Myasthenia gravis (MG) is an autoimmune disorder affecting the neuromuscular junction, usually caused by autoantibodies against the acetylcholine receptor (AChR) or the muscle-specific kinase (MuSK). Our aim is the development of a therapy based on the selective extracorporeal elimination of anti-AChR or anti-MuSK antibodies. To this end, the extracellular domains of the AChR subunits and MuSK have been expressed in yeast to be used as adsorbents, after optimization, and to obtain large quantities of proteins with near-native structure. We have characterized these proteins with respect to their use as specific immunoadsorbents for MG autoantibodies, and have begun large-scale experiments in order to verify the feasibility of application of the method for therapy. Furthermore, we have initiated animal studies to test possible toxicity and safety issues of the adsorbents or the procedure itself. The successful completion of the scale-up and safety tests will allow the initiation of clinical trials.
© 2012 New York Academy of Sciences.