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.
If you’re looking for a spark of inspiration during January’s long and sometimes dreary days, don’t miss this week’s roundup of headlines from in and around Children’s Hospital of Philadelphia Research Institute. Our scientists’ passionate work in the lab found a spotlight in the mainstream media as “TODAY” featured how our stem cell research can help today’s cancer survivors become tomorrow’s parents. Meanwhile, eye-opening findings from the Lifespan Brain Institute (LiBi) sparked a wider conversation about how pediatricians and parents can stay alert for suicidal thoughts in teens. In more news, a recent study highlighted the need for more antibiotic stewardship in non-children’s hospitals, while a successful device consortium based at CHOP officially became a statewide affair.
As teens transition to adulthood, being able to get around on their own is a big step toward independence, enabling opportunities for social activities, post-secondary education, and work.
But what about this rite of passage for adolescents on the autism spectrum? How does their experience differ from their peers? These are the types of questions Allison Curry, PhD, MPH, wants to answer with the help of a new grant to fund a groundbreaking project that has the potential to help change the lives of many teens and young adults with autism.
Looking into the eyes of a distressed parent, you want to be able to tell them you’re providing interventions that are based on good evidence for the care of their child. When a gap in knowledge prevents that clinical confidence, Martha Curley, RN, PhD, FAAN, pediatric critical care nurse and research scientist, is there to help find answers.
“The main reason I completed a PhD in nursing science was so I could ask and answer questions relevant to the patient population I cared for as a critical care nurse,” said Dr. Curley, professor of nursing and Ruth M. Colket Endowed Chair in Pediatric Nursing, Children’s Hospital of Philadelphia, and professor of anesthesia and critical care medicine, Perelman School of Medicine at the University of Pennsylvania.
Dealing with a mystery illness that doesn’t appear to match any conditions described in medical textbooks can entangle families on a diagnostic odyssey for years and even decades. Dozens of examinations, a barrage of tests, multiple misdiagnoses, and trips in and out of hospitals can leave them exhausted and no closer to knowing what is wrong with their child.
Those struggling to find a diagnosis can now turn to a new team of super sleuths from Children’s Hospital of Philadelphia and the Hospital of the University of Pennsylvania who are ready to unravel these perplexing diseases, in both children and adults. The two institutions received $2.5 million in research grants from the National Institutes of Health as a newly designated Undiagnosed Diseases Network (UDN) site.
Friedreich ataxia (FRDA) is a rare, progressive autosomal recessive neurodegenerative disease characterized by progressive gait and limb ataxia; cerebellar, pyramidal, and dorsal column involvement; visual defects; scoliosis; and cardiomyopathy. FRDA is caused by transcriptional silencing of the frataxin gene and consequential deficiency of frataxin, a mitochondrial protein crucial for iron–sulphur cluster biogenesis and adenosine triphosphate (ATP) production. ATP stores and transports chemical energy within cells. Currently, no therapy is available to slow down the progression of FRDA.
In this edition of In the News, learn about digital medicine and what it means for children with autism spectrum disorder and co-occurring attention deficit/hyperactivity disorder (ADHD). Read about a variation in care study, a recent FDA approved drug treatment for a specific childhood cancer, and why two Children’s Hospital of Philadelphia researchers are calling for improved pediatric palliative care measures. And lastly, join us in celebrating the addition of six new initiatives to CHOP’s Frontier Programs.
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.
One-month-old Connor only dimly perceives the world around him. Just two months later, when he sees his mother’s beaming face or hears his father’s hearty laugh, he smiles with recognition. And by the time Connor reaches his first birthday, he’s starting to put one foot in front of the other and verbally communicate with his parents. Just what accounts for these significant changes?
Editor’s Note: After over five years of discussion from the advance notice of proposed rulemaking and two implementation delays, it appears that changes to the Common Rule regulations overseeing human subjects research (45 CFR 46) are finally set to go into effect Jan. 21. Heather Cathrall, MBE, CIP, assistant director of Institutional Review Board (IRB) Operations at Children’s Hospital of Philadelphia, explains what researchers need to know about the revised Common Rule. Protecting human participants in biomedical research is a top priority at CHOP Research Institute, and the IRB team is revising its electronic IRB system, standard operating procedures, templates, and webpage to comply with these new regulations. Also, the IRB is leading a series of trainings for CHOP investigators, study staff, IRB members, and IRB staff.