Myasthenia gravis future or investigational therapies

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Associate Editor(s)-in-Chief: Hafiz M. Ahmed, M.D.[2]

Overview

Several therapies for myasthenia gravis (MG) remain under active investigation, and important questions persist regarding the optimal use of recently introduced agents. Areas of ongoing study include complement inhibition, targeted B-cell therapies, and the role of newer immunotherapies in disease subgroups and stages not yet well characterized.

Future Or Investigational Therapies

• Complement inhibition: Eculizumab, a monoclonal antibody against complement C5, is established for severe, refractory AChR antibody–positive generalized MG, but its longer-term role continues to evolve. Unresolved questions include the duration of therapy needed to reach and maintain treatment goals, efficacy in populations not studied in the pivotal trials (such as thymomatous and seronegative MG), and its potential value in additional disease stages, including myasthenic crisis, acute exacerbations, and earlier (non-refractory) AChR antibody–positive disease.
• B-cell–targeted therapy: Rituximab, an anti-CD20 monoclonal antibody, shows the most consistent benefit in MuSK antibody–positive disease, whereas its efficacy in refractory AChR antibody–positive MG remains uncertain. Optimal dosing regimens, timing, and patient selection are still being defined, and randomized data are limited.
• Steroid-sparing immunosuppressants: The comparative effectiveness of agents such as methotrexate relative to better-established steroid-sparing drugs remains incompletely defined, and high-quality randomized evidence is lacking.
• Other directions: Additional research priorities include the management of MG that arises as an immune-related adverse event of immune checkpoint inhibitors, and refinement of treatment strategies across the spectrum of disease severity, antibody status, and thymic pathology.