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Drug Discov Today. 2014 Apr;19(4):441-9. doi: 10.1016/j.drudis.2013.10.014. Epub 2013 Oct 27.

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

Author information

1
Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, USA.
2
Davee Department of Neurology and Clinical Neurological Sciences, Northwestern University, Feinberg School of Medicine, USA; Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Feinberg School of Medicine, USA; Cognitive Neurology and Alzheimer's Disease Center, Northwestern University, Chicago, IL 60611, USA. Electronic address: ozdinler@northwestern.edu.

Abstract

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).

PMID:
24171950
PMCID:
PMC4000750
DOI:
10.1016/j.drudis.2013.10.014
[Indexed for MEDLINE]
Free PMC Article

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