AAP Conference, PCORI Sickle Cell Research, Eagles Autism Challenge, Anxiety and Autism, New Immunotherapy Target for Neuroblastoma

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. As Children’s Hospital of Philadelphia experts made waves at the American Academy of Pediatrics (AAP) National Conference in Chicago, our local Philadelphia Eagles launched a new commitment to support autism research at CHOP and neighboring institutions back home. Read on to learn more about the latest headlines in CHOP research – including the detection of a potential immunotherapy target for neuroblastoma.

CHOP Experts Attend AAP Annual Meeting and Accept Awards

This week in Chicago, over 50 experts from CHOP attended the American Academy of Pediatrics’ National Conference and Exhibition to share their knowledge and research about some of the most important issues in children’s health today, from vaccine education, to sports injuries, to telemedicine, and more. Every year, the AAP organizes the event as a hub for clinicians and researchers across the U.S. to learn, network, and educate their colleagues. A handful of our researchers delivered presentations, including Paul Offit, MD, director of the Vaccine Education Center, who spoke on vaccine safety, and Susan E. Levy, MD, director of the Autism Integrated Care Program, who shared her insights into screening for autism.

In addition, three CHOP clinicians received distinguished awards for their work in diverse fields of pediatric research. Warm congratulations go out to Dr. Levy; Christopher Feudtner, MD, director of Research for the Pediatric Advanced Care Team; and Kathy Shaw, MD, emergency medicine physician. Dr. Feudtner received the William G. Bartholome Award for Ethical Excellence, an honor that recognizes an individual who has impacted public discussions of ethical issues in pediatric medicine. Dr. Shaw received the Jim Seidel Distinguished Service Award, a title bestowed on an individual for their contributions to pediatric emergency medicine. And Dr. Levy received the Arnold J. Capute Award, given to an AAP member for their outstanding contributions in the field of children with disabilities.

“I am truly honored to receive the Arnold J. Capute Award, particularly given the outstanding character and accomplishments of previous awardees,” said Dr. Levy in a press statement. “It has given me the opportunity to look back on my career to draw insight about the road I have traveled, and appreciate all who have contributed to the health and well-being of children with disabilities.”

Learn more about this year’s AAP Conference in the press release.

CHOP Sickle Cell Research Receives PCORI Funding

For young adults with sickle cell disease, going from pediatric to adult care is a critical time: Youth tend to experience increased rates of death and more dependence on healthcare services like the emergency department compared to other age groups. Without access to pediatric specialty care, these patients often miss the preventive care and screening visits that help them to manage the pain that accompanies the genetic disorder. Now, a new $8.5 million project funded by the Patient-Centered Outcomes Research Institute (PCORI) will help CHOP researchers discover and develop the strongest evidence-based methods to improve the quality of life for sickle cell disease patients. CHOP will conduct the study in partnership with Northwell Health’s Cohen Children’s Medical Center.

The project will explore the impact and strength of six-month community health worker programs and mobile health applications as compared to enhanced usual care through a randomized clinical trial. Led by David Rubin, MD, director of PolicyLab at CHOP, the team will include young adult patients with sickle cell disease, parents, pediatric and adult clinicians, policy makers, payers, and community and advocacy groups. PCORI has provided the funding as part of their $80 million investment in sickle cell disease research.

“While transitioning to the adult health care system is difficult to navigate for many youth and their families, those with chronic conditions and complex medical needs face particularly challenging barriers,” said Dr. Rubin in a press release. “We’ve heard from youth with sickle cell disease and their families that the transition of their care is their greatest concern, beyond their health. With this study, we’re seeking to understand what tools we can provide outside of the doctor’s office so these patients feel actively involved in the transfer of their healthcare.”

Learn more in the press release.

Philly Eagles Announce Eagles Autism Challenge

The Philadelphia Eagles are taking flight for autism again – but this time in the form of a fun-filled day of cycling, running, and walking for children of all ages. Jeffrey Lurie, chairman and CEO of the Eagles, announced the launch of the Eagles Autism Challenge last week, explaining that 100 percent of all participant-raised funds for the community event will go toward autism spectrum disorder (ASD) research at CHOP, Drexel University, and Thomas Jefferson University and Jefferson Health. All three institutions have robust research programs dedicated to improving how we care for individuals with autism. The Eagles Autism Challenge will help to provide the resources that doctors and scientists need to drive scientific breakthroughs for current and future generations touched by autism. Scheduled for Saturday, May 19, 2018, the Eagles Autism Challenge features a 5K run/walk as well as cycling routes of 15, 30, and 50 miles – all of which will begin at Lincoln Financial Field. Participants will join Eagles players, alumni, coaches, executives, cheerleaders, and SWOOP at the event.

