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In the News: CCMT Facility Honored, Autistic Drivers, Predicting EoE Milk Allergy, Minority Trainee Development Scholarship, PAS Preview

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April 30, 2021
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In the News

georges [at] chop.edu (By Sharlene George)

Whether it's building state-of-the art facilities or building thought leadership at prominent scientific meetings, Children's Hospital of Philadelphia researchers are always busy raising pediatric science to new levels. In this edition of In the News, find out why the Clinical Manufacturing Facility at CHOP impressed the International Society for Pharmaceutical Engineering. Gain insights about young autistic drivers from specialized driving instructors. Learn about a new approach to identify eosinophilic esophagitis milk allergy with accuracy and sensitivity. And see where our scientists will be exploring new ideas with the scientific community.

Center for Cellular and Molecular Therapeutics Honored by ISPE for Advancing Technology, Innovation

Congratulations to the leaders and staff at the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at Children's Hospital of Philadelphia, which the International Society for Pharmaceutical Engineering (ISPE) selected for a 2021 Facility of the Year Awards Honorable Mention.

The judging panel selected CCMT because of its impact on the future of cell and gene therapy facilities, noting that they were impressed with its synergy with the hospital, access to cutting edge medicines, effective use of space, and the social and sustainability value it offers Philadelphia and those receiving treatment. Established in 2005, currently under the leadership of Beverly Davidson, PhD, CCMT aims to lead research into gene therapy disease.

The CCMT's new state-of-the-art Clinical Manufacturing Facility is located in the Colket Translational Research Building. The facility is dedicated to the manufacture of adeno-associated and lentiviral vectors supporting clinical trials. The 13,000 square foot GMP facility offers the capability for a wide range of vectors and genes of interest — significant capability arises within a small footprint. The CCMT facility offers cell expansion, full cleanroom suites, fill finish, and support space. CCMT started clinical manufacturing in the new facility in January 2021, demonstrating a successful outcome of the project.

"Working in collaboration with architects and engineers since 2015, we have built a state-of-the-art GMP manufacturing facility that will help support the growing field of gene therapy not only locally, but nationally and internationally," said Johannes C. M. van der Loo, PhD, director of the Clinical Vector Core at CCMT.

The 2021 Facility of the Year Awards (FOYA) Category Winners will be formally recognized at the ISPE Facility of the Year Awards Banquet, held in conjunction with the 2021 ISPE Annual Meeting & Expo taking place Nov. 1-3, 2021.

Established in 2004, FOYA recognize state-of-the-art projects utilizing new, innovative technologies to improve the quality of products, reduce the cost of producing high-quality medicines, and demonstrate advances in project delivery. The FOYA program provides a platform for the pharmaceutical science and manufacturing industry to showcase its accomplishments in facility design, construction, and operation, while sharing the development of new applications of technology and cutting-edge approaches. Visit ISPE.org/FOYA for more information.

View a video announcement and press release for more details. Read more about the promise of gene therapy in a Q&A with Dr. van der Loo and Director of Quality Olga Zelenaia, PhD, of the CCMT's Clinical Vector Core.

Autistic Adolescents Learning to Drive Benefit From Individualized Training

Driving is an important part of leading an independent life and is one option for ensuring safe mobility for autistic adolescents and young adults. A collaborative study from the Center for Injury Research and Prevention (CIRP)and the Center for Autism Research (CAR) at CHOP identified clear strengths and a series of specific challenges autistic adolescents experience while learning to drive. Their insights stress the importance of providing specialized, scaffolded instruction where skills are taught one at a time, allowing students to develop mastery before adding new skills.

Researchers conducted in-depth interviews with 17 specialized driving instructors who were trained as occupational therapists, driving rehabilitation specialists, or licensed driving instructors and who had completed additional training related to teaching autistic individuals to drive. Instructors described specific behind-the-wheel challenges among young autistic drivers, including being overly rule-bound, becoming easily distracted, and having difficulty integrating what other drivers are doing with their own hand-eye-foot coordination required to drive.

