Vision Loss, New Division Chiefs, Allergies and Anxiety, SARS-CoV-2 Surveillance

Starting off this week's roundup of research news highlights is a new study evaluating a tool to measure progressive vision loss in neurofibromatosis type 1. Next up is a U.S. Food and Drug Administration approval that's on the horizon based on CHOP research for a debilitating disease. Then meet our new Ophthalmology Division Chief and Hematologic Malignancies Section Chief. Finish up with a study on anxiety and children with food allergies, and the continuation of a genomic surveillance study of SARS-CoV-2.

Research Collaborators to Validate a New Tool to Measure Progressive Vision Loss

CHOP researchers are teaming up with the Children's Oncology Group (COG) to investigate whether optical coherence tomography (OCT) is an effective tool to measure vision changes in patients with neurofibromatosis type 1 (NF1).

Robert Avery, DO, MSCE, pediatric neuro-ophthalmologist at CHOP, and Michael Fisher, MD, director of the Neurofibromatosis Program, will lead the study. It is an exploratory aim of an ongoing COG phase 3 trial investigating selumetinib for the treatment of patients with NF1-associated low grade glioma. Drs. Avery and Fisher will test the validity of OCT as a tool to objectively assess the visual system and its response to treatment in NF1 patients with optic pathway gliomas.

Building on previous research led by Dr. Avery which sought to characterize biomarkers to guide treatment decisions for optic pathway gliomas, the clinical study will collect three sets of OCT measurements — pre-treatment and after 6 and 12 months of treatment — for 60 patients with NF1-associated optic pathway gliomas who are enrolled in the phase 3 trial. Drs. Avery and Fisher will determine whether OCT measurements can predict treatment response prior to treatment initiation or provide early indication of treatments that are not working.

The Gilbert Family Foundation, a private nonprofit foundation founded by Jennifer and Dan Gilbert to accelerate a cure for NF1, will providing funding support for the study.

Learn more about the study in the CHOP press release.

Aggressive Pediatric Bone Disease Treatment Nears FDA Approval

The FDA accepted a new drug application for a drug identified by researchers at CHOP to treat fibrodysplasia ossificans progressive (FOP). The drug, palovarotene, is currently in a phase 3 trial, and it would be the first-ever approved treatment for FOP. The disease involves a process called heterotopic ossification (HO), which causes patients with FOP to form cartilage and bone at diverse sites outside the skeleton.

Maurizio Pacifici, PhD, director of Orthopaedic Research at CHOP, lead the research that found palovarotene, a retinoid agonist drug, inhibited HO formation in an animal model of FOP. In subsequent studies, Dr. Pacifici and his team found the drug also blocked HO in animal models that carried the exact human mutation that causes FOP.

"The dream of every biomedical researcher is to go from the lab to the bedside and discover a treatment that improves the lives of patients," Dr. Pacifici said. "We are very excited that the FDA will review this drug for final approval, as the patients currently lack an effective treatment that will slow down or block their debilitating disease."

Learn more about the research and FOP in the CHOP press release.

Gil Binenbaum, MD, MSCE, Appointed New Ophthalmology Division Chief

CHOP appointed Gil Binenbaum, MD, MSCE as chief of the Division of Ophthalmology. Dr. Binenbaum has been an attending surgeon at CHOP since 2007 and is also an associate professor of Ophthalmology in the Perelman School of Medicine at the University of Pennsylvania. Dr. Binenbaum is an active clinical researcher who is widely recognized for his expertise in retinopathy of prematurity (ROP); consultative pediatric ophthalmology, including ocular signs of child abuse; and complex pediatric and adult strabismus.

Dr. Binenbaum holds the Richard Safritz Endowed Chair in Ophthalmology Research and is the divisional director of research at CHOP. He is the chair of a 45-hospital collaborative ROP research group and has served as chair of the Research Committee of the American Association for Pediatric Ophthalmology and Strabismus (AAPOS). He has authored more than 300 peer-reviewed publications, scientific abstracts, chapters and editorials, and he has had continuous NIH research funding support since the beginning of his tenure at CHOP.

"We are delighted to continue working with Dr. Binenbaum to help advance care for patients within our Division of Ophthalmology," said N. Scott Adzick, MD, surgeon-in-chief at CHOP, and the founder and director of the Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment. "Dr. Binenbaum is a highly accomplished clinician, educator and scholar who has outstanding leadership abilities and will undoubtedly guide the Division of Ophthalmology to new heights."

