The drug eculizumab, a synthetic antibody that inhibits the inflammatory response, significantly reduced the risk of relapse with neuromyelitis optica spectrum disorder (NMOSD). This rare but severe autoimmune inflammatory disorder can cause blindness, paralysis, and death. Mayo Clinic researchers and international collaborators report their findings in a randomized, double-blind, placebo-controlled study published in the New England Journal of Medicine. Their work also will be presented in the Emerging Science Platform session, part of the American Academy of Neurology's 71st Annual Meeting in Philadelphia May 4–10.
"This study offers hope to patients, since each attack in NMO can cause loss of visual or motor function," says Sean Pittock, M.D., a Mayo Clinic neurologist and first author. "Stopping attacks can prevent disability and allow patients to maintain function and a better quality of life."
Mayo Clinic is a recognized center of excellence for diagnosing and treating neuromyelitis optica. In 2002, Mayo Clinic researchers led by Claudia Lucchinetti, M.D., and colleagues described the unique pathological features of NMO and proposed that NMO is an autoimmune disease caused by one or more harmful antibodies. In 2004, Vanda Lennon, M.D., Ph.D., Brian Weinshenker, M.D., and Mayo colleagues reported the discovery of the AQP4-IgG biomarker, a blood test that differentiates neuromyelitis optica from MS and similar disorders. The AQP4-IgG antibody was subsequently shown to be able to cause nerve cell damage consistent with what Dr. Lucchinetti and colleagues reported, and AQP4-IgG is now widely considered as the cause of NMO.
"The Mayo Clinic team subsequently demonstrated that when the antibody binds to the water channel AQP4 on nerve cells, a substance called 'complement' is activated and kills the cells, causing significant injury. We thought that if we could block complement activation, then perhaps we could prevent attacks," says Dr. Pittock, director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and Mayo’s Neuroimmunology Laboratory.
The news release can be found on the Mayo Clinic News Center.