As the new year approaches, we look back with gratitude for yet another year packed with scientific breakthroughs in children’s health. Continuing a remarkable run for personalized gene therapies, 2018 marked the European Commission’s approval of two gene therapies pioneered at Children’s Hospital of Philadelphia and the University of Pennsylvania, as patients in the European Union (EU) may now be treated with chimeric antigen receptor (CAR)-T cell therapy for aggressive forms of leukemia, and the very first gene therapy developed for inherited blindness.
Tag Archive: Valder Arruda
Thrombosis — the abnormal, life-threatening blood clots that form in the artery or vein — does not discriminate. Young. Old. Rich. Poor. Gender. Race. Ethnicity. We’re all in this together. We’re all susceptible. Some more so than others.
Up to 900,000 people in the U.S. are affected by blood clots each year; 100,000 will die, which is greater than the total number of people who lose their lives each year to AIDS, breast cancer, and motor vehicle crashes combined.
Editor’s Note: Jacqueline Hunter, PhD, a postdoctoral fellow in the Wolfe Laboratory, wrote this article as part of the Advanced Career Exploration (ACE) Fellowship at Children’s Hospital of Philadelphia Research Institute. The ACE program gives fellows the opportunity to pursue projects beyond their main research focus. We’re especially excited to share Dr. Hunter’s work during National Postdoc Appreciation Week.
Normally, the body responds to a bleeding event by forming a clot, which is a complicated process involving multiple proteins in an elegantly orchestrated cascade. When specific proteins of this cascade are absent, one of several debilitating disorders can occur that result in recurrent spontaneous bleeding into the joints and muscles. Researchers at Children’s Hospital of Philadelphia are using clever maneuvering to figure out new therapeutic options for patients with hemophilia A and B.
Hematologists have grappled for many years to understand the basic mechanisms underlying why some young patients with the inherited bleeding disorder hemophilia A develop inhibitory antibodies, also known as factor VIII (FVIII) inhibitors, to protein replacement therapy. The presence of FVIII inhibitors is a serious obstacle that makes it difficult and extremely expensive to treat their hemophilia, leads to complications such as joint disease, and increases mortality.
Such a major challenge requires major brain power. So the National Institutes of Health called on the scientific community to reframe the question: Why is FVIII so immunogenic?
From the discovery of stem cells that multiply after a lung injury, to new data that advances how we think about (and treat) childhood cancer, our first roundup of March is packed with discovery. Read on to learn how our researchers stay at the forefront of pediatric science with a new study that expands what scientists know about the body’s extraordinary ability to regenerate, a pan-cancer project that distinguishes how cancer develops in children versus adults, and a handful of updates on what our investigators have in store for the near future.
Buckle your seatbelts because this has been a busy week for research news at Children’s Hospital of Philadelphia.