Tag Archive: Gene therapy

by Nancy McCann

Editor’s Note: This occasional blog series features stories of CHOP research heroes who have participated in clinical research studies. Without the generosity and dedication of families, patients, and members of the public who take the time to be a part of research, many trials would not succeed.

The alarm rings annoyingly loud at 6:30 a.m. Megan Payne knows she must rise with that blaring beep, or else the day goes haywire. With five young children to monitor, mornings are a blur of making breakfasts, packing lunches, brushing hair, and searching for lost homework before four kids leave the house at different times to board different buses to attend different schools. Only her youngest, 3-year-old Jaxson, remains behind. Oh, and Megan works three jobs and is a college student studying Environmental Science. Whew.

Bundled up in winter clothes from head to toe, 7-year-old Eric rolls a snowball around the backyard with his older brother, building it into a sizeable snowman. Mom, watching from her perch at the kitchen window, can hear her boys laughing — until — Eric doubles over in acute chest pain, crying out for her. Rushing to his side, she wonders how many more of these excruciating episodes and trips to the hospital Eric can endure.

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.

For more than 20 years, researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania have been at the forefront of taking a system perfected by nature — a virus — and transforming it into breakthrough gene therapies for rare single-gene diseases. CHOP was the first pediatric research institution to develop chimeric antigen receptor T cell (CAR-T) therapy for acute lymphoblastic leukemia. In this approach, viral vectors are used to modify a patient’s own T cells, training them to track down and eliminate the circulating cancer cells.

Fall weather and football season have returned to us here at Children’s Hospital of Philadelphia Research Institute, bringing with them a fresh batch of research headlines. In this edition of our biweekly news roundup, catch up on the latest announcements for the second annual Eagles Autism Challenge, learn about new insights into the role mitochondrial DNA plays in heart disease progression, and stay updated on how CHOP helps to drive medical innovation and entrepreneurship forward in the Philadelphia community and beyond.

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.

Along with the start of school and settling into new routines, September marks Childhood Cancer Awareness Month: a special time to support cancer research and care for patients and families impacted by the disease. Fittingly, this week’s roundup of research news highlights the remarkable impact our oncologists have made for children with cancer around the world. And on top of that, we highlight new findings that challenge the traditional notion of the teen years as a reckless time of risky behavior.

Microbes, monitors, and miraculous medicine converge in this week’s roundup of research-related headlines at Children’s Hospital of Philadelphia. Keep reading to learn more about new concerns for commercially available baby monitors reported by our researchers, one family’s inspirational story of resilience and recovery after their baby daughter battled a brain tumor, and exciting upcoming events in the CHOP community, including the fifth annual symposium for microbiome research.

After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!