Clinical Trials Outside the United States: Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a devastating neurodegenerative disorder characterized by rapidly progressive weakness due to degeneration of motor neurons in the brain cortex, brain stem, and spinal cord. Treatments for ALS have been limited due to the diverse nature and unknown etiology of the disease. Riluzole (Rilutek) is the only FDA-approved treatment known to improve survival in ALS patients, but only by a span of several months. Much of this difficulty in finding treatment relates to the various etiologies and heterogeneity that exist within ALS. In recent years, much interest has been divested into the use of human pluripotent stem cells for the treatment of ALS. While the initial idea was to use the pluripotent stem cells to ...