What does the drug do?
Originally Ibudilast was thought to only suppress inflammation. It was believed to inhibit toxic microglia activation and reduce neuroinflammation. However, further research has shown Ibudilast potentially affects multiple pathways in the disease process. Research has shown that it has a protective effect on glutamate induced excitotoxicity (the process in which nerve cells are damaged or killed by excessive stimulation). Furthermore, recent research has shown, in MND/ALS models, that it helps to remove misfolded/abnormally aggregated proteins, such as TDP-43 and SOD1.
The Phase 1/2 biomarker study was completed in June 2020. This trialed a higher dose (100mg) in 35 participants taken across 36 weeks, while tracking their progression using an MND biomarker [11C]-PBR28. This marker is a protein that is abundant in toxic glial cells and can be traced using imaging. Effectiveness of using this marker to track progression after treatment with ibudilast is the primary objective. Secondary objectives include ALSFRS, muscle strength and slow vital capacity scores. You can read more about the trial here.
The Phase 2 randomised, placebo-controlled, double-blind clinical trial recruited 71 people with MND who were taking 60 mg of ibudilast daily together with riluzole across 6 months, with a follow-up six month open-label phase. The treatment was shown to be safe and well-tolerated. Results of secondary outcome measures showed that, in some participants, the ALSFRS-R score didn’t decline. More detailed analyses revealed a connection between reduced disease progression while on ibudilast and higher survival rates after the treatment. You can read more about the trial here.
The Phase 2b/3 trial is currently recruiting participants to evaluate the efficacy, safety and tolerability of Ibudilast for 12 months followed by a 6-month open-label extension phase. You can find out more about the trial here.
This trial is recruiting in the US and Canada only.
2019 - The trial started enrolling participants.