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Restoring brain barriers: an innovative approach for treating neurological disorders
Sighild Lemarchant1, Britta Engelhardt2, Francesca Cicchetti3,4
1Axoltis Pharma, Lyon, 69008, France. slemarchant@axoltis.com.
Fluids and Barriers of the CNS
|July 10, 2025
Summary
Over 41% of neurological diseases involve blood-brain barrier dysfunction, a key factor in disease progression. Therapeutic strategies targeting these barriers offer new hope for treating neurological conditions.
Area of Science:
- Neuroscience
- Vascular Biology
- Neurology
Background:
- Neurological disorder etiology is complex, hindering therapeutic candidate identification.
- Brain vascular dysfunction is an emerging therapeutic strategy.
- Blood-brain barrier (BBB) and blood-spinal cord barrier (BSCB) alterations are implicated in neurological diseases.
Purpose of the Study:
- To quantify the prevalence of BBB/BSCB dysfunction in neurological diseases.
- To explore the clinical relevance of barrier status in patient outcomes.
- To review drug candidates targeting barrier repair.
Main Methods:
- Analysis of existing data on neurological diseases and barrier function.
- Review of clinical evidence linking barrier status to patient outcomes.
- Survey of drug development pipelines for barrier-targeting therapies.
Main Results:
- At least 41% of neurological diseases exhibit co-pathology of BBB/BSCB alterations.
- This figure likely underestimates the true prevalence of barrier dysfunction.
- Clinical data suggest barrier status significantly impacts pathological and functional outcomes.
Conclusions:
- BBB/BSCB dysfunction is a widespread and significant factor in neurological disorders.
- Targeting vascular repair presents a promising disease-modifying strategy.
- Further research and drug development are crucial for harnessing this therapeutic avenue.
Keywords:
Alzheimer’s diseaseAmyotrophic lateral sclerosisBBB disruptionBSCB disruptionClaudin-5Huntington’s diseaseMultiple sclerosisParkinson’s diseaseTherapeutic strategiesTight junctionMore Related Videos
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