Henry J. Kaminski, MD, Professor of Neurology at The George Washington University, Lead of the Myasthenia Gravis Rare Disease Network (MGNET), explains “Myasthenia Gravis: The Future Is Here,” an article discussing advancements in the treatment of myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. The condition results from a transmission defect of nerve impulses to muscles, due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known. Weakness tends to increase during periods of activity and improve after periods of rest. Common symptoms include weakness of the muscles that control:
- Eye and eyelid
- Facial expressions
- Chewing
- Talking
- Swallowing
Recently, there has been a surge in innovative treatment options for MG. This is the result of a better understanding of the immunopathogenesis, quick progress in drug development, and financial incentives for rare disease research.
Managing Myasthenia Gravis
The paper highlights the transition from traditional therapies, like acetylcholinesterase inhibitors and corticosteroids, to more targeted approaches such as monoclonal antibodies and complement inhibitors. Recent clinical trials emphasize personalized medicine, focusing on specific pathogenic mechanisms.
Future MG treatments aim to achieve better outcomes with fewer side effects by targeting immune responses more precisely. The need for these advancements is urgent, given the high costs and variable effectiveness of current treatments. However, emerging research offers hope for improved quality of life and long-term remission for MG patients.
Additionally, Dr. Kaminski discusses thymectomies, a surgical treatment for early-onset AChR antibody-positive MG, that provides sustained benefits for up to five years. However, around 25% of patients do not respond well and continue to need medications like prednisone and immunosuppressives.
A recent study suggested thymectomy in adults might increase the risk of cancer and autoimmunity, resulting in patient weariness of the treatment. The exact mechanism of benefit from thymectomy is unclear, but it likely reduces antigenic stimulation, thereby lowering AChR antibody production, although some autoreactive cells may persist and continue to produce harmful antibodies. Dr. Kaminski stresses the importance of this treatment and the benefit it can have for patients. To read the rebuttal of this study, visit https://www.nejm.org/doi/full/10.1056/NEJMc2310640.
To learn more about MG and other rare musculoskeletal disorders, visit https://checkrare.com/diseases/musculoskeletal-diseases/
