Myasthenia Gravis (MG) is a debilitating and potentially deadly disease that can afflict anyone, characterized by severe muscle weakness, affecting control of facial expressions, arm and leg movements, chewing, swallowing, coughing and breathing. There is no cure, only treatment.
The diagnosis of MG can be difficult and many diagnostic tools require specialized training and equipment that is not widely available, and many patients are misdiagnosed and inappropriately managed. To treat weakness and avoid potentially fatal respiratory failure, patients with MG usually require long-term immunosuppression with oral drugs, which can cause adverse effects on the immune system. In addition, there is a long delay between onset of therapy and response with many oral immunosuppressive drugs and no clinical or laboratory methods predict response.
A better and more targeted treatment could be on the horizon, with a grant provided to Dr. Jeffrey T. Guptill, MD, MA, MHS, Assistant Professor, Department of Neurology, Duke University, by the Myasthenia Gravis Foundation of America, allowing Dr. Guptill and his team to use innovative immunologic techniques to identify biomarkers in patients with MG that will improve diagnosis and predict therapeutic responses.
The key primary goals of the grant are to investigate immune system pathways and cells that show evidence of MG. This is important because there are existing drugs that target these pathways, so if it can be determined that they are useful, it would pave the way for clinical trials for certain drug therapies. Most of the therapies available now are not very targeted. The goal is that these would allow targeted therapies, with less negative impact on the immune system, fewer unnecessary side effects and infections.
Because this disease is considered rare—approximately 20 out of 100,000 individuals in the U.S. have been diagnosed with MG, most research in this area has been done in small collaborations. As part of the grant, Dr. Guptill will also create a multi-center research network of MG centers to develop a repository of MG blood samples for use in future studies.
This network of repositories will not only benefit the MG community, but could be used for studying other autoimmune diseases, creating a ripple effect to improving treatments in other areas.