PAS 2019, NFL Helmet Safety, American Society of Gene and Cell Therapy, Bypass Prophylaxis Hemophilia, Lung Disease In Utero

Whether they study helmets on the football field or hemophilia in a lab, our scientists at Children’s Hospital of Philadelphia Research Institute are always on the cutting-edge of their respective fields, as the latest roundup of research news shows. This week, read about what to expect at next week’s Pediatric Academic Societies Meeting, discover how a CHOP engineer is helping to make the NFL safer, and learn about new results from our Center for Fetal Research about treating lung diseases in utero.

CHOP Experts Convene for Pediatric Academic Societies Meeting

Researchers and clinicians from diverse disciplines at CHOP will make their way to Baltimore next week for the 2019 Pediatric Academic Societies Meeting (PAS), held from April 24 to May 1. CHOP has an established history of presenting novel research on a multitude of topics at the international conference, which brings together thousands of pediatricians and healthcare providers to discuss how we can improve the health of children worldwide.

This year at PAS, Barbara Schmidt, MD, attending neonatologist at CHOP and director of Neonatology Clinical Research at Penn Medicine, will receive the Society for Pediatric Research (SPR) Douglas K. Richardson Award for Perinatal Research. The award was established to honor Dr. Richardson’s contributions to child health services and perinatal and pediatric research.

Meanwhile, this year’s platforms by CHOP researchers cover topics in everything from neonatology to public health to autism spectrum disorder and beyond. Some of the presentations that attendees can look forward to include:

• “Parent eReferral to Tobacco Quitline from Pediatric Primary Care: A Pragmatic Randomized Trial” presented April 27 by Brian Jenssen, Naveen Muthu, Mary Kate Kelly, Hilary Baca, Justine Shults, Robert Grundmeier, and Alexander Fiks.
• “Maternal Anxiety During Pregnancy and Infant Cord Blood DNA Methylation of the Glucocorticoid Receptor Promoter” presented April 27 by Heather Burris, Rachel Ledyard, Allyson Redhunt, Kasey Brennan, Andrea Baccarelli, and Michele Hacker.
• “Parent Concern Before Screening with the M-CHAT” presented April 28 by Kristin Fleming, Judith Miller, Whitney Guthrie, Katie Wallis, and Amanda Bennett.
• “Influence of Primary Care Parent-Targeted Interventions on Parent-Teen Communication about Sexual and Alcohol Behaviors: A Randomized Controlled Trial” presented April 28 by Carol Ford, Jessica Mirman, Felipe Garcia-Espana, Megan Fisher Thiel, Elizabeth Friedrich, and Elyse Salek.
• “GBS Intrapartum Antibiotic Prophylaxis is Associated With Increased Childhood Weight Gain” presented April 28 by Sagori Mukhopadhyay, Corinna Koebnick, Stephanie Schrag, Matthew Bryan, Jeffrey Gerber, Miren Dhudasia, Robert Grundmeier, William Quarshie, Darios Getahun, Margo Sidell, Emily Rozema, Sara Tartof, Anny Xiang, Andrea Sharma, Michael Spiller, and Karen Puopolo.

Kristy Arbogast Helmet Safety Research Featured by ESPN, WaPo

Research into helmet safety by our own Kristy Arbogast, PhD, who is an engineering consultant for the NFL Players Association (NFLPA), made headlines this week as the NFL and the NFLPA announced results from their 2019 Helmet Laboratory testing. The results included the addition of 11 helmets to the organizations’ joint list of approved equipment for 2019, according to ESPN.

Though the helmet testing conducted by the NFL and NFLPA is meant to maximize protection for adult professional athletes specifically, the work of Dr. Arbogast, who is co-scientific director of the Center for Injury Research and Prevention (CIRP) at CHOP, will be a “win for children” too, as the research may lead to new technologies for helmet design and protective equipment in youth sports.

“What we’ll learn at the pro level — thanks to all of the NFL’s resources — will trickle down to kids,” said Dr. Arbogast in a CHOP Breakthrough Report story. “The advances in material science and design will create a path to improved protective equipment for kids.”

