Four Penn studies named among nation’s top clinical research advances

Four federally-funded studies led by faculty from the Perelman School of Medicine at the University of Pennsylvania were selected for this year’s Clinical Research Forum's Top 10 Clinical Research Achievement Awards. Each year, the organization names 10 studies which 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, MD, 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.”

Three additional studies honored as 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. 

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, MD, 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, MD, PhD, the Barry J. Gertz Professor for Translational Research in the Perelman School of Medicine and Rebecca Ahrens-Nicklas, MD, PhD, 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 it’s 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, MD, MSCE, FASCO, the Mariann T. and Robert J. MacDonald Professor in Breast Cancer Research, and Lewis Chodosh, MD, PhD, 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, PhD, 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 Yong Chen, PhD, a professor of Biostatistics and the director the Penn Computing, Inference and Learning (PennCIL) lab.

Additional Penn finalists

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:

“A Phase 3 Trial of Upadacitinib for Giant-Cell Arteritis”

Clinical trial results showed that a common medication already used for autoimmune diseases such as rheumatoid arthritis is effective for patients with the rare blood vessel disorder giant cell arteritis, leading to sustained remission for almost half of the study participants. The trial results were published in the New England Journal of Medicine and led by Peter A. Merkel, MD, MPH, chief of Rheumatology and director of the Penn Vasculitis Center.

“Enhanced CAR T Cell Therapy for Lymphoma after Previous Failure”

A next-generation “armored” CAR T cell therapy showed promising results in a small study of patients whose B-cell lymphomas continued to resist multiple rounds of other cancer treatments, including commercially available CAR T cell therapies. The study was published in the New England Journal of Medicine and led by Jakub Svoboda, MD, an associate professor of Hematology-Oncology, and Carl June, MD, the Richard W. Vague Professor in Immunotherapy.

“Impact of the MISSION Act on Quality and Outcomes of Major Cardiovascular Procedures Among Veterans”

The 2018 MISSION Act expanded opportunities for veterans to obtain care outside the VA and cut travel times for veterans needing major heart procedures, but it also raised risks, worsening rates of major adverse cardiovascular events within 30 days of procedure. The analysis was published in JAMA and led by Peter Groeneveld, MD, MS, a professor of Medicine and a Leonard Davis Institute of Health Economics Senior Fellow.

 

Editor’s note: Meagher serves as the vice chair of membership and member engagement on the CRF board. While she took part in the selection process of papers, Meagher recused herself from evaluating and voting on any of the Penn-affiliated papers. Arthur Rubenstein, MBBCh, who served as Dean of the Perelman School of Medicine and Executive Vice President of the University of Pennsylvania for the Health System from 2001 to 2011, is a CRF board member as well and also recused himself from evaluating or voting on papers with Penn ties.