Cystic Fibrosis Research, Pitcher of Hope Award, Kidney Stones, Adolescent Health

 

In this week's research news, Children's Hospital of Philadelphia receives a collaborative grant to drive progress forward for cystic fibrosis (CF) treatment, and honors go to John Maris, MD, from Alex's Lemonade Stand and Eric Chien-Wei Liao, MD, PhD, from the American Association of Plastic Surgeons for meaningful contributions to their respective fields. We also highlight a new approach to improve adolescent health discussions in primary care, and screening to connect families with nutrition resources.

Emily's Entourage Awards Inaugural Collaborative Research Grant to CHOP in Support of Cystic Fibrosis Treatments

Emily's Entourage (EE) awarded a $858,932 collaborative grant to researchers from CHOP, the University of Iowa, and the Rochester School of Medicine and Dentistry. The nonprofit accelerates research for new therapies and a cure for individuals with CF who do not benefit from existing treatments.

CF is a genetic disorder caused by mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene that severely affects the lungs, digestive system, and other organs. Significant advancements have been made in CFTR therapies that have improved the quality of life and life expectancy for individuals with CF. However, approximately 10% of individuals with CF still lack effective treatment options.

Beverly Davidson, PhD, will lead the newly funded project, "Enhanced Airway Transduction and Therapeutic Cargo Delivery with Novel Adeno-associated Virus (AAV) Capsid Variants," for CHOP. Dr. Davidson, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics and chief scientific strategy officer at CHOP, will work collaboratively with scientists across the two other university systems to address barriers to effective treatments through the emergence of new technologies that restore CFTR function to people with CF.

Learn more about the new EE collaborative funding initiative to accelerate groundbreaking CF research.

John Maris, MD, Honored With Alex's Lemonade Stand 2024 Pitcher of Hope Award

World-renowned pediatric oncologist John Maris, MD, is this year's recipient of the Alex's Lemonade Stand Pitcher of Hope Award. Dr. Maris, the Giulio D'Angio endowed chair in Neuroblastoma Research at CHOP, accepted the honor during a ceremony held May 31 in the Hub for Clinical Collaboration on CHOP's Philadelphia campus. This award recognizes a CHOP employee who has had a positive impact on the CHOP Oncology Department and the lives of children with cancer.

Dr. Maris, who was Alexandra "Alex" Scott's oncologist when she was a patient at CHOP, leads a team of researchers dedicated to rapidly translating research about childhood cancers. His research focuses on improving pediatric cancer cure rates, minimizing long-term side effects, and designing studies to help benefit children who have refractory or relapsed diseases for which treatment options may be limited.

"John is arguably the premier pediatric cancer researcher," said Stephen Hunger, MD, chief of the Division of Oncology. "Because of discoveries made in this research group, new CAR T-cell therapies are in clinical trials for children with neuroblastoma, brain tumors, and other cancers. John is a fitting winner of this award."

Dr. Maris is an inaugural member of Alex's Lemonade Stand Foundation's Scientific Advisory Board and helped design its childhood cancer grant program. This program, having raised over $300 million, has funded more than 1,500 medical research grants at close to 150 institutions to find better treatments and cures for children with cancer.

Alex's parents, Liz and Jay Scott, were also on hand to congratulate Dr. Maris and mark the 20th year of Alex's Lemonade Stand at CHOP.

Read more about the event in this CHOP news release.

American Association of Plastic Surgeons Recognizes Dr. Eric Chien-Wei Liao

The American Association of Plastic Surgeons (AAPS) honored Eric Chien-Wei Liao, MD, PhD, with its Basic Science Research Award. Dr. Liao, a pediatric plastic surgeon-scientist and the founding director of the Center for Craniofacial Innovation at CHOP, accepted the award at the AAPS Annual Meeting.

"I am thrilled to shine this recognition on the work we are pioneering at CHOP and on our craniofacial team," Dr. Liao said. "CHOP is where the world comes, not just for answers but for cures."

Dr. Liao cares for surgical patients born with orofacial clefts and complex craniofacial conditions. He also leads the Department of Surgery as the Vice Chair of Academic Affairs, where he is dedicated to nurturing trainees and faculty toward impactful academic careers.

Read more about Dr. Liao and his work in this CHOP news release.

'The Pulse' Podcast Featuring Flaura Winston, MD, PhD, Now Available

Flaura Winston, MD, PhD, participated as a special guest in a live 10th anniversary celebration of "The Pulse," WHYY's weekly health and science radio program and podcast hosted by Maiken Scott. The resulting radio program recently aired, and the podcast of the milestone recording is now available for listening on various podcast platforms.

The milestone event, themed "Science That's Changed Our Lives," featured scientists whose innovative ideas and work have led to meaningful societal change.

Dr. Winston is the founder of the Center for Injury Research and Prevention (CIRP) at CHOP and director of the National Science Foundation Center for Child Injury and Prevention Studies. Her public health research established the evidence base for crucial injury prevention patents, products, programs, and laws.

