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White House Visit, Neonatal Resuscitation, Police Interactions with Black Autistic Youth

In this week's news roundup, Stephan Grupp, MD, PhD, joins cell and gene therapy experts and advocates at the White House, Children's Hospital of Philadelphia Research Institute provides funding and mentorship for promising early-career investigators, and researchers evaluate a novel strategy for safe and effective neonatal resuscitation.
Dr. Stephan Grupp Attends White House Cell and Gene Therapy Forum
Stephan Grupp, MD, PhD, Director of CHOP's Kelly Center for Cancer Immunotherapy and the Cancer Immunotherapy Program, Chief of the Cellular Therapy and Transplant Section, and Medical Director of the Cell Based Therapy Laboratory, joined thought leaders, researchers, patients, and industry partners invited by the White House Office of Science and Technology to attend a Cell and Gene Therapy Forum. The gathering at the White House Jan. 10 aimed to begin long-lasting collaborations to solve the biggest issues in the cell and gene therapy fields.
Dr. Grupp, who is also known for his role in treating Emily Whitehead, the first pediatric patient to receive chimeric antigen receptor (CAR) T-cell therapy, participated in discussions about reducing therapy development complexity, fostering sustainable funding, and expanding equitable access to cell and gene therapies.
Read a summary of the forum on LinkedIn
Early-career Investigators Receive CHOP Research Institute Support for Pilot Projects
CHOP Research Institute awarded five CHOP researchers with Clinical Fellow/Instructor K-Readiness Pilot Grants. These awards support individuals with clinical responsibilities who are trying to secure a National Institutes of Health K (Research Career Development) award and provide salary support for mentors who can assist the trainee in achieving their research objectives. The program favors innovative projects with potential to receive future federal funding.
Congratulations to the grant recipients:
Anand Bhagwat, MD, PhD, Attending Physician in the Cancer Center at CHOP
Mentor: Saar Gill, MD, PhD, Associate Professor of Medicine in the Division of Hematology-Oncology at the University of Pennsylvania
Pilot Project: Novel Mechanisms of Acute Myeloid Leukemia Resistance to Cell Therapy
Dr. Bhagwat's study aims to understand an unexpected mechanism by which acute myeloid leukemia (AML) cells become resistant to CAR T-cell therapy. With this knowledge, his goal is to develop a precision method to restore CAR T-cell efficacy and achieve better outcomes for pediatric patients with difficult-to-treat AML.
Haley Newman, MD, Attending Physician in the Cancer Center at CHOP
Mentor: David Teachey, MD, Co-leader, Immune Dysregulation Frontier Program
Pilot Project: Dr. Newman's project aims to determine the genetic ancestry impacts prognostic biology, drug sensitivity, and outcomes in T-cell acute lymphoblastic leukemia (T-ALL), the most common form of leukemia found in children and T-cell lymphoblastic lymphoma (T-LL), a type of non-Hodgkin lymphoma in children. This pilot project will enhance understanding of T-ALL and T-LL biology, which is critical to guide risk-stratification and targeted treatments to prevent more children from relapsing.
Kemar Prussien, PhD, Clinical Psychologist at CHOP and Assistant Professor of psychiatry at Penn
Mentor: Alexis Thompson, MD, MPH, Chief, Division of Hematology
Pilot Project: Structural and Interpersonal Racism and Health Disparities in Sickle Cell Disease: A Biobehavioral Investigation
This study aims to enhance the understanding of how structural and interpersonal racism impacts biobehavioral stress responses among adolescents and young adults with sickle cell disease. Dr. Prussien noted it is essential to identify the underlying physiological and behavioral mechanisms of health disparities to determine intervention targets and evaluate the effects of future multilevel interventions and policy change.
Hilary Whitworth, MD, MSCE, Attending Hematologist in the Division of Hematology and the Hemostasis and Thrombosis Center at CHOP
Mentor: Leslie Raffini, MD, MSCE, Director, Hemostasis and Thrombosis Center
Pilot Project: Predicting Recurrent Central Venous Catheter Associated Venous Thromboembolism
Central venous catheters are the most common risk factor for pediatric venous thromboembolism, with children who have one catheter-associated clot at risk for recurrent thrombosis when another catheter is in place. This single center prospective cohort study aims to identify risk factors for recurrent catheter-associated thrombosis, including an assessment of novel biomarkers. With these data, Dr. Whitworth's goal is to develop risk-adapted thromboprophylaxis strategies to decrease recurrent thrombosis in children.
Daniel Zheng, MD, MHS, Attending Physician in the Cancer Center and faculty member in CHOP's PolicyLab
Mentors: Richard Aplenc, MD, PhD, MSCE, Assistant Vice President and Chief Clinical Research Officer at CHOP and Professor of Pediatrics and Epidemiology at Penn; Kelly Getz, PhD, MPH, Research Scientist at CHOP and Assistant Professor of Epidemiology at Penn
Pilot Project: Characterizing Financial Toxicity and Caregiver Work Disruption During Pediatric AML Treatment
This prospective, mixed methods study aims to utilize surveys and interviews to better understand the longitudinal experience of how treatment for pediatric AML, a rare blood cancer, affects caregivers' experience with work and finances.
