Translating Preclinical Insights into Effective Human Trials in Als.
From: Department of Neurology, Massachusetts General Hospital, Boston, MA 02129, USA. adibernardo@partners.org
Biochimica et biophysica acta
- Publish Date:
- ISSN: 0006-3002
- Volume: 1762
- Issue: 11-12
- Pages: 1139-49
- Medium: Print
- Language: English
- Citation (JAMA): DiBernardo Allitia B, Cudkowicz Merit E, et al. Translating Preclinical Insights into Effective Human Trials in Als.. Biochim. Biophys. Acta ;1762:1139-49
Abstract
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive, adult-onset neurodegenerative disease characterized by selective dysfunction and death of motor neurons in the brain and spinal cord. The disease is typically fatal within 3-5 years of symptom onset. There is no known cure and only riluzole, which was approved by the FDA in 1996 for treatment of ALS, has shown some efficacy in humans. Preclinical insights from model systems continue to furnish ample therapeutic targets, however, translation into effective therapies for humans remains challenging. We present an overview of clinical trial methodology for ALS, including a summary rationale for target selection and challenges to ALS clinical research.
Mesh Headings (Keywords): Amyotrophic Lateral Sclerosis, Animals, Clinical Trials as Topic, Disease Models, Animal, Drug Design, Humans, Mice, Rats
Check for Full Text / PubMed Unique Identifier (PMID): 16713196
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