Did You Know? Research Recap — Annual Report 2023

Xilma Ortiz-Gonzalez, MD, PhD, attending physician and assistant professor of Neurology:

Did you know?

CHOP research paved the way for the first approved drug for Friedreich’s ataxia, a major event not only for FA, but also for all ataxias and the rare disease community.

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CHOP and its Neuromuscular Program served as one of the clinical trial sites for the first FDA-approved gene therapy for Duchenne Muscular Dystrophy. The approval marks the seventh cell and gene therapy offered to patients at CHOP. And we continue to be a site for more than 30 gene therapy clinical trials — with more on the horizon.

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Decades-long research informed the implementation of the Virtual Driving Assessment program in CHOP’s Care Network. This innovative, first-of-its-kind program provides teens with a comprehensive evaluation of real-world driving skills in a safe, controlled environment.

Yi Xing, PhD, executive director of the Department of Biomedical and Health Informatics:

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Researchers at CHOP and the University of California developed a new computational tool that expands the pool of cancer immunotherapy targets.

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CHOP researchers make ESPRESSO. It’s an acronym for a new, open-source computational tool that can more accurately discover and quantify RNA molecules from error-prone long-read RNA sequencing data.

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Researchers from CHOP and Drexel University used a novel technique to analyze 53 million patient notes from more than 1.5 million individual patients. They identified similarities in their medical histories. This can help pinpoint potential risks for developing future diseases and the trajectory of those conditions.

Sarah Tasian, MD, attending physician in the Division of Oncology and chief of the Hematologic Malignancies Program:

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CHOP received its first grant from the Bill & Melinda Gates Foundation. The multimillion-dollar funding will help propel critical mitochondrial research that focuses on the importance of mitochondrial DNA in the severity of COVID-19 cases.

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Philanthropy empowers our strategic investments with the potential to transform children’s health. In fiscal year 2023, major gifts helped to establish the Topoleswski Pediatric Heart Valve Center, the Center for Epilepsy and Neurodevelopmental Disorders, and advanced critical research through the Neurofibromatosis Center.

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The CHOP Cancer Immunotherapy program treated its 500th patient with CAR T-cell therapy this year. In addition, as many as 25,000 patients across the globe have received CAR T-cell immunotherapy more safely because of toxicity management protocols that were developed at CHOP.

Elizabeth Foglia, MD, MA, MSCE, attending physician in the Division of Neonatology and leader of the Delivery Room of the Future Frontier Program:

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CHOP researchers are also thought leaders who informed international guidelines for neonatal resuscitation studies, a rare neurological disorder known as MOGAD, and concussion and diagnosis treatment.

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CHOP researchers are focused on informing effective interventions to keep children safe from unsecured firearms. 1,650 gun cable locks were distributed through CHOP in fiscal year 2023.

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“Clinical Futures” is the name of a Center of Emphasis that was selected to participate in a new funding opportunity by the Patient-Centered Outcomes Research Institute to promote the uptake of useful research results in practice.

Wendy Reed Williams, PhD, senior director of Academic Training and Outreach Programs:

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This summer, almost 60 high school students and undergrads joined us on campus as part of CRISSP and CHOP-RISES, research-centric outreach programs that aim to inspire future leaders in pediatric healthcare.

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CHOP and the University of Pennsylvania jointly launched the Penn-CHOP Kidney Innovation Center and hosted its inaugural Kidney Life Course Research Symposium to promote collaboration and grow local talent.

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CHOP funded five new frontier programs in fiscal year 2023 — trailblazing initiatives that conduct visionary research, which translates to cutting-edge clinical care.

Richard Aplenc, MD, PhD, MSCE, assistant vice president and chief clinical research officer:

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7,577 people participated in clinical research studies at CHOP during fiscal year 2023. And we’re launching a new CHOP Biobank that will accelerate basic and translational research across the enterprise, ultimately helping fuel diagnostic and treatment breakthroughs.

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The new Schuylkill Avenue Research Building, which opens in 2025, will be 14 stories high — including seven floors of wet bench space, and a conference room with a 250-person lecture hall.

Susan Furth, MD, PhD, Executive Vice President and Chief Scientific Officer:

Now you know a few of the ways that CHOP Research Institute has a powerful influence on discovering, developing, and delivering solutions that change children’s lives.

Hi, I’m Dr. Susan Furth, executive vice president and chief scientific officer of CHOP Research Institute, where we:

• Accelerate research for clinical integration
• Create leading-edge technologies
• Strengthen partnerships
• Cultivate a world-class academic medical research community
• And spread our thought leadership worldwide.

Our work has a tangible impact: We find novel ways to help children with rare and complex diseases and their families thrive.

With an unparalleled depth of talent and highly interdisciplinary collaborations, we’re laser-focused. We provide the right people, with the right resources, at the right time to come together for children’s healthier futures. That’s what we’ve done throughout a century of breakthroughs at CHOP Research Institute.

We connect. We innovate. We translate. We amplify.

That’s what we know.