Masitinib for ALS
Also known as: AB1010
A tyrosine kinase inhibitor targeting neuroinflammation — the immune-driven component of ALS progression.
Mechanism of Action
Masitinib selectively inhibits c-Kit, PDGFR, and CSF1R tyrosine kinases, reducing mast cell and microglial activation. By dampening neuroinflammation, it aims to slow the non-cell-autonomous component of motor neuron death driven by activated glia and infiltrating immune cells.
General mechanism: Selective tyrosine kinase inhibitor (c-Kit, PDGFR, CSF1R). Anti-neuroinflammatory.
Current Evidence
Phase III trial (AB19001) showed significant slowing of functional decline in normal/fast progressors. EMA conditional approval expected. Second confirmatory trial results pending.
Clinical Status: Phase III complete. Under regulatory review in Europe. Not yet FDA-approved.
Safety Profile
Rash, edema, GI effects. Requires monitoring for myelosuppression. Generally manageable side effect profile.
Key Research Questions
- Can masitinib biomarkers (CSF neurofilament, inflammatory cytokines) predict responders?
- What is the optimal combination strategy with riluzole and/or edaravone?
- Does masitinib benefit slow progressors as well?