Abstract:
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.
journal_name
Expert Rev Neurotherjournal_title
Expert review of neurotherapeuticsauthors
Garbuzova-Davis S,Thomson A,Kurien C,Shytle RD,Sanberg PRdoi
10.1080/14737175.2016.1207530subject
Has Abstractpub_date
2016-12-01 00:00:00pages
1397-1405issue
12eissn
1473-7175issn
1744-8360journal_volume
16pub_type
杂志文章,评审abstract:INTRODUCTION:Pharmaceutical companies and the NIH have invested heavily in a variety of potential disease-modifying therapies for Alzheimer's disease (AD) but unfortunately all double-blind placebo-controlled Phase III studies of these drugs have failed to show statistically significant results supporting their clinica...
journal_title:Expert review of neurotherapeutics
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journal_title:Expert review of neurotherapeutics
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journal_title:Expert review of neurotherapeutics
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journal_title:Expert review of neurotherapeutics
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journal_title:Expert review of neurotherapeutics
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