In this week’s news roundup, there’s a lot to be proud of as our researchers make impactful discoveries, such as new-found variability in a mitochondrial disease-causing gene and encouraging findings about an antibody-drug conjugate that targets a surface protein expressed in childhood neuroblastomas, effectively killing cancer cells. Wanjiku Njoroge, MD, and colleagues followed mothers of very preterm infants to determine stress in the NICU and its effects five years later, and Allison Curry, PhD, MPH, is changing perceptions about visually impaired drivers.
Tag Archive: Cancer Center
By Jillian Rose Lim
Yael Mossé, MD, remembers meeting Patricia “Pat” Brophy, beloved nurse practitioner at Children’s Hospital of Philadelphia, nearly 23 years ago. Dr. Mossé was completing her pediatric residency, while Brophy was already a seasoned nurse caring for the sickest of children with cancer. Intrigued by the prospect of specializing in neuroblastoma, a cancer of the nervous system, Dr. Mossé reached out to Brophy to learn more about the disease that left children with limited treatment options. What began as a mentorship soon blossomed into a friendship spanning two memorable decades.
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.
The scientific wonder of stem cell research and its implications for medicine have come a long way in the last decade: At Children’s Hospital of Philadelphia Research Institute, our investigators’ innovative use of stem cell science to approach complex pediatric conditions continues to inspire for their potential to improve outcomes in children’s health. In our latest news roundup, learn about novel stem cell research from our Cancer Center and Division of Urology that aims to preserve the future fertility of boys who undergo childhood cancer treatment. Discover a new project co-led by a CHOP neurology researcher that takes a stem cell approach to restore vision cells in blind dogs.
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.
As we move full-speed ahead into fall, the new season brings with it a handful of headlines from our researchers at Children’s Hospital of Philadelphia. This week, we share the latest news about investigators who have been recognized for their research legacies and for their current cutting-edge work, from discoveries in the basic and clinical science of HIV/AIDS, to the development of learning health system-based training in outcomes research, to the design of innovative approaches to childhood cancer.
Acute myeloid leukemia (AML), the second most common leukemia in children, affects different populations of pediatric patients in different ways. With the support of a new Epidemiology Grant from Alex’s Lemonade Stand Foundation, Richard Aplenc, MD, PhD, assistant vice president and chief clinical research officer at Children’s Hospital of Philadelphia, is leading a powerful research project based on the automated extraction of data to learn more about racial and ethnic disparities observed in African American children with AML.
“We’ve known for a long time that African American children do worse with treatment for acute myeloid leukemia than non-African American children,” Dr. Aplenc said. “But we’ve never really understood why that is, and there are a lot of different possibilities.”
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
Debbie Eaise remembers the day her 18-year-old son, Kevin, walked onto the University of Pennsylvania campus where he is set to play for the Penn Quakers baseball team this fall. From Meiklejohn Stadium, Penn’s home field, you can pitch a baseball and reach Children’s Hospital of Philadelphia, said Debbie — a proximity so palpable that she could see the change in her son’s face and sense his comfort level at ease while he toured his future school.
As her husband, Kevin Sr., and others have noted, Kevin has come full circle. It was right there on the Penn-CHOP campus, after all, with its skyline of medical buildings, that Kevin and his family’s life changed inextricably, ushering in what Debbie describes as their “new normal.”
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.