Moving forward in clinical trials for ALS: Motor neurons lead the way please

Bariş Genç, P. Hande Özdinler*

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

24 Scopus citations


Amyotrophic lateral sclerosis (ALS) is one of the most complex motor neuron diseases. Even though scientific discoveries are accelerating with an unprecedented pace, to date more than 30 clinical trials have ended with failure and staggering frustration. There are too many compounds that increase life span in mice, but too little evidence that they will improve human condition. Increasing the chances of success for future clinical trials requires advancement of preclinical tests. Recent developments, which enable the visualization of diseased motor neurons, have the potential to bring novel insight. As we change our focus from mice to motor neurons, it is possible to foster a new vision that translates into effective and long-term treatment strategies in ALS and related motor neuron disorders (MND).

Original languageEnglish (US)
Pages (from-to)441-449
Number of pages9
JournalDrug Discovery Today
Issue number4
StatePublished - Apr 2014

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery

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