Dr. Arbogast told ESPN in regards to the latest helmet results: “One of the most exciting things is seeing how our years of testing protocol is really driving innovation. It’s phenomenal that we are driving innovation, and we are driving design to release products that perform better on the field.”

Coverage from the Washington Post, USA Today, the The Seattle Times and more also quoted Dr. Arbogast. Learn more about Dr. Arbogast’s work with the NFLPA on Bench to Bedside.

Beverly Davidson Elected VP, Board of Directors of American Society of Gene and Cell Therapy

A warm congratulations goes out to Beverly Davidson, PhD, Chief Scientific Strategy Officer at CHOP, for her recent election as vice president of the board of directors for the American Society of Gene and Cell Therapy (ASGCT). As the largest association of individuals involved in gene and cell therapy research, ASGCT’s mission is to advance knowledge and awareness leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease.

“As a member of the Society for more than 30 years, I have seen enormous growth in the field of genetic and cellular therapies, as well as the incredible potential that lies ahead,” said Dr. Davidson in a press release. “I am honored to help guide decisions for an organization I care deeply about during this exciting time.”

Dr. Davidson’s research in the field of genetic and cellular therapies has focused on inherited genetic diseases, and some of her breakthroughs include the development of novel methods to deliver therapeutic genes to the central nervous system. As director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP, Dr. Davidson has also overseen the opening of the Clinical Vector Core at CHOP, where clinical-grade viral vectors will be produced to deliver cell and gene therapy for a variety of difficult-to-treat diseases.

Read more about Dr. Davidson’s election in the press release, and learn about the new Clinical Vector Core on Cornerstone.

CHOP Study Finds Gene-based Clotting Factor Prevents Bleeding in Animals With Hemophilia

A treatment currently used on an urgent basis to control bleeding in patients with hemophilia may hold promise as a preventive treatment as well, a recent paper from our hematology researchers suggests. The study appeared in the journal Blood Advances and describes how the team’s preclinical work with an animal model offers the first target levels for bypass agents to act prophylactically by preventing bleeding. The findings set the stage for a potential new therapy for patients with hemophilia who develop antibodies to the standard maintenance treatment and then require bypass therapy.

“Patients who develop antibodies to the coagulation factors usually prescribed for hemophilia have a complicated treatment,” said study leader Paris Margaritis, DPhil, a hematology researcher in the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at CHOP. “A different factor, called coagulation factor VIIa, restores blood clotting when given after a bleed occurs, but we don’t know the target level of circulating factor VIIa that would prevent bleeds before they start. Our new preclinical results are the first to show target levels that could act prophylactically.”

Learn more in the press release.

CHOP Researchers Treat Lethal Lung Disease In Utero via CRISPR Gene Editing

A team of scientists from CHOP and Penn Medicine have successfully treated a lethal lung disease using CRISPR gene editing, as described in a new Science Translational Medicine paper. The proof-of-concept study working with animal models illustrates how in utero editing could be a novel approach for treating lung diseases before birth. Specifically, the team hopes to address congenital diseases that are characterized by respiratory failure at birth, such as cystic fibrosis and surfactant protein deficiency.

“The developing fetus has many innate properties that make it an attractive recipient for therapeutic gene editing,” said William Peranteau, MD, the study’s co-leader and an investigator at CHOP’s Center for Fetal Research as well as a pediatric and fetal surgeon in our Center for Fetal Diagnosis and Treatment in a press release. “Furthermore, the ability to cure or mitigate a disease via gene editing in mid- to late gestation before birth and the onset of irreversible pathology is very exciting. This is particularly true for diseases that affect the lungs, whose function becomes dramatically more important at the time of birth.”

This novel research already has made headlines in the media, with coverage from STAT and Wired.

Read more about this breakthrough work in the press release.

ICYMI

Catch up on our headlines from our April 5 In the News:

• Risk Genes for Osteoporosis May Lead to Future Treatments
• Patient Safety Not Impeded by Residents’ Longer Work Shifts
• CHOP Researchers Look for New Ways to Detect Pediatric Spinal Deformity
• Standard Treatment Lung Inflation Prevails for Extremely Preterm Infants
• Pediatric Cell Atlas to Provide Window Into Child Health and Disease

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