Journal
EXPERT REVIEW OF NEUROTHERAPEUTICS
Volume 16, Issue 12, Pages 1397-1405Publisher
TAYLOR & FRANCIS LTD
DOI: 10.1080/14737175.2016.1207530
Keywords
ALS; animal models; blood-CNS barrier; clinical trials; drug therapy; patients
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Funding
- Center of Excellence for Aging and Brain Repair, Department of Neurosurgery and Brain Repair at the University of South Florida
- National Institutes of Health [1R01 NS090962-01]
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Introduction: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by motor neuron degeneration in the brain and spinal cord. Treatment development for ALS is complicated by complex underlying disease factors.Areas covered: Numerous tested drug compounds have shown no benefits in ALS patients, although effective in animal models. Discrepant results of pre-clinical animal studies and clinical trials for ALS have primarily been attributed to limitations of ALS animal models for drug-screening studies and methodological inconsistencies in human trials. Current status of pre-clinical and clinical trials in ALS is summarized. Specific blood-CNS barrier damage in ALS patients, as a novel potential reason for the clinical failures in drug therapies, is discussed.Expert commentary: Pathological perivascular collagen IV accumulation, one unique characteristic of barrier damage in ALS patients, could be hindering transport of therapeutics to the CNS. Restoration of B-CNS-B integrity would foster delivery of therapeutics to the CNS.
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