Alex's Lemonade Stand, Button Batteries, Mitochondrial Medicine, Project EVO, Citizen Diplomat Award

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.

CHOP Takes a Stand for Childhood Cancer

In a summertime tradition for 15 years and counting, the Cancer Center held its annual Alex’s Lemonade Stand Day on Friday, June 8, serving up lemonade at 28 stands throughout the city of Philadelphia. Every year, CHOP dedicates a special day to support ALSF’s mission of “changing the lives of children with cancer” by funding research, raising awareness, and empowering everyone to help cure childhood cancer. The day also honors Alexandra “Alex” Scott, the nonprofit organization’s founder. At 4-years-old while battling neuroblastoma, Alex raised more than $2,000 for cancer research by selling lemonade in her front yard. Over the years, ALSF has played a key role in helping to drive breakthroughs by supporting a number of CHOP oncology investigators and their novel research.

This year, CHOP awarded its annual “Pitcher of Hope” award to Ann-Marie Leahey, MD, an attending physician at the Cancer Center at CHOP who specializes in treating children with malignant eye tumors. In Alex Scott’s honor, the Department of Pediatrics at CHOP also announced the establishment of a $2 million endowed chair in the name of Pat Brophy, who was Alex’s favorite nurse at CHOP. According to the Cancer Center, endowed chairs allow CHOP to “fund the most talented doctors and scientists in the world, providing critical funding for clinical work and research.”

Thank you to all who participated in Friday’s event!

New Research Shows Honey Helps Protect Against Button Battery Injuries

Button batteries (also known as lithium batteries) have posed a risk to toddlers and young children for decades: More than 2,500 times a year in the U.S., children ingest the small and smooth candy-shaped discs found in remote controls, cell phones, watches, and other household objects. When swallowed, the batteries can get lodged in a child’s throat where saliva triggers an electric current and a chemical reaction that burns the esophagus in as little as two hours. Now, a new study from a team of ear, nose, and throat (ENT) specialists at CHOP and Nationwide Children’s Hospital has shown that eating honey after swallowing a button battery can potentially reduce serious injuries.

Reporting their findings in The Laryngoscope, the research team tested several types of liquids in a live animal model for those that can create a protective barrier for throat tissue and neutralize harsh alkaline levels. Out of a variety of common household and medicinal liquids, they found that honey and sucralfate had the most protective effects.

“Our recommendation would be for parents and caregivers to give honey at regular intervals before a child is able to reach a hospital, while clinicians in a hospital setting can use sucralfate before removing the battery,” said Ian Jacobs, MD, medical director of the Center for Pediatric Airway Disorders at CHOP and co-principal investigator of the study, in a press release. “While future studies could help establish the ideal volume and frequency for each treatment, we believe that these findings serve as a reasonable benchmark for clinical recommendations.”

Learn more in the press release and check out coverage from philly.com.

Mitochondrial Medicine Researchers Study Trials From a Patient’s Perspective

As research into mitochondrial disease, a highly variable collection of energy deficiency disorders, continues to ramp up at CHOP, investigators in our Mitochondrial Medicine Frontier Program have published a PLOS ONE paper that details the perspectives of our two most important stakeholders in pediatric research: patients and their families.

Led by Marni Falk, MD, executive director of the Mitochondrial Medicine Frontier Program, the study provides key insights into how researchers can best design clinical trials and measure clinical outcomes that are meaningful to patients and families, while also developing those trials with the goal of attaining U.S. Food and Drug Administration (FDA)-approval for mitochondrial medicine treatments.

Through online electronic surveys conducted in two patient cohorts, Dr. Falk and her team gathered information about mitochondrial patients’ most prevalent and disabling symptoms as well as the factors that would motivate or dissuade them to participate in a clinical trial. Factors like taking a new drug not previously studied in people, or experiencing disease progression, were some reasons why individuals might not join a clinical trial. Taking a self-administered drug (like pills or vitamins), and guaranteed access to treatment during and after the study, were some positive reasons to participate in research.

“Understanding and applying patient preferences to trial design and outcome selection are essential to achieve successful therapies for all of the rare, complex diseases that are now readily recognized due to advances in genetic diagnostics,” said Dr. Falk in a CHOP press release.

Read more about the study’s findings in the press release.

Project: EVO Leads the Field in Digital Treatments for ASD & ADHD

Earlier this year, we shared an exciting research initiative from CAR that features a video game as an interventionfor dual diagnoses of autism spectrum disorder (ASD) and attention-deficit/hyperactivity disorder (ADHD). Poised to be the first prescription digital medicine approved by the FDA to improve attention in individuals with autism and ADHD, the intervention is a fun and challenging iPad-based game designed by Akili Interactive Lab. Though it looks and feel like an ordinary video game, Project: EVO involves selective targeting and activation of specific cognitive neural systems in the brain.

Last week, Akili Interactive Lab released early data on the game’s impact in a randomized, controlled clinical trial. According to a CAR blog post about the update, the results showed that the game improved a key component of cognition thought to contribute to attention difficulties in children with ADHD. A pilot study led by Benjamin Yerys, PhD, a child psychologist at CAR, previously found that children with both ASD and ADHD liked and engaged with the game. Furthermore, on a classic attention test, their attention improved to what was seen in children with ADHD alone.

Dr. Stephan Grupp Named 2018 Citizen Diplomat of the Year

On June 7, Citizen Diplomacy International of Philadelphia, a nonprofit international relations organization, awarded our own Stephan Grupp, MD, PhD, director of the Cancer Immunotherapy Program at CHOP, with their 2018 Citizen Diplomat of the Year Award. According to Citizen Diplomacy International, the award recognizes Dr. Grupp for “raising Philadelphia’s international profile through his innovative advancements in medicine and his commitment to ensuring that people all around the world have access to this treatment.”

In collaboration with the Perelman School of Medicine and Novartis, Dr. Grupp led global clinical trials of the innovative life-saving chimeric antigen receptor (“CAR”) T-cell therapy for children with advanced acute lymphoblastic leukemia. Dr. Grupp has also traveled around the world to educate doctors and teachers about the cancer immunotherapy. In 2013, 60 Minutes Australia featured a segment on Dr. Grupp and CAR T-cell therapy.

Learn more about Dr. Grupp’s Citizen Diplomat of the Year Award here.

ICYMI

Recently on Cornerstone, we shared a guest blog from our friends at PolicyLab in recognition of the Center of Emphasis’ 10th anniversary, brought you highlights from the Symposium on Advances in Genomics, Epidemiology and Statistics (SAGES) hosted by CHOP and Penn, congratulated nephrology fellow Melissa Meyers, MD, for receiving the National Kidney Foundation’s National Young Investigator Forum Award, and celebrated three major distinctions earned by clinical geneticist Elizabeth Bhoj, MD, PhD.

Catch up on our headlines from our June 1 edition of In the News:

• CIRP Webinar Discusses Child Mortality Rates
• Premature Infants Continue to Receive Early Exposure Antibiotics
• CHOP Hosts IBD and VEO-IBD Symposium
• For Breakthroughs: Recent Fundraisers Support Research
• A Lifetime of Achievement for Robert Campbell, MD

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