Potential new complication in drug therapy development for amyotrophic lateral sclerosis.

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 Neurother

authors

Garbuzova-Davis S,Thomson A,Kurien C,Shytle RD,Sanberg PR

doi

10.1080/14737175.2016.1207530

subject

Has Abstract

pub_date

2016-12-01 00:00:00

pages

1397-1405

issue

12

eissn

1473-7175

issn

1744-8360

journal_volume

16

pub_type

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