Journal
LANCET NEUROLOGY
Volume 13, Issue 11, Pages 1127-1138Publisher
ELSEVIER SCIENCE INC
DOI: 10.1016/S1474-4422(14)70129-2
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Funding
- National Institute of Environmental Health Sciences (NIEHS)
- Muscular Dystrophy Association
- Spastic Paraplegia Foundation
- Agency for Toxic Substances and Disease Registry (ATSDR)
- Neuraltus
- Biogen Idec
- Cytokinetics
- Genervon
- NINDS/National Institute of Health/ORDR
- MDA
- ALS Association
- ALS Society of Canada
- Motor Neuron Association (UK)
- Judith & Jean Pape Adams Charitable Foundation
- Ride for Life
- Les Turner ALS Foundation
- Outreach of Westchester ALS
- ALS Hope Foundation
- Knopp Biosciences
- Sanofi-Aventis
- Avanir
- ALS Therapy Alliance and ALS Association
- National Institute of Neurological Diseases and Stroke through NorthEast ALS Consortium
- Massachusetts General Hospital-Harvard Medical School and Johns Hopkins University School of Medicine
- Muscular Dystrophy Association-ALS Division
- Carolinas ALS Research Fund
- Pinstripes Fund
- Carolinas Garden of Hope
- Heineman Medical Research Fund Carolinas Healthcare Foundation
- Cytokinetics Pharmaceuticals
- Biogen-Idec Pharmaceuticals
- Avanir Pharmaceuticals
- Glaxo-Smith-Kline Pharmaceuticals
- Neuraltus Pharmaceuticals
- Carolinas Healthcare Foundation
- Italian Ministry of Health
- Agenzia Italiana per la Ricerca sulla SLA - AriSLA [NOVALS 2012]
- Ministry of Health
- EU Joint Programme-Neurodegenerative Disease (JPND) Research-the Italian Ministry of Health
- EU Joint Programme Associazione Amici Centro Dino Ferrari
- Weill Medical College of Cornell University
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Amyotrophic lateral sclerosis (ALS) is one of the most rapidly progressive neurodegenerative diseases of unknown cause. Riluzole is the only drug that slows disease progression. More than 50 randomised controlled trials (RCTs) of proposed disease-modifying drugs have failed to show positive results in the past half-century. In the past decade, at least 18 drugs have been tested in large phase 2 or 3 RCTs, including lithium, which was tested in several RCTs. Potential reasons for the negative results can be classified into three categories: first, issues regarding trial rationale and preclinical study results; second, pharmacological issues; and third, clinical trial design and methodology issues. Clinical trials for stem cell therapy and RCTs targeting pharmacological or non-pharmacological symptomatic treatment in ALS are examples of areas that need novel design strategies. Only through critical analyses of the failed trials can new and important suggestions be identified for the future success of clinical trials in ALS.
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