Patients are currently being enrolled in the first clinical trial to investigate the efficacy of immunological therapy for chronic pulmonary sarcoidosis. The trial is being conducted by researchers at the Icahn School of Medicine at Mount Sinai.
Mount Sinai has the largest Sarcoidosis Service in the world and is one of only two institutions in the country participating in the trial; the other is the University of Cincinnati. Mount Sinai is a National Institutes of Health Center of Excellence for research in sarcoidosis.
"The current standard treatment for chronic pulmonary sarcoidosis is corticosteroids," said Adam Morgenthau, MD, principal investigator of the study and Director of the Sarcoidosis Clinic and the Alvin S. Teirstein Sarcoidosis Support Group at Mount Sinai. "Many patients don't respond to these drugs and those who do often develop long-term complications. We are hopeful this study will lead to new treatments to improve lung function and quality of life."
Sarcoidosis is a rare inflammatory disease that can affect any organ but most commonly involves the lungs. Patients with pulmonary sarcoidosis typically exhibit symptoms of shortness of breath, cough and/or wheeze. It affects men and women of all ages and races worldwide. However, it occurs mostly in people ages 20 to 40, African Americans, especially women, and people of Asian, German, Irish, Puerto Rican and Scandinavian origin, according to the National Institutes of Health.
The randomized placebo Phase II clinical trial at Mount Sinai for patients with sarcoidosis is designed to assess the safety, tolerability and efficacy of an antibody directed against macrophage colony-stimulating factor (m-CSF), a protein associated with the development of sarcoidosis. During a Phase I clinical trial for patients with rheumatoid arthritis (RA), the antibody was well tolerated in healthy people as well as patients with RA.
"This study has both clinical and basic science applications," said Dr. Morgenthau. "In addition to determining whether patients' symptoms improve with this treatment, we will examine cell signaling pathways and immune responses in the trial participants which will help us better understand the biology of sarcoidosis and ultimately lead to the development of therapies that target the immune response."