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. 2008 Oct 1;5(4):516–527. doi: 10.1016/j.nurt.2008.08.002

ALS drug development: Reflections from the past and a way forward

Swati Aggarwal 1,✉, Merit Cudkowicz 1
PMCID: PMC4514695  PMID: 19019302

Summary

Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.

Key Words: Amyotrophic lateral sclerosis, clinical trial, trial design, conduct, critical review

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