Henry Kaminski, MD, Professor of Medicine at The George Washington University ,provides a brief history lesson on our understanding of myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease. Common symptoms include weakness of the muscles that control the eyes, eyelids, facial expressions, chewing, talking, and swallowing. The condition is usually due to the presence of antibodies against acetylcholine receptors in the neuromuscular junction.
As noted by Dr. Kaminski, we have known about MG for decades as a disease that attacks the acetylcholine receptors at neuromuscular junctions. Based on that understanding, one treatment that has historically been used is cholinesterase inhibitors that can increase the level of acetylcholine in the neuromuscular junction to counteract the lower number of receptors available.
MG is now established an autoimmune disease but the reason why the body attacks its own acetylcholine receptors is not known. Based on that understanding, various corticosteroid therapies and immunosuppressive therapies have been studied, to varying efficacy. Further studies noted that medications like rituximab, which are not that effective in most MG patients, are effective in MG patients with anti-muscle-specific kinase (MuSK) antibodies, who make a small percentage of MG patients. As Dr Kaminski notes, understanding why rituximab is effective in MUSK positive patients but less effective in people with the more common anti-acetylcholine receptor antibodies has further enhanced our understanding of this disease.
Much of this work is continuing at the Myasthenia Gravis Rare Disease Network (MGNET), that is led by Dr. Kaminski and is a consortium of the Rare Disease Clinical Research Network (RDCRN).
To learn more about MGNET visit reporter.nih.gov/project-details/9804343
To learn more about RDCRN, visit ncats.nih.gov/rdcrn
CheckRare is collaborating with RDCRN to educate health care professionals one ways they can be part of this network to advance clinical research. Visit our collaboration page at checkrare.com/rare-diseases-clinical-research-network/