Five years ago, David C. Fajgenbaum, both a Penn Medicine researcher and patient, tried an experimental treatment based on his laboratory research findings in the hopes of saving his own life. He has been in remission ever since. Now his research is shedding new light on why it worked, paving the way for further testing of a new treatment approach in Castleman disease, a rare and deadly condition with limited options for patients.
The work is led by Fajgenbaum, who is both the director of the Center for Study & Treatment of Castleman’s & Inflammatory Lymphadenopathies (CSTL) in the Perelman School of Medicine, as well as Patient 1 in the study. The findings show patients who do not respond to the only drug currently approved by the U.S. Food and Drug Administration for the treatment of the disease may have another option that targets a specific pathway called PI3K/Akt/mTOR. The research is published in the Journal of Clinical Investigation.
Castleman disease isn’t actually a single disease. The term describes a group of inflammatory disorders that share a common appearance under the microscope. It’s diagnosed in about 5,000 people of all ages each year in the United States, which makes it roughly as common as Lou Gehrig’s disease, also called ALS. Patients experience a range of symptoms—from a single abnormal lymph node with mild flu-like symptoms to abnormal lymph nodes located throughout their entire body, abnormal blood cell counts, and life-threatening failure of multiple organ systems, such as the kidneys, liver, heart, and lungs.
“Patients who don’t respond to siltuximab have limited options. They typically receive chemotherapy but often relapse,” says Fajgenbaum, who is also an assistant professor of medicine in the division of Translational Medicine & Human Genetics at Penn and executive director of the Castleman Disease Collaborative Network. The study’s senior authors are Thomas S. Uldrick, the deputy head of Global Oncology at Fred Hutchinson Cancer Research Center who was also Fajgenbaum’s treating physician while practicing at the National Institutes of Health, and Frits van Rhee, the clinical director of the Myeloma Center at the University of Arkansas for Medical Sciences.
Read more at Penn Medicine News.
