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.
Monthly Archive: December 2018
Leading the charge to develop new, targeted approaches to treat disease, Children’s Hospital of Philadelphia Research Institute investigators have uncovered more of the mechanistic underpinnings of the migratory T cell response, and CHOP announced the inaugural Patricia Brophy Endowed Chair in Neuroblastoma Research to advance innovation into less toxic treatment options for specific cancers. Out in the community, CHOP has teamed up with the Philadelphia Housing Development Corporation for the pilot program of its Healthier Together initiative, and researchers are learning how best to help survivors of violence after they are discharged from the hospital.
Our basic function as human beings is our ability to communicate, yet we often take for granted how a baby babbles, a child chatters, or a teen raps. A fragile interplay along the vocal tract — from lungs, through the larynx and the vocal cords, to the lips — allows us to speak, swallow, and breathe. When an airway insult or injury interferes with these passages and processes, experts at the Center for Pediatric Airway Disorders combine extraordinary clinical care and innovative translational research to give children the best possible outcomes.
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.
Obsessive compulsive symptoms (OCS), which include intrusive thoughts, urges, mental images, and repetitive behaviors, are common in community youth. But while OCS are often considered developmentally appropriate, researchers in the Lifespan Brain Institute (LiBi) at Children’s Hospital of Philadelphia and the University of Pennsylvania have found that some OCS patterns – specifically bad intrusive thoughts – have stronger associations with serious psychiatric conditions such as depression, psychosis, and suicidal ideation. Other types of OCS, such as those surrounding cleaning or contamination, were not associated with major psychiatric conditions.
The end of the year inevitably arrives with a handful of things to celebrate, from memorable moments to astounding achievements to milestones made. With less than three weeks left in 2018, our list of celebratory moments at Children’s Hospital of Philadelphia Research Institute continues to grow, as this edition of our biweekly research news roundup shows. Read on to learn about two gene therapies pioneered at CHOP and the University of Pennsylvania that recently reached important milestones, a novel discovery from our scientists that could help to improve cancer immunotherapies, the 10-year anniversary of our Center for Autism Research, and more.
Clinician-researchers in the Pediatric Research Consortium (PeRC) at Children’s Hospital of Philadelphia didn’t have to look far to find specialists to provide novel insights into their research projects: They are the families who are the experts on their children visiting CHOP’s primary care practices.
Four parents from different backgrounds, levels of education, and geographic areas partnered with PeRC in 2013 as a Parent Advisory Board to provide meaningful input to guide implementation of PeRC’s portfolio of research studies. PeRC is a 31-practice, two-state research network with 205 physicians and 41 nurse practitioners who have published about 200 peer-reviewed accepted published manuscripts over the last decade or so.
When young investigators bring big ideas to Kristy Arbogast, PhD, this year’s winner of the Award for Excellence in Mentoring Research Trainees, she is eager to help them figure out their research vision from the ground up. That’s because Dr. Arbogast knows firsthand the value early career mentors have in shaping how investigators learn to interrogate a scientific problem, analyze the data, and use the results to improve the world.
The voice of Kai Tan, PhD, rises and quickens when he considers the potential of single cell technology to zero in on pathogenesis of cancer and other diseases. He points to the attention this course of research is receiving via funding from organizations such as the National Cancer Institute (NCI) Moonshot Initiative and the Chan Zuckerberg Initiative’s Human Cell Atlas project. In fact, Dr. Tan is himself is a valued contributor to the single cell revolution, and his work will continue with a recent NCI grant for the development of a pediatric tumor cell atlas.
The Center for Autism Research at Children’s Hospital of Philadelphia is celebrating a decade of conducting autism research to understand the causes of autism spectrum disorder, develop effective therapies, and train the next generation of master clinicians and scientists in state-of-the-science best practices for autism screening, diagnosis, and treatment.