"Teams at Children's Hospital of Philadelphia care for thousands of children on the autism spectrum each year," said Madeline Bell, president and CEO of CHOP in a press release. "These patients and their families need answers, solutions and treatments that can only come from breakthroughs in research – and they are counting on us to make those breakthroughs. Fundraising events like the Eagles Autism Challenge give our teams the resources they need to make discoveries that will change our patients' lives today and help them thrive in adulthood."

Learn more about CHOP’s involvement in the Eagles Autism Challenge in the press release.

CHOP Study Shares New Insights Into Anxiety and Autism

A new study from the Center for Autism Research (CAR) at CHOP sheds new light on how the size of an individual’s amygdala – the region of our brains that processes emotions – might relate to autism. According to the research, published in the Journal of Autism and Developmental Disorders, children with both autism and anxiety tend to have a smaller amygdala than those with autism but no anxiety. This difference in size may relate to how certain subgroups of individuals on the autism spectrum process emotions.

The researchers had measured the volume of various brain regions in 53 children with autism using magnetic resonance imaging. Twenty-nine of those children had anxiety as well as autism, diagnosed using a clinical questionnaire. The findings underscore the importance of thinking beyond the idea of one single “autism brain,” and toward a more nuanced perspective.

“It is no longer sufficient to say, ‘Is the amygdala different in children with autism?’” John Herrington, MD, assistant professor of psychology in psychiatry at CHOP and first author of the study, told Spectrum News. “The question you have to start asking is, ‘What components of autism are related to the amygdala?’” This research was part of CAR’s wider efforts to discover biological indicators of anxiety in children with autism.

You can learn more about this research on Spectrum News.

CHOP Researchers Identify Potential Target for Neuroblastoma Treatment

Neuroblastoma, a cancer of the developing peripheral nervous system, accounts for 50 percent of all cancers in infants – making it the most common cancer for babies. Most children with neuroblastoma are diagnosed before they reach the age of 5, when a solid tumor may be discovered at the top of the kidneys (the adrenal glands), the chest, spinal cord, or abdomen. Over the last decade, oncology investigators at CHOP have made strides in neuroblastoma research and treatment. Now, a new study on the cover of Cancer Cell’s September issue describes the discovery of a new target for the prevention and treatment of neuroblastoma using immunotherapy – where potent drugs harness the power of the immune system to fight cancer cells.

Led by Kristopher Bosse, MD, attending physician in the division of Oncology and first author of the study, the researchers began their research by scanning the surface of neuroblastoma cells to detect molecules that could be targeted by immunotherapy without harming healthy cells. They zeroed in on a cell-surface protein found on neuroblastoma called glypican-2, or GPC2 – and found that GPC2 is necessary for neuroblastoma cells to proliferate. With this promising new discovery, the researchers worked with the National Cancer Institute to develop an antibody-drug conjugate (ADC) that was able to kill neuroblastoma cells in cell cultures and mouse models – with no apparent harm to normal cells.

“These findings establish that this type of immunotherapy could be potentially safe and effective against neuroblastoma,” said John Maris, MD, pediatric oncologist at CHOP and the study’s supervisor. “Our next steps will be to further evaluate this ADC and also develop other immune-based therapies directed against GPC2. Because other glypicans in addition to GPC2 are overexpressed in other childhood cancers, it may also be possible to apply this approach across various types of high-risk pediatric cancers.”

Learn more about the exciting breakthrough in the press release.

ICYMI

Recently on Cornerstone, we sat down with Barbara Schmidt, MD, attending neonatologist at CHOP and recipient of the American Academy of Pediatrics’ William A. Silverman Lectureship Award, to chat about the honor. We also met our September Research Hero, 2 ½ year old Clay Maresca, and took a quick look at a new discovery about the development of bones well into the teenage years.

Meanwhile, on Instagram, we handed our account over to the Office of Academic Training and Outreach Programs to get a glimpse into life as a postdoc.

Check out the photos from their daily celebrations of National Postdoc Appreciation Week! Catch up on our headlines from our Sept. 8 edition of In the News:

• FDA Approves World’s First CAR-T Cell Therapy
• Hyundai Gives CHOP Research Hope on Wheels
• Philly Eagles Take Flight for Autism Research
• Dr. Arbogast Leads NFL Engineering Roadmap for Concussion Research
• Diane Spatz Joins Congressional Task Force
• Doug Wallace Receives Dr. Paul Janssen Award at Symposium

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