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CTL019 Wins FDA Panel Support, Driving with ADHD, High School Football, New Genetic Syndrome, Teen Bone Growth, Missed Nursing Care

Published on July 13, 2017 in Cornerstone Blog · Last updated 4 months 4 weeks ago


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CAR T-cell therapy tops this week’s research roundup, with news about the experimental immunotherapy designed to re-engineer a patient’s cells to fight cancer making late-breaking and captivating headlines across the nation. Also in this week’s news: Our researchers are continually studying how we can advance the health and safety of teens in new and novel ways, from football games to that first driving license. Read on to get the latest news from Children’s Hospital of Philadelphia Research Institute.

FDA Panel Votes “Yes” To Recommend CTL019 Approval

News broke Wednesday that a panel of experts in the FDA Oncology Drugs Advisory Committee voted a unanimous “yes” in favor of recommending the first CAR T-cell therapy for approval. If approved, the CAR T-cell treatment, CTL019, would be commercially available for the treatment of children and young adults with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (B-cell ALL). The FDA panel consisted of 10 experts who put CTL019’s safety and efficacy data under a microscope before making their recommendation. As we reported previously, Novartis’ Biological License Application for CTL019 currently sits under priority review by the FDA.

The story of CTL019’s development is one we know well at CHOP Research Institute, beginning with the treatment’s earliest trials in children and young adults led by Stephan Grupp, MD, director of the Cancer Immunotherapy program at CHOP. The very first pediatric patient in the world to receive the experimental immunotherapy, Emily Whitehead, was treated right here at CHOP at the age of 6. Emily now lives cancer-free at 12 years old.

"We know firsthand from treating children and young adults with relapsed/refractory B-cell ALL that they desperately need innovative medicines that provide a new approach to managing this aggressive disease," stated Dr. Grupp in a Novartis press release. "Today's vote in favor of CTL019 is a positive step, and we appreciate Novartis' commitment to pediatric patients."

You can read more about CTL019’s recommendation in the Novartis press release or the New York Times.

Dr. Thomas Power Talks ADHD and Driving for Medscape

A JAMA Pediatrics study from researchers at the Center for Injury Research and Prevention (CIRP) at CHOP made waves in the recent press when they reported that young drivers with attention-deficit/hyperactivity disorder (ADHD) have an elevated crash risk – but one that is significantly lower than what previous studies have reported. Now, Thomas J. Power, PhD, director of the Center for Management of ADHD at CHOP and co-author of the paper, joined forces with the online health news outlet, Medscape, to explain what these findings mean for teen drivers, parents, and clinicians. According to Dr. Power, the risk is “manageable” for most teens with ADHD, but concern levels should vary depending on subgroups of adolescents, such as those with substance use disorders or antisocial behavior problems.

“For those individuals, we would be very concerned and want to make sure that their problems are being addressed before we advance them to the learning-to-drive stage,” wrote Dr. Power. In his commentary, Dr. Power also touched on how parents can kick-start conversations about driving with their teens early, and he discussed the role that physicians and the proper medication play in promoting safer driving.

You can read Dr. Power’s segment (and watch a quick video) on Medscape.

JAMA Neurology Study Looks at Brain Health Years After High School Football

Years after playing high school football, does the heavy-contact sport leave its mark on the brain? In a new study published in JAMA Neurology, a team of researchers that included Christina Master, MD, sports medicine pediatrician at CHOP, studied a group of men who graduated high school in Wisconsin in 1957 and a group of healthy controls to see whether exposure to head trauma through football had any associations with cognitive impairment or depression at 65 years of age. The researchers conducted assessments of verbal fluency, memory, and attention as well as a depression scale in over 3,000 men. The study team concluded that participating in high school football showed no statistically or clinically significant harmful association with poor brain health later in life. While the findings are pretty positive for young athletes, the researchers noted that the risks of playing football in our day and age may have changed since the 1950s.

