Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery. (July 2014)

• Record Type: Journal Article
• Authors: Van Damme, Philip; Robberecht, Wim
• Is Part Of: Expert opinion on investigational drugs. Volume 23:Number 7(2014:Jul.)
• Page Start: 955
• Page End: 963
• Publication Date: 2014-07
• Subjects: amyotrophic lateral sclerosis -- BBB -- frontotemporal dementia -- -- intracerebroventricular -- intrathecal; Drugs -- Design -- Periodicals; Drugs, Investigational -- Bibliography; Drugs, Investigational -- Periodicals; 615.1
• DOI: 10.1517/13543784.2014.912275
• Languages: English
• ISSNs: 1354-3784

Abstract

Abstract : Introduction: Amyotrophic lateral scleroses (ALS) are neurodegenerative disorders primarily affecting the motor system. These incurable disorders are relentlessly progressive and typically limit survival to 2 – 5 years after disease onset. An improved knowledge about disease-causing genes, disease proteins and pathways has revealed considerable heterogeneity in ALS. Novel targeted therapies are being developed, but getting these beyond the BBB remains a challenge. Areas covered: The authors review the intracerebroventricular and intrathecal delivery of drugs for the treatment of ALS in preclinical and clinical studies. Expert opinion: Lack of BBB permeability should not hold back the development of promising treatments for ALS, as the available evidence suggest that direct intrathecal or intracerebroventricular administration of drug is a feasible delivery route in patients with ALS.