Observed strengths of young autistic drivers included carefully following the rules of the road, paying close attention to their driving environment, and limiting risk-taking.

"Through our interviews with specialized driving instructors who worked specifically with young autistic drivers, we learned about teaching strategies perceived to be effective and recommendations to improve the learning-to-drive process for these adolescents and young adults," said Rachel K. Myers, PhD, lead author of the study and a scientist at CIRP. "Rigorous, individualized training is needed for their behind-the-wheel instruction. More research is needed to standardize best practices for autistic adolescent driver instruction."

The findings appeared in the American Journal of Occupational Therapy. Read more details in the CHOP press release.

Minimally Invasive Assay Accurately Predicts Clinical EoE Milk Allergy

CHOP researchers found a simple assay using a blood sample could identify eosinophilic esophagitis (EoE) milk allergy with accuracy and sensitivity. The disease occurs when eosinophils, a type of white blood cell, accumulate in the esophagus causing pain and injury.

The researchers analyzed blood samples from 17 patients with EoE milk allergy, nine patients with IgE-mediated milk allergy (traditional food allergy), and 17 control patients who had no food allergy. The researchers found that patients with EoE milk allergy had elevated milk-dependent memory T helper cells, and that these cells produced more of a cytokine known as interleukin-4 that contributes to allergy. The elevation of these biomarkers occurred whether the patient had stopped consuming milk or was still actively consuming it.

This finding, if applied to a regularly used assay, could help determine not only what foods an individual should avoid, but also guide timing on reintroducing causal foods, since emerging evidence shows that some children can outgrow EoE after long periods of food avoidance. The test would involve a simple blood draw and could yield accurate results quickly.

"This study outlines a minimally invasive assay that accurately predicts clinical EoE milk allergy with high sensitivity and specificity," said senior author David A. Hill, MD, PhD, an attending physician with the Division of Allergy and Immunology at CHOP. "Future studies should determine whether similar assays could apply to other foods that cause EoE, which would allow for easy and accurate diagnosis and more effective treatment of this condition."

The findings appeared in Allergy. Read more details in the CHOP press release. And find out more about Dr. Hill's research in a Cornerstone faculty spotlight.

Nurse Scientist Receives ATS 2021 Minority Trainee Development Scholarship

Way to go, Mallory Perry, PhD, RN! Dr. Perry is a nurse scientist and a participant in the Postdoctoral Fellowship for Academic Diversity program at CHOP's Research Institute who received the American Thoracic Society (ATS) Conference 2021 - Minority Trainee Development Scholarship (MTDS). The ATS created the scholarship to increase representation of underrepresented racial and ethnic groups in pulmonary, critical care, and sleep medicine research. MTDS awardees receive a certificate of achievement, free registration to the ATS 2021 International Conference, and one free year of ATS membership. They also will be honored during the Diversity Forum at ATS 2021.

Learn more about Dr. Perry in a Q&A written for Diversity Month.

See You at the Virtual Pediatric Academic Societies Meeting

Stop by CHOP's virtual booth for updates on our research at the 2021 Virtual Pediatric Academic Societies (PAS) Meeting being held April 30 to June 4. PAS offers opportunities for a global audience of physician-scientists, clinicians, and educators to share research, explore new ideas, build career opportunities, and collaborate on future projects. CHOP's Division of Neonatology is an event sponsor. Visit the PAS online program guide to find CHOP speakers and their presentation topics. And see how sharp your diagnostic skills are in the PAS Make the Diagnosis Raffle.

ICYMI

Catch up on our headlines from our April 16 In the News:

  • CHOP Cancer Researchers Win 2021 AACR Team Science Award
  • Chief of Neurosurgery Teams Up With M. Night Shyamalan to Fund CHOP Research
  • EVAN Foundation Pledges Continued Support to Neuroblastoma Research Scholars Program
  • Michael Nance, MD, Elected to Penn Academy of Master Clinicians
  • Scott Adzick, MD, Receives Clinical and Translational Science Distinguished Investigator Award
  • Flaura Winston, MD, PhD, Receives Clinical and Translational Science Distinguished Investigator Award

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