Read more in the CHOP press release.

Sarah Tasian, MD, Appointed Chief of Section of Hematologic Malignancies

The Division of Oncology at CHOP appointed Sara Tasian, MD, as chief of the Section of Hematologic Malignancies. In this new role, Dr. Tasian will oversee a dynamic group of faculty, instructors, nurse practitioners, nurses, social workers, and clinical and laboratory research staff within the Cancer Center at CHOP.

Dr. Tasian, also an associate professor of Pediatrics in the Perelman School of Medicine at the University of Pennsylvania, is a physician-scientist at CHOP whose primary area of interest is the development of molecularly targeted therapeutics for children with high-risk leukemias. Her bench-to-bedside and bedside-back-to-bench translational laboratory research program focuses upon testing of kinase inhibitors and chimeric antigen receptor (CAR) T cell immunotherapies in high-risk genetic subsets of childhood acute lymphoblastic leukemi and acute myelogenous leukemia. She leads or co-leads several national or international early phase clinical trials of precision medicine therapies in children with high-risk leukemias.

"[Dr. Tasian] is uniquely suited for this new role, and, as a current CHOP faculty member, has established herself as a world leader in basic, translational, and clinical research in leukemia, with a focus on developing new precision medicine therapies and immunotherapies for high-risk and relapsed acute leukemias," said Stephen Hunger, MD, chief of the Division of Oncology. "She holds multiple NIH grants, is an outstanding teacher and educator, and a consummate physician."

Learn more in the CHOP press release.

Screening Tool to Measure Anxiety in Children with Food Allergies

Researchers at CHOP developed a tool to help assess anxiety among children with food allergies and their families. The tool, called the Scale of Food Allergy Anxiety (SOFAA), was developed in partnership by medical professionals in CHOP's Food Allergy Center and a cognitive-behavioral psychologist specializing in pediatric anxiety, in consultation with parents of children with food allergies.

SOFAA includes two separate questionnaires: the SOFAA-C for children and the SOFAA-P for parents or caregivers. Each version consists of 21 items to assess food allergy-related anxiety that are scored on a 5-point rating scale. The researchers tested the validity and reliability of the new scale on 77 parent-child pairs and found strong agreement between answers by children and their parents. The findings appeared in The Journal of Allergy and Clinical Immunology: In Practice.

"We hope that with further validation and research, the SOFAA can be used to determine the prevalence of excessive anxiety in children with food allergies and provide guidance on managing that anxiety when flare-ups occur," said study co-author Megan O. Lewis, MSN, CRNP, program manager of the Food Allergy Bravery Clinic at CHOP. "Researchers who study quality of life in children with food allergies now have a more precise tool at their disposal, as we have chosen to make the SOFAA free to use and easily accessible online."

Read more about the study in the CHOP press release.

Planet Lab Receives CDC Award to Continue SARS-CoV-2 Genomic Surveillance

The Centers for Disease Control and Prevention announced 29 awards as part of the SARS-CoV-2 Sequencing for Public Health Emergency Response, Epidemiology, and Surveillance (SPHERES) Initiative. The SPHERES collaboration aims to accelerate the application of SARS-CoV-2 genome sequencing for genomic epidemiology and pandemic response.

One of the award recipients is Paul Planet, MD, PhD, attending physician in the Division of Infectious Diseases at CHOP, who will be using the funds to continue his research on SARS-CoV-2 diversity for the next two years. The research has four aims, according to Dr. Planet: perform surveillance of new variants and model spread around Philadelphia; test whether super-spreader events are more likely with specific variants; test whether severe disease, MIS-C, and vaccine breakthrough are associated with specific variants; and develop the team's bioinformatic tool, GNUVID, for classifying variants, and make the tool widely available.

Learn more about and Dr. Planet's research in this Q&A on Cornerstone.

ICYMI

Catch up on our headlines from our July 9 In the News:

• ADHD Medications May Reduce Suicide Risk in Children With Behavioral Symptoms
• Humanized CAR T-Cell Therapy Shows Potential for Relapsed B-ALL
• How Has the COVID-19 Pandemic Affected Childhood Obesity?
• Researchers Elucidate How Malaria Parasites Survive Temperature Shifts
• Philadelphia County Medical Society Honors N. Scott Adzick, MD

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