Four federally-funded studies led by faculty from the Perelman School of Medicine at the University of Pennsylvania have been selected for Clinical Research Forum’s Top 10 Clinical Research Achievement Awards. Each year, the organization names 10 studies that best exemplify how the United States’ investment in research benefits Americans’ health and well-being. The winning Penn studies span some of the most impactful areas of modern medicine, probing the health impacts of important policy issues, harnessing new technologies to deliver highly personalized therapies, repurposing drugs to attack “sleeping” cancer cells, and examining the risks of long COVID.

“The Penn studies selected for Top 10 Clinical Research Achievement Awards offer a window into what federally supported science can achieve,” said Jonathan A. Epstein, Dean of the Perelman School of Medicine and Executive Vice President of the University of Pennsylvania for the Health System. “These discoveries are the result of a national commitment to advancing knowledge and improving human health. The discoveries and progress in this esteemed group are tangible examples of that funding in action, turning bold ideas into new therapies, new knowledge, and new hope for patients.” 

Many of these studies are the culmination of years of work, including decades of basic science research to lay the groundwork for first-in-human clinical trials. Overall, the winning Penn Medicine research was supported by more than $50 million in grants in recent years from the National Institutes of Health, Department of Defense, Department of Veterans Affairs, and the Agency for Healthcare Research and Quality, plus generous philanthropic donations and other funding support. The federal funding currently supports more than 50 researchers, trainees, and support staff who have devoted their careers to making progress in science and medicine.

“These studies underscore how deeply clinical research touches everyone’s lives,” said Emma Meagher, senior vice dean for clinical and translational research. “Whether serving veterans, older adults, or individuals facing rare diseases or common cancers, Penn Medicine is forging advances that improve health across the nation and permeate throughout the world.” 

The Penn-affiliated work honored this year is:

“Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease”: In a historic medical breakthrough, KJ Muldoon, an infant diagnosed with a rare genetic disorder, became the first person in the world successfully treated with a customized CRISPR gene editing therapy. The work, published in the New England Journal of Medicine, was led by Kiran Musunuru, the Barry J. Gertz Professor for Translational Research in the Perelman School of Medicine and Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Program (GTIMD) at Children’s Hospital of Philadelphia (CHOP).

“Targeting Dormant Tumor Cells to Prevent Recurrent Breast Cancer”: This first-of-its-kind, randomized Phase II clinical trial showed that it is possible to identify breast cancer survivors who are at higher risk of their cancer coming back due to the presence of dormant cancer cells and to effectively treat these cells with repurposed, existing drugs, offering proof-of-concept for a strategy to prevent breast cancer recurrence. The study, published in Nature Medicine, was led by Angela DeMichele, the Mariann T. and Robert J. MacDonald Professor in Breast Cancer Excellence, and Lewis Chodosh, chair of cancer biology.

“Loss of Subsidized Drug Coverage and Mortality Among Medicare Beneficiaries”: Losing Medicaid coverage—and with it, the Medicare Part D Low-Income Subsidy (LIS), which helps 14.2 million low-income Medicare beneficiaries afford their medications—is associated with significant increases in mortality, ranging from 4 to 22 percent. The analysis, published in the New England Journal of Medicine, was led by Eric T. Roberts, an associate professor of general internal medicine and a Leonard Davis Institute of Health Economics senior fellow.

“Long COVID Associated with SARS-CoV-2 Reinfection Among Children and Adolescents”: The more often someone is infected with COVID, the more likely they are to develop “long COVID,” as young people and children infected with the COVID virus for a second time were twice as likely to develop long COVID than their peers who were infected just once. The research, published in The Lancet Infectious Diseases, was led by Yong Chen, a professor of biostatistics and director of the Penn Computing, Inference and Learning (PennCIL) lab.

Three other Penn studies were included in the Clinical Research Forum’s list of finalists, which are akin to honorable mentions for the Top 10 list, rounding out the Top 20 clinical research papers of the year. These finalists included a Phase III clinical trial for a rare blood vessel disorder, a Phase I clinical trial investigating the next generation of cell therapy, and an analysis of the impacts of a health policy that expanded options for where veterans can receive care. 

With seven of the Top 20 research papers—selected from submissions drawn from nearly 60 research institutions and hospitals across the United States and around the world—Penn Medicine’s showing underscores its exceptional leadership in advancing clinical research.