Related Experiment Video
Updated: Apr 30, 2026
Lumbar Intrathecal Injection of SOD1-ASOs for Precise CNS Targeting and Predictive Efficacy in Human SOD1-G93A ALS Mice
Published on: February 24, 2026
Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery
Philip Van Damme1, Wim Robberecht
1KU Leuven (University of Leuven), Department of Neurosciences, Experimental Neurology and Leuven Research Institute for Neuroscience and Disease (LIND) , Leuven , Belgium.
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.
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