Listen to The Pulse 10th Anniversary Show: The Science Changing Our Lives, and read more about Dr. Winston's research and future directions for CIRP.

Gregory Tasian, MD, MSCE, Presents Data Supporting Shock Wave Lithotripsy for Kidney Stones in Children

Gregory Tasian, MD, MSCE, director of the Pediatric KIDney Stone (PKIDS) Care Improvement Network, presented findings at the 2024 American Urological Association (AUA) annual meeting that showed shock wave lithotripsy (SWL) resulted in similar stone clearance and better-lived experiences compared with ureteroscopy (URS) in pediatric patients with small- to medium-sized kidney or ureteral stones.

The PKIDSCare Improvement Network embedded a prospective observational trial of patients ages 8 to 21 years old in clinical care at 30 medical centers in North America from 2020-2023 to compare URS and SWL for kidney and ureteral stones. Based on their findings, Dr. Tasian and his co-authors recommended three revisions to the AUA guidelines based on their findings:

• Changing the size threshold to 15 mm for kidney stones from the current threshold of 20 mm
• Recommending SWL over URS for kidney stones less than 10 mm
• Considering possible greater stone clearance and worse patient experience with URS for kidney stones 10-15 mm.

This work was supported through a Patient-Centered Outcomes Research Institute Project Program Award.

Learn more about the findings in this Urology Times release. Read the abstract in The Journal of Urology.

CHOP Researchers Aim to Improve Adolescent Health Discussions and Screening

CHOP researchers developed a comprehensive adolescent health questionnaire (AHQ) integrated with the electronic health record (EHR), to better capture adolescent strengths and risks in systematized and actionable ways in pediatric primary care. They then evaluated the AHQ's impact on the collection of information on prioritized health-related domains. The study team published their findings in Pediatrics.

Following a pilot test of the AHQ, the researchers scaled and assessed the questionnaire's impact on data collection. Scaling and post-scaling outcomes included RE-AIM measures:

• Reach (total questionnaires completed)
• Effectiveness (capture of key information across health domains pre- vs post-AHQ scaling)
• Adoption (proportion of practices that adopted the AHQ)
• Implementation (proportion of eligible adolescents who completed the AHQ)
• Maintenance (monthly completion rates).

The study team suggests that successful implementation of the AHQ in a widely used EHR provides a model for improved primary care for adolescents.

Brian P. Jenssen, MD, MSHP, faculty member at Clinical Futures, a CHOP Research Institute Center of Emphasis, is first author on the paper, joined by Alexander G. Fiks, MD, MSCE, director of Clinical Futures, and other CHOP experts.

Learn more about this work on the Clinical Futures blog.

CHOP Researchers Develop Screening Tool to Help Improve Access to Federal Nutrition Programs

Researchers from The Possibilities Project and Clinical Futures, a CHOP Research Institute Center of Emphasis, developed, implemented, and successfully tested a nutrition screener to improve access to health resources for families eligible for federally funded food benefits. The findings were in the journal Annals of Family Medicine.

The Special Supplemental Nutrition Program for Women, Infants, and Children (WIC) and the Supplemental Nutrition Assistance Program (SNAP) have been linked to improved health outcomes and decreased healthcare costs. However, many eligible families do not receive these program benefits due to the complex application and enrollment process.

"Prior studies at CHOP show that 50% of people who are eligible for services like WIC might not actually be taking advantage of them," said study co-author George Dalembert, MD, associate director of the Center for Health Equity at CHOP.

The Possibilities Project at CHOP aims to design innovative strategies and implement new ideas to better serve patients. Researchers designed and tested a standardized process for linking families to WIC and SNAP during pediatric primary care visits. A tablet-based questionnaire asked eligible families whether they were interested in help applying for WIC or SNAP or wanted information about local food banks.

"The approach we developed may be one important way to normalize social needs assessment and provide families with additional nutrition support," said lead study author Aditi Vasan, MD, an attending physician with the Division of General Pediatrics at CHOP.

In the first month of the pilot in November 2022, 433 families completed the screener (74% in person, 26% via patient portal), and 73 (17%) requested nutrition resources, including 17 who requested information about local food banks, and 56 families who requested benefits application support.

Read more in this CHOP press release

ICYMI

Catch up on our headlines from our May 24 In The News:

• CSO Dr. Susan Furth Inducted into the College of Physicians of Philadelphia
• Cell and Gene Therapy Collaborative Announce FY25 Grant Recipients
• Bone Marrow Atlas Offers First of Its Kind Biomolecular Breakdown of Cells
• Genetics, Environment, Health Disparities Linked to Increased Stress in Adolescents
• Manufacturing PA Innovation Program Awards Wound Dressing Research
• Surgeon-in-Chief Dr. N. Scott Adzick Wins 2024 Robert E. Gross Award
• CHOP Attending Physician Shares Cytokine IL-18 Research

Keep up with our news, stories, and updates in real time by following us on X, Facebook, LinkedIn, or Instagram. Meet the minds behind the science in the Bench to Bedside podcast. Or subscribe to our newsletter to receive an email every other Friday.