Can Selection of Rapidly Progressing Patients Shorten Clinical Trials in Amyotrophic Lateral Sclerosis?

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Abstract
Amyotrophic lateral sclerosis (ALS) is an inevitably progressive disease, but the individual rate of clinical deterioration is quite variable. Prognostic indicators include the clinical presentation (worse for respiratory or bulbar onset), age at symptom onset (better for young patients), delay from first symptom to entering an ALS clinic (a shorter delay meaning more rapid progression), rate of change in scores on the Appel ALS Rating Scale or the ALS Functional Rating Scale (ALS-FRS), forced vital capacity at diagnosis and rate of change in respiratory function, decline in muscle strength measured by maximal voluntary isometric contraction or manual muscle testing, and reduction in motor unit number estimation (MUNE).1-5 These considerations suggest that selection of patients with rapid progression might provide a more homogeneous patient population for entry into clinical trials. It is notable that neurophysiologic studies have not been applied as an end point in trials.3,4