Institute of Oncology, Rabin Medical Center, Petah Tikva, Faculty of Medicine, Tel Aviv, Israel
Research Article
Oral inhaled cromolyn for mild-to-moderate amyotrophic lateral sclerosis: A randomized, open label phase 2a study
Author(s): David Brazier, Salomon M. Stemmer, William Garofano, Atul Gupta, Peter S. Cont and David R. Elmaleh*
Background: Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder of the motor neuron system for which there is currently no effective treatment. This study aims to evaluate the safety and efficacy of two doses of oral inhaled cromolyn in mild to moderate ALS over 12 weeks.
Methods and findings: The primary efficacy parameter is the plasma neuroinflammatory biomarkers. The exploratory parameters are the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), Forced Vital Capacity (FVC%), and Peak Inspiratory Flow Rate (PIFR). The study recruits 12 participants. A favorable decrease in proinflammatory plasma biomarkers is observed in 12 of the 18 biomarkers following 12-week treatment. Additionally, exploratory efficacy assessments demonstrated highly favorable trends: the ALSFRS-R score declined by -0.77 points per month (-2.6 poin.. View More»
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