, PhD, assistant professor of Pathology and Medicine at CHOP, received one of 11 Young Investigator Awards from the Melanoma Research Alliance (MRA) in honor of melanoma awareness month in May. Dr. Ma's current research explores the development of biomaterial-based synthetic vaccines to enhance adoptive T-cell therapy for long-term melanoma control. The Ma Laboratory for Immune Engineering focuses on dissecting immune cell crosstalk in normal and pathological conditions using genetic and engineering tools, then leveraging these crosstalk mechanisms to develop novel precision immunotherapies.

"This award not only provides a timely support for our early-stage research in designing a novel cell therapy to treat melanoma, it also invites us to an exceptional melanoma research community where we can form collaborations and establish new research directions," Dr. Ma said. "I hope to build on the findings and collaborations resulting from this award to advance my career in melanoma research and develop safe and potent immunotherapies."

The Young Investigator Awards are part of a $6.3 million commitment by the MRA to support 24 scientists at institutions across the United States and Europe.

Learn more about the 2023 MRA grant awards in this news release.

American Thoracic Society Recognizes Ariel Williamson, PhD, DBSM, for Outstanding Achievements in Sleep Health

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Ariel Williamson, PhD

The American Thoracic Society (ATS) awarded Ariel Williamson, PhD, DBSM, a psychologist in the Department of Child and Adolescent Psychiatry and Behavioral Services at CHOP with the 2023 Carole L. Marcus Outstanding Achievement Award. This award recognizes the career achievements in pediatric sleep and respiratory neurobiology of new investigators who are not yet fully established but beyond formal training.

"As a former mentee of Carole's, I am incredibly honored to receive this award," Dr. Williamson said. "Carole truly valued and supported interdisciplinary pediatric sleep research and clinical care, recognizing the importance of integrating medical and behavioral perspectives. My interdisciplinary research, which aims to understand and address pediatric sleep health disparities, is a direct result of Carole's mentorship, making this award both personally and professionally meaningful."

Dr. Williamson's current research leverages pediatric sleep as a modifiable factor that can promote positive development and prevent child physical and behavioral health problems. One of her recent studies is a direct extension of the work she initiated with Dr. Marcus, in which researchers studied the co-occurrence of insufficient sleep and sleep-disordered breathing in children through the lens of racial disparity. Dr. Williamson strives to understand how socio-ecological factors found within families, environments, and cultures may contribute to sleep problems and sleep health inequities.

Learn more about the award on the American Thoracic Society's website.

Novel Computational Tool Identifies More Targets for Cancer Immunotherapy

Yi Xing
Yi Xing, PhD

Researchers at CHOP, including Yi Xing, PhD, director of the Center for Computational and Genomic Medicine and executive director of the Department of Biomedical and Health Informatics, and the University of California, Los Angeles, developed a tool that expands access to possible cancer immunotherapy targets. Their findings appeared in Proceedings of the National Academy of Sciences.

The computational tool, called "Isoform peptides from RNA splicing for Immunotherapy target Screening" (IRIS), can discover tumor antigens derived from alternative splicing (AS) of RNA, a largely unexplored source of potential immunotherapy targets.

Researchers used RNA sequencing-based transcriptomics data and mass spectrometry-based proteomics data to show that hundreds of IRIS-predicted T cell receptor-engineered T cell (TCR-T) targets are presented by human leukocyte antigen (HLA) molecules, the part of the human immune system that presents antigens to T cells. The researchers applied IRIS to RNA sequencing data from neuroendocrine prostate cancer, a metastatic and highly lethal disease known to involve shifts in RNA splicing. They were able to predict 1,651 peptides as potential TCR targets, then confirm the immunogenicity and T cell recognition of AS-derived T cell receptor targets.

"This proof-of-concept study demonstrates that alternatively spliced RNA transcripts are viable targets for cancer immunotherapy and provides a big data and multiomics-powered computational platform for finding these targets," said Dr. Xing, co-senior author. We are applying IRIS for target discovery across a wide range of pediatric and adult cancers. We are also developing a next-generation IRIS platform that harnesses newer transcriptomics technologies, such as long read and single cell analysis."

Read more about this study in the CHOP news release.


Catch up on our headlines from our May 12 In the News:

  • Researchers From CHOP and Princeton Discover Novel Genetic Disorder
  • Peanut Patch Demonstrates Effective Results in New Study
  • Global Health Experts Highlight Global Neonatal Healthcare
  • Renowned Pediatric Urologic Oncologist Appointed Chief of Urology Division
  • Pediatric Medical Device Consortium Awards Seed Grants
  • Cell and Gene Therapy Collaborative Announces Grant Recipients

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