From the Research
Ibudilast is an investigational medication for ALS that shows promise but is not yet FDA-approved for this condition, and patients interested in it should discuss clinical trial participation with their neurologist. It works as a phosphodiesterase inhibitor and anti-inflammatory agent that may help slow disease progression by reducing neuroinflammation and microglial activation in the central nervous system 1. In clinical trials, ibudilast (MN-166) has demonstrated potential neuroprotective effects and may help preserve motor function in ALS patients, although a recent study found no significant reductions in motor cortical glial activation or CNS neuroaxonal loss at a dosage of up to 100 mg/day 1. The typical dosage studied in trials is 60-100 mg daily, administered orally. Early research suggests it may be most beneficial when started early in the disease course and used alongside standard ALS treatments like riluzole or edaravone, which have been shown to improve survival in ALS patients 2, 3, 4. Ibudilast appears to have a favorable safety profile with common side effects including gastrointestinal symptoms, headache, and depression. However, dose reductions and discontinuations due to treatment emergent adverse events were common at the higher dosage in ALS participants 1. Future pharmacokinetic and dose-finding studies of ibudilast would help better understand tolerability and target engagement in ALS. Given the current state of research, the most appropriate course of action for patients interested in ibudilast is to discuss clinical trial participation with their neurologist, as this remains the primary way to access this treatment while research continues to determine its long-term efficacy in ALS management.