PHILADELPHIA - David Fajgenbaum, MD, MBA, MSc, a research assistant professor of Medicine, division of Hematology/Oncology, in the Perelman School of Medicine at the University of Pennsylvania, has received the RARE Champion of Hope award for science.
The honor recognizes Fajgenbaum’s efforts in driving forward clinically meaningful research for Castleman disease as a researcher at Penn and through co-founding and serving as executive director of the Castleman Disease Collaborative Network (CDCN), a global network of physicians, researchers, patients, and loved ones dedicated to accelerating research and treatment for Castleman disease and supporting patients with the condition. The award was presented by Global Genes, a global rare disease patient advocacy organization.
Castleman disease describes a group of inflammatory disorders with symptoms that range from a single enlarged lymph node to life-threatening multiple organ failure. The disease involves the body’s immune system becoming activated and releasing inflammatory proteins that can shut down the liver, kidneys, and bone marrow. The disease has an incidence similar to ALS (Lou Gehrig’s disease), being diagnosed in about 6,500 to 7,700 patients of all ages each year in the US.
Fajgenbaum is a physician-scientist, advocate, and a patient. He was diagnosed with idiopathic multicentric Castleman disease (iMCD) in 2010. The multicentric subtype has a 65-percent-five-year survival rate, which is nearly as deadly as the average for all cancers combined. Treating the iMCD subtype has been challenging because it is so poorly understood. Since Fajgenbaum began spearheading research in 2012, he and his associates have:
- connected a global community of over 300 physicians and researchers through an online community and the three largest-ever Castleman disease meetings;
- published a new model of pathogenesis for iMCD, in the leading hematology journal, Blood;
- engaged over 3,000 patients and loved ones through online forums and in-person meetings;
- crowdsourced to establish an international research agenda with prioritized studies to uncover iMCD pathogenesis;
- recruited experts to launch four high-impact studies; and
- finalized plans for a global patient registry, which will help to improve patient care and accelerate research.
With the correct disease model in place, the CDCN is now focused on identifying what triggers the intense immune activation, which immune cells are activated, what cellular pathways are activated, and what existing or novel therapies may be effective for patients that do not respond to the only FDA-approved therapy.
CDCN is currently launching four studies and a $250,000 fundraising campaign. Arthur Rubenstein, MBBCh, Vera Krymskaya, PhD, MBA, Kojo Elenitoba-Johnston, MD, and Megan Lim, MD, PhD, among other Penn faculty members, are also actively engaged in Castleman disease research.
For these efforts, Fajgenbaum was recognized in Forbes magazine’s 2015 30 Under 30 list for health care. He has also recently accepted a position as associate director of patient impact for the Penn Orphan Disease Center.
Fajgenbaum and recipients in other categories were recognized at the 4th Annual RARE Tribute to Champions of Hope this month in Huntington Beach, Calif.
Penn Medicine is one of the world’s leading academic medical centers, dedicated to the related missions of medical education, biomedical research, excellence in patient care, and community service. The organization consists of the University of Pennsylvania Health System and Penn’s Raymond and Ruth Perelman School of Medicine, founded in 1765 as the nation’s first medical school.
The Perelman School of Medicine is consistently among the nation's top recipients of funding from the National Institutes of Health, with $550 million awarded in the 2022 fiscal year. Home to a proud history of “firsts” in medicine, Penn Medicine teams have pioneered discoveries and innovations that have shaped modern medicine, including recent breakthroughs such as CAR T cell therapy for cancer and the mRNA technology used in COVID-19 vaccines.
The University of Pennsylvania Health System’s patient care facilities stretch from the Susquehanna River in Pennsylvania to the New Jersey shore. These include the Hospital of the University of Pennsylvania, Penn Presbyterian Medical Center, Chester County Hospital, Lancaster General Health, Penn Medicine Princeton Health, and Pennsylvania Hospital—the nation’s first hospital, founded in 1751. Additional facilities and enterprises include Good Shepherd Penn Partners, Penn Medicine at Home, Lancaster Behavioral Health Hospital, and Princeton House Behavioral Health, among others.
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