Clinical Trials, U.S.: Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a devastating [Page 232]neurodegenerative disorder characterized by rapidly progressive weakness due to degeneration of motor neurons in the brain cortex, brain stem, and spinal cord. Patients can present with a variety of symptoms, usually starting with signs of muscle weakness and/or atrophy in the upper or lower limbs. Patients may also present with “bulbar” onset, referring to the functions of the medulla oblongata, such as trouble with speaking or swallowing or difficulty with tongue movement. Over time, patients experience progressive difficulty with voluntary movement to eventual paralysis and death within an average of three to five years from disease onset.

Treatments for ALS have been limited due to the diverse nature and unknown etiology of ...