Developments in treatments for amyotrophic lateral sclerosis via intracerebroventricular or intrathecal delivery

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.

Keywords::

• amyotrophic lateral sclerosis
• BBB
• frontotemporal dementia
• intracerebroventricular
• intrathecal

Notes

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