Allegheny Health Network
Find a
Request An Appointment Login to
News Classes &
Call 412.Doctors

Innovative Investigational Myasthenia Gravis Medication

Innovative Investigational Myasthenia Gravis Medication

Allegheny General Hospital Chosen as Test Site for Innovative Investigational Myasthenia Gravis Medication

By Stephanie Waite, Staff Writer

The Myasthenia Gravis Association of Western Pennsylvania at Allegheny General Hospital (AGH) has been selected to participate in a clinical trial of a new investigational medication believed to have the potential to treat myasthenia gravis, a chronic, autoimmune neuromuscular disease that can be controlled but not cured.

The trial is studying the safety and effectiveness of CK-2017357, an investigational new drug that earlier clinical trial results suggest may increase muscle strength by a different mechanism than traditional medications used to treat myasthenia gravis, according to neurologist George A. Small, MD, Medical Director of the AGH Myasthenia Gravis Center.

“This is a novel substance that increases muscle strength, but not by affecting the patient’s immune system, as other drugs do,” Dr. Small said. “Most therapies for myasthenia gravis depress the immune system’s functioning, and one of the most common therapies, prednisone, has numerous side effects.

“It also works differently than the most common medication used for short-term symptom relief. This is a new approach that could benefit many myasthenia gravis sufferers who are not adequately treated by current medications and therapies.”

Myasthenia gravis sufferers have muscle weakness that increases with activity. Weakness in myasthenia gravis most often affects the muscles that control chewing, swallowing and eye movement, followed by the muscles that control the arms and legs. Myasthenia gravis is caused by antibodies that attack the connections between muscles and the nerve fibers that control them.

Medications that improve the connection between nerve and muscle, as well as treatments that suppress the body’s immune function, are the most common treatments, but myasthenia gravis can also be treated with plasmapheresis, a process that removes abnormal antibodies from the patient’s plasma, or by thymectomy, the surgical removal of the thymus gland.

The AGH Myasthenia Gravis Center is one of about 15 centers nationwide selected to participate in this phase 2 clinical trial. The trial is funded by the National Institute of Neurological Disorders and Stroke and Cytokinetics, Inc., the California company that discovered and is developing CK-2017357.

As many as 36 patients may enroll in the trial. Each will receive a single dose of a placebo, 250 mg of CK-2017357 or 500 mg of CK-2017357 in random order (given 1 week apart) during the course of the trial.

Investigational new drug CK-2017357 has not been approved by the United States Food and Drug Administration (FDA) or any other health regulatory authority for the treatment of any disease or condition. Consequently, CK-2017357 is available only through participation in a clinical trial such as the myasthenia gravis trial at the AGH Myasthenia Gravis Center. Cytokinetics has already completed two other early clinical trials of CK-2017357, one in patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease) and another in patients with claudication (muscle pain and fatigue) associated with peripheral artery disease.

Information regarding the results of these two recently completed trials can be found at the company’s website (

For more information, contact the Myasthenia Gravis Association of Western Pennsylvania at Allegheny General Hospital at 412.566.1545 or visit and reference identifier NCT01268280.