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The approval of riluzole as the first agent to treat ALS has raised a number of important questions and brought the treatment of ALS into a new era. In addition to patients with classic ALS in whom a modest improvement in life expectancy was shown compared with placebo, what other patients might benefit from riluzole? Should patients with progressive muscular atrophy (PMA) be given the drug? Benign focal amyotrophy? At-risk patients with familial disease in whom the SOD1 gene defect has been identified? What about healthy patients from families with autosomal dominant ALS without identifiable gene defects?
Because each treatment trial has used different outcome measures, when insulin-like growth factor-I (IGF-I) or the next agent is approved, how will we decide which agent or agents to use? Recognizing that no data currently exist to …
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