Visit the Grants Hub to learn about about this and other CHOP Research Institute internal funding opportunities.
PCORI Funding Supports Investigation of Optimal Neonatal Resuscitation Strategies
The Patient-Centered Outcomes Research Institute (PCORI) awarded funding to Elizabeth Foglia, MD, MA, MSCE, an Attending Physician in the Division of Neonatology, to investigate optimal implementation strategies for neonatal resuscitation.
Ten percent of the 3.8 million babies born in the United States each year require resuscitation immediately after birth. Facemasks do not always provide effective resuscitation for newborns, and intubation is technically challenging for providers and poses risks for these patients.
Christopher Bonafide, MD, MSCE, Associate Chief of the Division of General Pediatrics and Co-associate Director for Implementation Science of Clinical Futures, a CHOP Research Institute Center of Emphasis, will co-lead the Supraglottic Airway for Resuscitation (SUGAR) Trial.
"We want to ensure healthcare providers are using evidence-based practices to support newborns, particularly at birth when so many critical health decisions need to be made quickly," said Dr. Foglia, who is also the principal investigator of the Delivery Room of the Future Frontier Program at CHOP.
The SUGAR trial will compare two implementation strategies to promote use of supraglottic airways, a group of devices that are inserted into the pharynx to enable ventilation without intubation during newborn resuscitation.
Learn more in the CHOP news release and this Research in Practice blog post.
Survey of Trochleoplasty Experts Provides Insights to Guide Practice and Future Research

Brendan Williams, MD
Researchers in the Sports Medicine Performance Center at CHOP shared results from the first international survey of trochleoplasty experts. Their findings could help guide current practice and future research related to this surgical procedure that is designed to correct the improper development of the femoral groove, which can lead to kneecap dislocation.
"Further understanding of appropriate indications for trochleoplasty and the safety of this procedure is critical," said Brendan Williams, MD, an Attending Pediatric Orthopedic Surgeon with the Orthopedic Center at CHOP. "Our findings shed light on important directions for future research."
Survey results from 32 trochleoplasty surgeons with an average of 14 years in practice indicated that variation exists in surgical indicators, technique, and postoperative rehabilitation practices. Specific differences between high- and low-volume trochleoplasty surgeons were noted. Overall, the researchers identified areas of similarity and variation in treatment that should direct future research related to trochleoplasty.
The Orthopaedic Journal of Sports Medicine published the results. Learn more in this CHOP news release.
Center for Autism Researchers Identify Opportunities to Improve Police Interactions with Black Autistic Youth
Researchers in the Center for Autism Research (CAR) at CHOP evaluated perceptions and concerns of 43 Black caregivers of Black autistic children about police interactions with their autistic children. The study highlights the importance of including racially and ethnically diverse individuals in research that explores policing and underscores the need for culturally responsive adaptations within police training efforts designed for autistic people.
"By making these families the focus of our study, we want them to know that their voices are being heard, and that what they tell us provides important next steps for implementing meaningful change," said Ashlee Yates Flanagan, PhD, Research Psychologist at CAR and lead study author.
The study analysis revealed four dominant themes: Caregiver concerns regarding the quality of police officers' training, children's normal autistic behaviors being inappropriately policed, the explicit threat of harm and murder, and the hope for more mindful policing. Study participants worried that police officers could over-react to movements and responses that are typical of autistic youth but might differ from their peers, such as reduced eye contact.
"Our findings suggest that there is a lot of room for growth in training for police officers, and that Black parents of Black autistic youth carry hope that outcomes can be improved," said senior author Julia Parish-Morris, PhD, a Senior Principal Scientist with CAR, and Associate Professor in Psychiatry at CHOP.
Their findings were published in the journal Autism. Learn more in this CHOP news release.
Nurse Researcher Received Presidential Award for Exceptional Leadership Potential
Catherine McDonald, PhD, RN, Pediatric Nurse Scientist and Senior Fellow in the Center for Injury Research and Prevention at CHOP, is among the recipients of the prestigious Presidential Early Career Award for Scientists and Engineers (PECASE). Established by President Clinton in 1996, PECASE recognizes scientists and engineers who show exceptional potential for leadership and is the highest honor bestowed by the U.S. government on outstanding scientists and engineers early in their careers.
"I am incredibly humbled by the honor," said Dr. McDonald, who is the Dr. Hidegarde Reynolds Endowed Term Chair of Primary Care Nursing and Professor of Nursing at Penn. "I am particularly proud that nursing science and adolescent health are brought to the forefront with this award. When children and adolescents can attain their highest level of health and wellness, their health outcomes as adults can be improved."
Dr. McDonald was nominated for her National Institute of Nursing Research-funded work in concussion and driving in adolescents, which examines driving behaviors of recovering concussed adolescents. Her long-term goal with this research is to establish the evidence base for return-to-drive recommendations for adolescents following a concussion.
Learn more in this news release.
ICYMI
Catch up on our headlines from our Jan. 10 In The News:
- Susan Furth Honored as 'Chief Innovation Officer to Know'
- Review Highlights Disparities in Early-onset Sepsis Research
- FUSED Award Winners Seek to Improve Patient Outcomes
- CHOP Scientist Featured in Washington Post for Kidney Stone Research
- Cell Line Database to Accelerate Brain Tumor Research
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