ST. LOUIS — Saint Louis University investigators will look at a new option for patients with myasthenia gravis, a highly debilitating chronic autoimmune disorder that causes severe muscle weakness and fatigue. Together with University of Texas Southwestern and Vanderbilt University, researchers will enroll 10 patients in a six month pilot study examining an under-the-skin immunoglobulin treatment that has been used for other immune deficiencies to determine if it also is effective for myasthenia gravis.
|Ghazala Hayat, M.D.|
Myasthenia gravis is a chronic neuromuscular disorder that affects about 400 to 600 per 1 million people — roughly 1,100 to 1,700 people in the St. Louis area. Symptoms include weakness in the arms and legs, chronic muscle fatigue, difficulty breathing, difficulty chewing and swallowing, slurred speech, droopy eyelids and blurred or double vision.
Ghazala S. Hayat, M.D., professor of neurology and lead investigator of the study at SLU, says new treatment options are important for patients with myasthenia gravis.
“A treatment that is relatively well tolerated and easily self-administered would allow patients a chance to go into remission or become relapse-free without enduring many side effects of current treatments like prednisone or chemotherapy.” said Hayat.
Myasthenia gravis often is treated with prednisone, a corticosteroid. Prednisone can be effective in treating the disorder, but it can carry a host of very severe side effects, including pronounced weight gain, osteoporosis, glaucoma and diabetes.
Another treatment, intravenous immunoglobulin (IGIV) and plasma exchange, has been used most often as a maintenance therapy to keep symptoms from reappearing once prednisone has reduced or eliminated symptoms. IGIV involves infusing a patient’s blood with the pooled immunoglobulin from the plasma of several thousand healthy blood donors.
Because IGIV is given intravenously, it must be administered by health care professionals, usually in hospitals or infusion centers. In addition, it also can cause side-effects, including fever, headache, nausea and allergic reaction.
In this study, researchers will examine a new option that has been used for other autoimmune disorders but not yet tested for myasthenia gravis. Subcutaneous immunoglobulin treatment (IGSC), like IGIV, uses the plasma of healthy blood donors, but is administered subcutaneously (under the skin).
Patients or family members administer IGSC at home, rather than in a hospital. IGSC also has a lower rate of side effects than IGIV. Should it prove effective, doctors expect it would be safer, more convenient, and more cost effective.
Researchers will examine IGSC as a maintenance therapy, enrolling patients between the ages of 18-80 years old who have myasthenia gravis. Participants must currently be receiving at least 30 mg of prednisone daily, no other immunosuppressive drugs and no more than 240 mg of cholinesterase inhibitor per day. The study is being funded by CSL Behring.
Those who are interested in learning more about this study should call 314-977-4900.
Established in 1836, Saint Louis University School of Medicine has the distinction of awarding the first medical degree west of the Mississippi River. The school educates physicians and biomedical scientists, conducts medical research, and provides health care on a local, national and international level. Research at the school seeks new cures and treatments in five key areas: cancer, liver disease, heart/lung disease, aging and brain disease, and infectious disease.