An international, phase 3 clinical trial led by investigators at Mass General Brigham could improve the treatment of a rare disease that can cause debilitating symptoms. The study, published in the New England Journal of Medicine , found that treatment with inebilizumab greatly reduced the symptoms of immunoglobulin G4–related disease (IgG4-RD), compared to placebo. This is a huge day in the history of this disease.
We are thrilled to have worked so closely with patients to undertake a trial specifically focused on their disease, with the goal of finding a therapy that we hope will be approved shortly for them." John Stone, MD, MPH, lead author, rheumatologist in the Division of Rheumatology, Allergy, and Immunology at Massachusetts General Hospital, a founding member of the Mass General Brigham healthcare system IgG4-RD is estimated to affect fewer than 200,000 people in the United States. Although IgG4-RD is an orphan disease first recognized to be a unique condition in 2003, review of the medical literature as far back as the late 1800s confirms that the disease was around even then – and likely much longer.
People who have the condition suffer from a build-up of immune cells that produce the IgG4 antibody in certain organs. The disease can affect any organ and frequently involves multiple organs, most commonly the pancreas, bile ducts, salivary glands, abdominal tissue, eyes and lungs. The symptoms are driven by which organs are affected.
For example, if the disease a.