Molecular Therapy in Myasthenia Gravis

Abstract

Myasthenia gravis (MG) is an autoimmune disorder caused by antibodies that act against the myoneural junction. Conventional immunosuppressants such as corticosteroids, azathioprine and mycophenolate are associated with long-term side effects and many patients do not achieve remission and may become refractory. Thus, there is an unmet need for target-specific therapies that act faster, have fewer side effects and lead to stable disease remission. However, many of the novel therapeutic agents being described are not meeting their primary endpoints. We reviewed the current status of novel immunotherapies for MG, their mechanisms of action, along with the side effect profiles. Fast onset of action, sustained disease remission and relatively low frequency of side effects of the new agents are attractive. However, the unknown long-term safety and high cost are precluding factors. Better preclinical studies and more randomized trials are needed before novel agents are routinely employed.