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

Bariş Genç; P. Hande Özdinler

doi:10.1016/j.drudis.2013.10.014

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

Bariş Genç, P. Hande Özdinler^*

^*Corresponding author for this work

Neurology

Research output: Contribution to journal › Review article › peer-review

30 Scopus citations

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).

Original language	English (US)
Pages (from-to)	441-449
Number of pages	9
Journal	Drug Discovery Today
Volume	19
Issue number	4
DOIs	https://doi.org/10.1016/j.drudis.2013.10.014
State	Published - Apr 2014

Funding

This work is supported by grants from the Les Turner ALS Foundation and the Wenske Foundation (PHO). BG was supported by an NIH postdoctoral MAD (Mechanisms of Aging and Dementia) training grant ( NIH 5T32AG020506-09 ).

ASJC Scopus subject areas

Drug Discovery
Pharmacology

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10.1016/j.drudis.2013.10.014

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title = "Moving forward in clinical trials for ALS: Motor neurons lead the way please",

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).",

author = "Bari{\c s} Gen{\c c} and {\"O}zdinler, {P. Hande}",

note = "Funding Information: This work is supported by grants from the Les Turner ALS Foundation and the Wenske Foundation (PHO). BG was supported by an NIH postdoctoral MAD (Mechanisms of Aging and Dementia) training grant ( NIH 5T32AG020506-09 ).",

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AB - 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).

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

Abstract

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