You can read the study abstract online

Healthy Teen Years Make for Strong Bones

Here’s another reason why teens should stay active and eat healthy if they want to build lifelong health: Even after they’ve reached their adult height, adolescents in their late teens are still in a key period for building strong bones. In a study published in JAMA Pediatrics, a team led by Shana McCormack, MD, MTR, pediatric researcher at CHOP, found that roughly 10 percent of bone mass continued to accumulate in teens even after they stopped growing height-wise. Because we gain height faster than we gain bone mineral, that may help explain why so many fractures happen while we are young. Roughly 30 percent to 50 percent of children experience at least one fracture before becoming adults. For this multicenter investigation, the researchers had analyzed data from the Bone Mineral Density in Childhood Study (BMDCS), funded by the National Institutes of Health, which includes the bone and growth measurements of over 2,000 healthy children, adolescents, and young adults taken in annual visits for up to seven years.

“Late adolescence is when some teenagers adopt risky behaviors, such as smoking and alcohol use, worse dietary choices and decreased physical activity, all of which can impair bone development,” Dr. McCormack said. “This period is a time for parents and caregivers to encourage healthier behaviors, such as better diets and more physical activity.”

Read the press release online and the study abstract in JAMA Pediatrics.

Pediatric Nurses Thrive in Positive Work Environments

A supportive work environment with a reasonable patient load is essential for nurses to provide optimal care – and a new study from CHOP and the University of Pennsylvania’s School of Nursing’s Center for Health Outcomes and Policy Research has the data to prove it. In new research published in the journal  Hospital Pediatrics, investigators conducted surveys of 2,187 registered pediatric nurses at 223 hospitals across the U.S.

The surveys included questions about patient loads, adequate staffing and resources, a nurse’s relationship with physicians and nurse managers, and whether they skipped on any care activities because of a lack of time. These reports helped the researchers to determine the quality of the nurse’s environment (which were categorized into “poor,” “better,” and “mixed”). Ultimately, the researchers found that for every additional patient added to their load, nurses were 70 percent more likely to miss care, while 61 percent of nurses reported a missed care activity in “poor” environments. The most common missed care activities included comforting, teaching, planning, and counseling.

“This study points to the importance of examining what our nurses need to provide the best care possible,” said Paula M. Oliver, PhD, BSN, Senior Vice President and Chief Nursing Officer at CHOP in a press release. “Pediatric nurses care for some of the most fragile patients, and we need to be sure they are provided a supportive environment in which to work.”

Learn more in the press release.

CHOP Team Identifies New Genetic Syndrome

Researchers have identified a new genetic syndrome that will bring answers to some parents struggling through a diagnostic odyssey for their child’s unclassified condition. In novel research led by Matthew Deardorff, MD, PhD, pediatric geneticist at CHOP, a team of scientists have classified a distinctive set of symptoms that includes developmental delays, an abnormal gait, unique facial features, and seizures with reduced expression of the WDR26 gene. The scientists were able to identify 15 individuals between the ages of two and 24 years old who shared the WDR26 gene mutation along with intellectual disabilities of some degree, subtle abnormalities in their walk, similar facial features (such as a wider mouth, fuller cheeks, and a prominent upper lip and gums), and the experience of seizures.

“Prior to our identification of individuals with changes in this gene, it was not even listed in some of the most commonly used databases,” said Dr. Deardorff in a press release. “The notable efforts by our colleagues here in the Division of Genomic Diagnostics at CHOP, and at key labs in the Netherlands and Maryland, helped us to make this discovery possible.” Dr. Deardorff and his fellow researchers published their findings in the American Journal of Human Genetics. Read more about WDR26 haploinsufficiency in the press release.


Recently on Cornerstone, we took a look at the latest from Healthy NewsWorks, a Philadelphia-based educational program in which Drs. Paul Offit, MD and Senbagam Virudachalam are involved. We also held a Q&A session with the research team in Paula Oliver’s lab to learn more about how cells known as “peacekeepers” prevent autoimmune disease.

Catch up on our headlines from our June 30 edition of In the News:

  • CHOP Collaborates to Develop the ‘Flexibility Scale’ for Children with Autism
  • New Study: Electronic Health Record Tools can Strengthen Concussion Management
  • CHOP Immunologist Awarded Pew Scholar Designation
  • Study Finds Technology Addiction May Lead to Distracted Driving
  • CHOP Tops U.S. News & World Report Rankings for Children’s Hospitals

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