Abstract
Cell-based therapies are gaining momentum as promising treatments for rare neurological autoimmune diseases, including neuromyelitis optica spectrum disorders and myelin oligodendrocyte glycoprotein antibody-associated disease. The development of targeted cell therapies is hampered by the lack of adequate animal models that mirror the human disease. Most cell-based treatments, including HSCT, CAR-T cell, tolerogenic dendritic cell and mesenchymal stem cell treatment have entered early stage clinical trials or have been used as rescue treatment in treatment-refractory cases. The development of antigen-specific cell-based immunotherapies for autoimmune diseases is slowed down by the rarity of the diseases, the lack of surrogate outcomes and biomarkers that are able to predict long-term outcomes and/or therapy effectiveness as well as challenges in the manufacturing of cellular products. These challenges are likely to be overcome by future research.
Highlights
myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD) is increasingly regarded as a separate disease entity from AQP4-immunoglobuline G (IgG) positive (AQP4+) neuromyelitis optica spectrum disorders (NMOSD) [8,9]
MOGAD diseases are accompanied by relapses and episodes of remission that are variable in duration [14]
These pathological observations have to be contrasted with the fact that, to date, no myelin oligodendrocyte glycoprotein (MOG)-specific T cells have been found in the peripheral blood of patients with MOGAD [48]
Summary
Publisher’s Note: MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations. Various types of cell-based therapies may hold promise for treatment of potentially severe autoimmune neurological diseases, including neuromyelitis optica spectrum disorders (NMOSD) and myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD). It became clear that a proportion of these seronegative NMO patients carried autoantibodies towards another autoantigen, myelin oligodendrocyte glycoprotein (MOG) This disease is referred to as MOG antibody-associated disease or MOGAD [6]. More recently, reports of coexisting NMDAR antibodies [12] and concurrent peripheral and central demyelination with concomitant presence of anti-neurofascin antibodies [13] have challenged this concept Both NMOSD and MOGAD diseases are accompanied by relapses and episodes of remission that are variable in duration [14]. After providing an overview of the current knowledge on immunopathogenesis, the fundamental, translational and clinical research approaches in the field of cell-based therapies in NMOSD and MOGAD are reviewed and challenges and areas open to research are discussed
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