• Philip Van Damme and Wim Robberecht.
• KU Leuven (University of Leuven), Department of Neurosciences, Experimental Neurology and Leuven Research Institute for Neuroscience and Disease (LIND) , Leuven , Belgium.
• Expert Opin Investig Drugs. 2014 Jul 1; 23 (7): 955-63.

IntroductionAmyotrophic 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 CoveredThe authors review the intracerebroventricular and intrathecal delivery of drugs for the treatment of ALS in preclinical and clinical studies.Expert OpinionLack 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.

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