World-renowned fetal surgery pioneer N. Scott Adzick, MD, MMM, received the John Scott Award, one of America’s oldest and most prestigious science awards, dedicated to the memory of Benjamin Franklin. Adzick, CHOP’s surgeon-in-chief and chief of the Department of General, Thoracic and Fetal Surgery, was recognized for his contributions to fetal surgery—performing highly complex procedures on babies while still in the womb, to correct debilitating or life-threatening birth defects.
An innovator in fetal medicine, Adzick has dedicated his career to the pursuit of groundbreaking prenatal treatment for birth defects. He joined CHOP in 1995 and founded the Center for Fetal Diagnosis and Treatment, which is now the largest and most comprehensive fetal program in the world. He also has a special interest and expertise in surgery for complex pediatric endocrine conditions such as hyperinsulinism and pediatric thyroid disorders.
Kristy Arbogast, PhD, director of engineering at CHOP’s Center for Injury Research and Prevention, was one of the experts tapped to author the recently released Institute of Medicine and National Research Council extensive report on sports-related concussions in children and teens. The report found helmets do not prevent concussions in youth athletes and that, despite increased awareness of concussions, there remains a culture “that resists both the self-reporting of concussions and compliance with appropriate concussion management plans.”
Arbogast says that while helmets “have been proven effective at preventing skull fractures and more serious traumatic brain injuries and should continue to be used in competitive and recreational sports … the Committee found that research was needed to better understand the biomechanics of how pediatric concussions occur before any protective device can be scientifically proven to prevent them.”
Because of this, there must be a sea change in the way athletes and coaches approach concussions, she says. “There must be a shift in the culture of athletics—among parents, coaches, school personnel, and the youth athletes themselves—to treat concussion as an injury that requires serious attention, even if it means missing ‘the big game’ or an entire season of play.”
To help families more easily understand concussion, CHOP has created a series of infographics and video FAQs on a variety of concussion topics as part of its Mind Matters initiative. Go to www. chop.edu/concussion and click on “Concussion Educational Tools,” to find resources you can download to share with families or even print as posters for your office.
Arbogast is one of the course directors of CHOP’s annual CME training on diagnosing and managing youth concussion. It is scheduled for April 12, 2014.
The Healthcare Accreditation Colloquium announced in September that CHOP became the first Accredited Pediatric Heart Failure Institute in Pennsylvania and third in the nation following nearly a year of work culminating in an in-depth onsite review. The reviewers assessed and complimented CHOP in four major domains: community, hospital, clinician and science.
Within the Community Domain, CHOP was found to have an exceptional program for genetic screening of entire families going beyond focusing only on one child. In the Hospital Domain, CHOP is a dominant provider of advanced heart care including VADs, artificial heart, and heart transplantation services. Its efforts are supported by participation in many research areas within the Science Domain.
“CHOP is an excellent example of constancy of purpose seen even in their improvement efforts that always put the child and the family first, reflecting holistic care across the entire continuum,” says David Wayne, Temple University adjunct faculty, who was the lead reviewer.
CHOP’s commitment to the development of gene therapies took a big step in October when it made a $50 million capital commitment to launch Spark Therapeutics, a new, fully integrated company formed to develop, advance, and commercialize gene-based medicines.
Spark will assume control from CHOP over two gene therapy clinical trials: a Phase 3 study for inherited blindness and a Phase 1/2 study for hemophilia B. Both of these trials build on a decade of work from CHOP’s Center for Cellular and Molecular Therapeutics (CCMT) under the direction of Katherine A. High, MD. The company is also advancing toward the clinic with gene therapy programs to address neurodegenerative diseases and additional hematologic disorders and other forms of inherited blindness.
High will serve as scientific co-founder and adviser to Spark. J. Fraser Wright, PhD, who has directed the CCMT’s vector manufacturing since its establishment, is scientific co-founder and chief technology adviser at Spark.
“Gene-based medicines are among the most complex therapeutics ever developed,” says High. “We at CCMT have persevered through more than a decade of scientific and clinical development and are now closer than ever to realizing the ambitious vision of one-time, potentially curative therapies to address serious genetic conditions. The team at Spark has incredible goals for the treatment of diseases including hemophilia B and inherited blindness, and we look forward to working with them to deliver groundbreaking new treatments to patients in need.”
To learn more, visit www.sparktx.com.
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