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mpact Report, FDA Approval, PRESERVE Town Hall, ‘Immature Neurons,’ Lung Disease

Published on August 5, 2022 in Cornerstone Blog · Last updated 4 months 1 week ago


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CHOP Research in the News

CHOP Research in the News

As we enter August – and another heat wave – stay cool in the air conditioning and catch up with In the News. In this week’s roundup, we highlight the PolicyLab 2022 Impact Report, an FDA drug approval based on CHOP research, and the PRESERVE study to improve outcomes in pediatric chronic kidney disease. Read on about a study showing that “immature neurons” develop throughout the lifespan, and find out how daycare affects premature children with chronic lung disease.

PolicyLab Releases 2022 Impact Report

David Rubin MD, MSCE

David Rubin MD, MSCE

PolicyLab has released its second annual Impact Report, which highlights how PolicyLab took an evidence-to-action approach to improve children’s health and well-being during the past year. The report highlights the work of PolicyLab team members and partners.

“Throughout this busy year, we all navigated shifting landscapes,” said David Rubin, MD, MSCE, director of PolicyLab. “Drawing on the strength of our model that pairs researchers with policy and communications experts, we forged forward with new research areas, strengthened our community partnerships, and provided local and state policy support across a range of topics.”

Research highlights in the report include study on supporting the transition from pediatric to adult care, an issue brief highlighting policy changes needed for a quality childcare system, a former PolicyLab student’s Fulbright Grant to study maternal health among migrant populations, and a spotlight on the PolicyLab Morning Speaker Series.

Read the full Policy Lab Impact Report.

FDA Approves Drug for Rare Cancer

Yael Mossé, MD

Yael Mossé, MD

The FDA approved crizotinib (Xalkori) for treatment of adults and children with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT). The approval was based on the results of two clinical trials, one of which was led by researchers at CHOP through the Children’s Oncology Group (COG). Principal investigator Yael P. Mossé, MD, attending physician in CHOP’s Cancer Center, and professor of Pediatrics at the University of Pennsylvania Perelman School of Medicine, has studied ALK alterations in several cancers including neuroblastoma.

“The approval of Xalkori for ALK-positive IMT will bring hope to many pediatric patients and families who previously had highly limited treatment options,” Dr. Mossé said. “It is very exciting to have approval for frontline unresectable IMTs. This is an important milestone for these patients, and for pediatric oncology researchers within COG and CHOP, who continue to seek out more rational therapies.”

Crizotinib is a first-generation ALK inhibitor that inhibits ROS1, a tyrosine kinase closely related to ALK. It is already approved for the treatment of patients with metastatic non-small cell lung cancer that is ALK- or ROS1-positive, and for the treatment of relapsed or refractory systemic anaplastic large cell lymphoma that is ALK-positive.

Find out more in the CHOP press release.

CHOP Hosts Town Hall for PRESERVE Project

CHOP recently hosted the Semi-Annual PRESERVE Town Hall, a virtual interactive session where participants discussed updates of the Preserving Kidney Function in Children with Chronic Kidney Disease (PRESERVE) study. Christopher Forrest, MD, PhD, director of the Center for Applied Clinical Research, leads PRESERVE, a research study involving 16 hospitals that are part of the National Patient-Centered Clinical Research Network and 19 parent and youth partners from across the United States. It’s aim is to improve outcomes for patients diagnosed with pediatric chronic kidney disease (CKD). Michelle Denburg, MD, MSCE, co-director of the Penn-CHOP Kidney Innovation Center and associate professor of Pediatrics and Epidemiology, joins Dr. Forrest as PRESERVE co-investigator.

Hypertension, a major and potentially modifiable contributor to loss of kidney function, affects 50% of children with CKD. PRESERVE focuses on the lived experiences of pediatric patients and their caregivers by evaluating the effectiveness of blood pressure monitoring and treatment strategies for pediatric CKD and providing new knowledge to inform shared decision making.

Penn/CHOP Study Finds Newly Formed ‘Immature’ Neurons Appear in Brain Throughout Life

In close collaboration with researchers at the University of Pennsylvania Perelman School of Medicine, scientists and clinicians from the Neuroscience Center at CHOP found that immature, plastic neurons are present in the brain’s hippocampus, a key memory region, throughout the human lifespan. The study, which appeared in the journal Nature, helps confirm the existence of “adult neurogenesis,” or the production of new immature neurons in the mature human brain. The findings help provide insight into how adult neurogenesis may be involved in disorders such as Alzheimer’s disease.

The researchers used single-nucleus RNA sequencing and machine learning to confirm the presence of immature hippocampal neurons, particularly a type called granule cells, across a wide range of human brain samples from infants to elderly adults. The analysis further uncovered a broad pattern of gene activity characteristic of immature granule cells and showed that the pattern is altered in Alzheimer’s disease.

CHOP researchers involved in the program include Benjamin C. Kennedy, MD, attending neurosurgeon and co-director of the Epilepsy Neurogenetics Initiative Frontier Program, Angela Viaene, MD, PhD, pediatric neuropathologist, Ingo Helbig, MD, attending neurologist, and Sudha K. Kessler, MD, MSCE, attending neurologist.

Read more in the CHOP press release.

Daycare Linked to Increased Complications in Preterm Children with Chronic Lung Disease

Sharon A. McGrath-Morrow
Sharon McGrath-Morrow, MD

Children who were born prematurely and had a form of chronic lung disease are at increased risk for lung complications if they attend daycare in the first three years of life, according to new findings from CHOP researchers. The study, which appeared in The Journal of Pediatrics, found that preterm children with bronchopulmonary dysplasia (BPD) who attend daycare were more likely to visit the emergency department, use systemic steroids, and have chronic respiratory symptoms. The researchers analyzed data from 341 former preterm infants with BPD between the ages of 0 and 3 years, including daycare attendance, clinical characteristics, acute care use, and chronic respiratory symptoms.

“Preterm children born with BPD have a short window to improve their lung function trajectory, so we are always looking for ways to minimize exposures in early childhood to prevent lung function problems in adult life,” said Sharon McGrath-Morrow, MD, MBA, associate chief of the Division of Pulmonary and Sleep Medicine and leader of the Post-preemie Lung Disease Clinic at CHOP. “This study shows that daycare is a modifiable risk factor that is linked with poorer outcomes in preterm children with BPD. Providers should advise families with infants and young children with BPD about the potential risks of daycare attendance, particularly prior to 1 year of age, while also bearing in mind the financial needs of the family.”

Read more about the study in the CHOP press release.


Catch up on our headlines from our July 22 In the News:

  • New Algorithm Distinguishes ADHD Symptoms from Related Conditions
  • Study Reveals Cellular and Molecular Mechanisms of Skull Formation
  • New AAP Policy Statement on Vision Disorders After Concussion

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