Michael R Yeaman
Background: Neuromyelitis optica spectrum disorder (NMOSD) is a rare autoimmune disease of the central nervous system that manifests via inflammation and demyelination typically targeting optic nerves & spinal cord. Patients often experience a relapsing course of cumulative neurological debility. Latest epidemiologic data suggest hundreds of thousands of cases worldwide, many of which remain undiagnosed and ineffectively treated. Despite its potential for devastating and irreversible neurologic disabilities, no treatment had been proven safe and effective in prospective, masked and adequately powered clinical trials in over a century. Key Advances: Integration of innovative molecular and cellular immunology, epidemiology, clinical characterization & treatment effects clearly differentiate NMOSD from multiple sclerosis (MS). Once viewed as solely B-cell driven, autoreactive T cells and loss of immune tolerance to aquaporin-4 or other autoantigens are now recognized as crucial to NMOSD immunopathogenesis. Four phase IIb/III clinical trials have now reported positive findings in evaluating three drugs (eculizumab, inebilizumab, satralizumab) for safety and efficacy to delay or prevent relapses in NMOSD. In 2019, the first of these drugs received regulatory approval having achieved >98% relapse-free efficacy among patients on drug in the trial period. The other agents are now in regulatory review. Prospectus: In just a single decade, The Guthy-Jackson Charitable Foundation has led a phenomenal story of research innovation, patient advocacy and industry synergy to revolutionize the field and provide a promising future for NMOSD patients. Significant next steps include identifying predictive biomarkers of relapse and restoring immune tolerance for cures to spare patients from cancer and infectious disease risk of lifelong immunosuppressive therapy. Preventative research areas include etiology, genetic or environmental risk factors & epidemiologic correlates of disease course or severity. Scientific and therapeutic breakthroughs in NMOSD will benefit patients with other autoimmune diseases.
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