More than six years after Emily Whitehead became the first child to receive chimeric antigen receptor (CAR) T-cell therapy, doctors have had remarkable success in turning the immune systems of even more children with acute lymphoblastic leukemia (ALL) into top-notch fighters against the disease. For some patients, however, these superhero T-cells still fail in their mission to find and fight their cancer targets.
Tag Archive: National Cancer Institute
Creating the opportunity for every child with cancer to enjoy a healthy life beyond their cancer experience is the dream for pediatric oncology researchers. New grants awarded by the National Cancer Institute Moonshot Initiative through a multi-institutional, collaborative group — the Pediatric Immunotherapy Discovery and Development Network (PI-DDN) — aim to bring this dream closer to reality by fundamentally changing our understanding of how to harness the power of the immune system to treat childhood cancers.
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 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.
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.
Long before he entered medical school, John M. Maris, MD, pediatric oncologist and the recent recipient of the National Cancer Institute’s Outstanding Investigator Award, became captivated by the mysteries of neuroblastoma. A cancer of the peripheral (not brain) nervous system, neuroblastoma accounts for 7 percent of all childhood cancers and 15 percent of all childhood cancer deaths.
Notable awards, new autism initiatives, and a novel approach to managing sickle cell disease are all part of this week’s roundup of research news.
Read on for more exciting headlines from this week, including highlights from our inaugural “Deciphering Beckwith-Wiedemann Syndrome” conference.
This week we’re all about getting smart in our highlights of research news from The Children’s Hospital of Philadelphia. Getting smart in the approach to tackling childhood cancer means identifying strategies that will make a decade’s progress in half the time.