– Investigators in the Immune Tolerance Network (ITN), an international consortium supported by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, have made a major advance in treating anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis, which is a rare but very serious disease that causes inflammation of the blood vessels.
In a multi-center, national study published in the New England Journal of Medicine, (NEJM2010:363:221-32) investigators found that a new treatment strategy – rituximab (marketed under the brand name Rituxan ®) in combination with glucocorticoids – is equally as effective with fewer treatments than the current standard of care (cyclophosphamide with glucocorticoids) and thereby lowers the risk of toxicity. This is the first new treatment for ANCA-associated vasculitis in more than 40 years. Based on this research, the U.S. Food and Drug Administration (FDA) recently approved an expanded indication for rituximab to include treatment of this form of vasculitis. Rituxan® has been FDA-approved since 1997 as a treatment for patients with non-Hodgkin's lymphoma, chronic lymphocytic leukemia, and rheumatoid arthritis.
The study was led by John Stone, M.D., M.P.H., of Massachusetts General Hospital, Boston, and Ulrich Specks, M.D., of the Mayo Clinic, Rochester, Minn., and conducted by the ITN. Rho, supported by NIAID, served as the Statistical and Data Coordinating Center (SDCC) for the study, providing safety monitoring, data management, and statistical data analysis for the trial.
Ronald Helms, Ph.D., Rho’s Chief Scientific Officer and Professor Emeritus at UNC in Chapel Hill, described the importance of this research. “Unfortunately, rare diseases like ANCA-associated vasculitis are often overlooked in pharmaceutical research,” Dr. Helms said. “As a result, patients with these rare diseases often miss out on the benefits of cutting-edge scientific and medical research. With ANCA-associated vasculitis, the current standard of care has not changed in over 40 years. That is why it is so rewarding to be a part of new research that improves the way these rare diseases are treated, and hopefully, improves the quality of life for people who suffer from them. While this new therapy may seem unimportant in a broad economic sense, it is extremely important to every individual vasculitis patient.”
David Iklé, Ph.D., is the senior statistician at Rho responsible for coordinating much of the biostatistics and data management on the trial. He has more than 25 years experience in the design and analysis of clinical trials and is especially experienced in autoimmune diseases. According to Dr. Iklé, “this trial has been challenging in its complexity but rewarding in its outcome. It is especially pleasing to be able to contribute significantly to the very large team effort that made this trial possible and has now produced a new treatment that is both safe and of great benefit to patients with this rare and debilitating disease.”
As the SDCC for the ITN, Rho played a central role in this important research. The research areas of interest for the ITN are quite broad and include organ transplantation, rheumatoid arthritis, lupus, multiple sclerosis, diabetes, allergies and asthma – among others. These are all immunologic-based diseases in which improvements in immune tolerance may lead to better treatments and hopefully cures. Rho provides support for patient safety, as well statistical analysis and management of the clinical data collected by the ITN researchers.
For more information about this research, please see press releases from FDA and NIAID.