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In the News: CAR-T Cell Therapy Benefits, Leukodystrophy Research, Social Innovation Awards, Teen Screen Time

Published on
February 9, 2018


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We’re finishing off the week with a winning touchdown of news that gives us as much pride for our researchers as we have for our Philadelphia Eagles in Sunday’s Super Bowl. In this roundup of recent headlines, we cover new results from the game-changing immunotherapy for acute lymphoblastic leukemia (ALL) pioneered by Children’s Hospital of Philadelphia and University of Pennsylvania scientists, announce a generous family donation that will benefit research at our Leukodystrophy Center of Excellence, and share new awards and advances from our talented investigators and clinicians.

CAR-T Cell Benefits Persist in Leukemia Patients

Children and adults treated with the pioneering chimeric antigen receptor (CAR)-T cell therapy, tisagenlecleucel, for acute lymphoblastic leukemia (ALL) continue to show high rates of complete remission, according to updated results published in the New England Journal of Medicine by CHOP oncologists. The study, which reports on new data from the ELIANA trial — sponsored by Novartis and pivotal in the FDA’s approval in August — involved longer-term followups about safety and efficacy from 75 patients ages 25 and younger.

It deemed that overall survival in the 75 patients was 90 percent at six months and 76 percent at 12 months — statistics that show the drug has a durable response rate. Additionally, the CAR-T cells stayed in the patients’ bodies for as long as 20 months after a single infusion of the T cells. This is significant, since infused T cells work to actively seek and destroy leukemia cells in the body after being genetically modified in the lab. Finally, the study also found that any side effects appeared short-lived and reversible.

“One of our more challenging questions — ‘Can we manage the serious side effects of CAR-T cell therapy?’ — was asked, and answered in this global study,” said lead investigator Stephan Grupp, MD, PhD, director of the Cancer Immunotherapy Program at CHOP in a press statement. “Some of our patients get very sick, but we showed that most toxic effects can be short-lived and reversible, with the potential for our patients to achieve durable, complete remissions. That’s a pretty amazing turnaround for the high-risk child who, up until now, had little chance of surviving.”

Learn more about the exciting findings in the press release.

Calliope Joy Foundation Donates $110K Toward Leukodystrophy Research

The Leukodystrophy at Center of Excellence at CHOP received a generous gift of $110,000 from two families affected by leukodystrophy on behalf of the Calliope Joy Foundation. Mari Kefalas and Patt Car, and Matt and Lauren Hammond bestowed the check to support research that might one day lead to breakthrough treatments for leukodystrophy and other similar disorders. Leukodystrophies are a group of inherited disorders characterized by damaged white matter in the brain and spinal cord. They can affect movement, speech, vision, hearing, and overall development. While researchers currently know of 30 types of leukodystrophy, there may be many more yet to be identified.

The Carr-Kefalases, who founded the Calliope Joy Foundation, have first-hand experience in the challenge of leukodystrophy: In June of 2012, they learned that their daughter, Cal, had late infantile onset metachromatic leukodystrophy (MLD). Later that year, the Hammonds found out that their daughter, Loie, had the same diagnosis. Together, the families have raised thousands of dollars to support leukodystrophy research.

“The Children’s Hospital of Philadelphia’s Leukodystrophy Center is one of a handful of centers in the nation that brings together a multidisciplinary group of caregivers to provide cutting-edge, comprehensive diagnostic testing, clinical care, and the most advanced treatment to infants and children with inherited white matter disease,” said Amy Waldman, MD, medical director of the Leukodystrophy Center. “We have built an incredible team, which would not have been possible without the generosity of the Carr-Kefalases and Hammonds. Their donation will help power innovative treatments and breakthrough research.”

Learn more in the press release and read up on recent research from the Leukodystrophy Center on Bench to Bedside.

Dr. Tyra Bryant-Stephens Recognized for Community Asthma Prevention Program

For over two decades, the Community Asthma Prevention Program (CAPP) at CHOP has worked to improve asthma outcomes for children in Philadelphia by harnessing the power of strong community ties, high-performing collaborations, community health workers, and care coordination. Led by its medical director, Tyra Bryant-Stephens, MD, the program continues to study and implement asthma evidence-based interventions, including a recent study focused on West Philadelphia called the West Philadelphia Asthma Care Collaborative. The Social Innovations Journal, Institute, and Lab recognized Dr. Bryant-Stephens for her passionate work in CAPP with a first-place award in the category of “Healthy Communities” at the Greater Philadelphia Social Innovation Awards. Congratulations to Dr. Bryant-Stephens!

Through the West Philadelphia Asthma Care Collaborative, Dr. Bryant-Stephens and her research team completed a community needs assessment of West Philadelphia and its community, health, education, and home stakeholders to create a sustainable asthma care implementation program for children ages 6 to 12 who live in West Philadelphia. The collaborative will roll out the program through community health workers and lay health educators and evaluate outcomes based on asthma control, symptom days, and school absenteeism.

Dr. Katherine Dahlsgaard Weighs in On Teen Screen Time

Does screen time steal happiness from teens? It’s a question on many parents’ minds, and the headline of a recent article written by our own Katherine Dahlsgaard, PhD, ABPP, clinical director of the Anxiety Behaviors Clinic at CHOP. In the story, Dr. Dahlsgaard addresses the flood of new research into the subject of recreational internet and teens, including a recent study that found adolescents who spent more time on the internet also reported less happiness, lower self-esteem, and less satisfaction with their lives.

Speaking directly to parents in the article, Dr. Dahlsgaard writes: “So what is a parent to do? Banning smartphones and the internet isn’t realistic, as those glorious rainbow-colored electronic stallions are most definitely out of the barn. Rather, parents can take comfort in the finding that a moderate amount of recreational screen use does your kids no harm.”

No stranger to offering clear and balanced advice about the latest health research, Dr. Dahlsgaard has been recognized for playing an active role in mediating mainstream headlines: Last July, the Pennsylvania Psychological Association gave Dr. Dahlsgaard the 2017 Psychology in Media Award, offered to a member of the medical community who translates evidence-based science to the public and demonstrates the importance of research in psychological treatment.

Read Dr. Dahlsgaard’s full story on


Recently on Cornerstone, we congratulated Richard Aplenc, MD, PhD, MSCE, on his new role as assistant vice president and chief clinical research officer, heard from our Chief Scientific Officer Bryan Wolf, MD, PhD, about rallying together for the Eagles Autism Challenge, and took a snapshot of new research into the factors that influence a child’s response to acute respiratory tract infections.

Catch up on our headlines from our Jan. 26 edition of In the News:

  • Concussion Research Told Through the Eyes of a Student Athlete
  • Will Vitamins and Supplements Help Mito Patients? Our Experts Weigh In
  • Researchers Study Interactions With Police for Children With Autism
  • Navigating the Pros and Cons of Teens’ Smartphone Use
  • Scientific American Features Dr. Wallace’s Portfolio of Work

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