Tag Archive: clinical trials
With the release of over 200 genomic tumor models spanning 25 different types of childhood cancer, researchers may now have the ability to skip lengthy preclinical work in their development of novel treatments. With funding from Alex’s Lemonade Stand Foundation (ALSF), the Pediatric Preclinical Testing Consortium (PPTC) announced their data sets will now be made available to any qualified academic petitioner — a move that John Maris, MD, oncologist at Children’s Hospital of Philadelphia Cancer Center and principal investigator of the PPTC’s CHOP site, believes is the first of its kind.
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.
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.
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.
It’s still a month before teachers and students are officially back to school, but here at Children’s Hospital of Philadelphia Research Institute, you can learn something new every day. In this edition of our biweekly news roundup, discover the latest findings from our ophthalmologists on how clinicians should choose to screen premature babies for a potentially blinding eye disorder, find out how CRISPR-based technology allowed scientists to reveal insights into sickle cell disease, and prepare for an educational and exciting speech from the recently announced keynote speaker at PolicyLab’s upcoming 10th Anniversary Forum
Stephen P. Hunger, MD, has received numerous honors throughout his decorated career. However, winning the George R. Buchanan Lectureship Award from the American Society of Pediatric Hematology/Oncology (ASPHO) had particularly special meaning to the chief of the Division of Oncology and director of the Center for Childhood Cancer Research at Children’s Hospital of Philadelphia.
Recognized internationally in the field of pediatric leukemia clinical care and research, Dr. George R. Buchanan— past president of ASPHO and a renowned pediatric hematology physician-researcher — was very supportive of Dr. Hunger early in his career.
Summer at Children’s Hospital of Philadelphia is definitely off to a sweet start: We kick off this week’s research roundup with a new way of thinking about honey (thanks to novel findings from our ear, nose, and throat specialists) and congratulate the Cancer Center at CHOP on yet another successful day of serving up lemonade to support the Alex’s Lemonade Stand Foundation (ALSF). On top of that, we cover research updates from our Mitochondrial Medicine Frontier Program and the Center for Autism Research (CAR), and share a special new award for Stephan Grupp, MD, PhD, director of the Cancer Immunotherapy Program.
Clinical research coordinators are the heart and soul of our research breakthroughs, as many of our investigators and staff at Children’s Hospital of Philadelphia know. Whether they’re ensuring that a study falls within regulatory protocols, explaining the science behind a health condition to a family, or simply spending time with patient participants to put them at ease, coordinators bridge the gap between an idea and its execution, as well as between scientists and the patients whose outcomes they hope to improve.
Nineteen-year-old Ben Hartranft remembers the first research study he participated in at the Center for Autism Research (CAR) nearly eight years ago. Though he was just 12, he didn’t feel nervous or scared about the functional magnetic resonance imaging machine that would capture images of his brain. Instead, Ben’s mom recalls him joking that the researchers could duct tape his legs to the chair to help him keep still (which, of course, wasn’t necessary).
“I just stayed still, and it was very fun,” Ben said.