Myasthenia Gravis: Current and Emerging Treatment Options

James Howard, MD, Neurologist at University of North Carolina at Chapel Hill, discusses the current and emerging treatment options of myasthenia gravis.

Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control:

The eye and eyelid
Facial expressions
Chewing
Talking
Swallowing

Weakness tends to increase during periods of activity and improve after periods of rest. The condition results from a defect in the transmission of nerve impulses to muscles. This is due to the presence of auto-antibodies against acetylcholine receptors.

Traditionally, myasthenia gravis has been treated with immunosuppressants and corticosteroids. In recent years, targeted therapies have been developed to address the pathophysiology of myasthenia gravis. As Dr. Howard explains, these treatment options lead to reduced adverse event profiles, faster onset of action, and an improvement to the quality of life of these patients.

Zilucoplan is the first self-administered, once-daily, subcutaneous, targeted C5 complement inhibitor for myasthenia gravis. The treatment was approved following the results of the phase 3 RAISE study where significant improvements in efficacy outcomes compared to placebo were observed.

Dr. Howard also discusses CAR-T cell therapies, which have been successful in the oncology space. Recent preliminary data has also shown their effectiveness in lupus and myasthenia gravis, suggesting that further research may lead to better treatment options for those with rare autoimmune diseases.

Chapters:
00:00 Intro
1:22 Current Myasthenia Gravis Landscape
2:29 Clinical Trial For Subcutaneous Zilucoplan
5:49 The Future of Autoimmune Disease Treatments