Therapeutic neuroprotective agents for amyotrophic lateral sclerosis

Rachna S. Pandya, Haining Zhu, Wei Li, Robert Bowser, Robert M. Friedlander, Xin Wang

Research output: Contribution to journalReview articlepeer-review

60 Scopus citations


Amyotrophic lateral sclerosis (ALS) is a fatal chronic neurodegenerative disease whose hallmark is proteinaceous, ubiquitinated, cytoplasmic inclusions in motor neurons and surrounding cells. Multiple mechanisms proposed as responsible for ALS pathogenesis include dysfunction of protein degradation, glutamate excitotoxicity, mitochondrial dysfunction, apoptosis, oxidative stress, and inflammation. It is therefore essential to gain a better understanding of the underlying disease etiology and search for neuroprotective agents that might delay disease onset, slow progression, prolong survival, and ultimately reduce the burden of disease. Because riluzole, the only Food and Drug Administration (FDA)-approved treatment, prolongs the ALS patient's life by only 3 months, new therapeutic agents are urgently needed. In this review, we focus on studies of various small pharmacological compounds targeting the proposed pathogenic mechanisms of ALS and discuss their impact on disease progression.

Original languageEnglish (US)
Pages (from-to)4729-4745
Number of pages17
JournalCellular and Molecular Life Sciences
Issue number24
StatePublished - Dec 2013
Externally publishedYes


  • Amyotrophic lateral sclerosis pathogenesis
  • Glial cells
  • Motor neurons
  • Muscle
  • Neuroprotective agents

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Pharmacology
  • Cellular and Molecular Neuroscience
  • Cell Biology


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