Oral Inhaled Cromolyn for Mild-to-Moderate Amyotrophic Lateral Sclerosis: A Randomized, Open Label Phase 2a Study

This is the authors' abstract. We don't add key points for this paper.

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. 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 points over 12 weeks), markedly better than the typical decline of around -1 point per month seen in ALS patients. The decline in FVC was equally impressive at just -0.51% per month (−1.53% over 12 weeks), significantly lower than the usual 3-5% monthly decline. Both doses are found to be safe and well-tolerated. Despite fewer participants, this study provides preliminary evidence supporting oral inhaled cromolyn’s safety and potential efficacy in ALS treatment.