| Property | Value | [^2] |----------|-------| [@see] | Drug Name | Masitinib | | Brand Name | Masivet, Tyl couplings | | Drug Class | Tyrosine Kinase Inhibitor | | Target | CSF1R, KIT, PDGFR | | Route of Administration | Oral | | Development Status | Phase 3 (ALS), Phase 2 (AD, PD) | | Company | AB Science |
</div>
Overview
flowchart TD
MAS["Masitinib"]
MC["Mast Cells"]
INFLAMMATION["Inflammation"]
MAS -->|"inhibits"| MC
MC -->|"trigger"| INFLAMMATION
style MAS fill:#81c784,stroke:#333,color:#000
style MC fill:#4fc3f7,stroke:#333,color:#000
style INFLAMMATION fill:#ef5350,stroke:#333,color:#000
Masitinib (AB1010) is an oral tyrosine kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), KIT, and platelet-derived growth factor receptor (PDGFR). It is being developed for the treatment of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), and Parkinson's disease (PD) based on its ability to modulate [microglia](/entities/microglia) and mast cell function.
Mechanism of Action
Masitinib exerts its therapeutic effects in neurodegenerative diseases through several interconnected mechanisms:
...
Masitinib for Neurodegenerative Diseases
Introduction
<div class="infobox">
| Property | Value | [^2] |----------|-------| [@see] | Drug Name | Masitinib | | Brand Name | Masivet, Tyl couplings | | Drug Class | Tyrosine Kinase Inhibitor | | Target | CSF1R, KIT, PDGFR | | Route of Administration | Oral | | Development Status | Phase 3 (ALS), Phase 2 (AD, PD) | | Company | AB Science |
</div>
Overview
Mermaid diagram (expand to render)
Masitinib (AB1010) is an oral tyrosine kinase inhibitor that targets colony-stimulating factor 1 receptor (CSF1R), KIT, and platelet-derived growth factor receptor (PDGFR). It is being developed for the treatment of neurodegenerative diseases including amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), and Parkinson's disease (PD) based on its ability to modulate [microglia](/entities/microglia) and mast cell function.
Mechanism of Action
Masitinib exerts its therapeutic effects in neurodegenerative diseases through several interconnected mechanisms:
Microglial Modulation
CSF1R Inhibition: Masitinib inhibits CSF1R, which is critical for microglial survival, proliferation, and activation
M1 to M2 Shift: Promotes the transition from pro-inflammatory (M1) to neuroprotective (M2) [microglia](/cell-types/microglia-neuroinflammation) phenotype
Reduced Neuroinflammation: Decreases production of pro-inflammatory cytokines including TNF-α, IL-1β, and IL-6
Mast Cell Inhibition
KIT Inhibition: Blocks KIT receptor on mast cells, reducing mast cell activation and degranulation
Reduced Neuroinflammation: Mast cells contribute to neuroinflammation through release of histamine, tryptase, and cytokines
[Blood-Brain Barrier](/entities/blood-brain-barrier) Modulation: May help maintain BBB integrity
Oxidative Stress Reduction: Decreases [reactive oxygen species](/entities/reactive-oxygen-species) (ROS) production
Mitochondrial Function: May improve mitochondrial function in [neurons](/entities/neurons)
Clinical Development
Amyotrophic Lateral Sclerosis (ALS)
Phase 3 Trial (AB10015): Randomized, double-blind, placebo-controlled study in sporadic ALS patients
Results: Demonstrated significant slowing of disease progression in a subgroup of patients with baseline ALSFRS-R decline ≤20 points/month
Primary Endpoint: Change in ALSFRS-R score from baseline
Secondary Endpoints: Survival, respiratory function, quality of life measures
Alzheimer's Disease (AD)
Phase 2 Trial (AB20004): Study in mild-to-moderate AD patients
Rationale: Microglial activation and neuroinflammation are key pathological features in AD
Primary Endpoint: Change in ADAS-Cog and MMSE scores
Parkinson's Disease (PD)
Phase 2 Trial: Study in early PD patients
Rationale: Microglial activation contributes to dopaminergic neuron degeneration
Primary Endpoint: Change in MDS-UPDRS score
Therapeutic Targeting
| Target | Role in Disease | Therapeutic Rationale | |--------|----------------|---------------------| | CSF1R | Microglial survival and activation | Reduce pro-inflammatory microglia | | KIT | Mast cell activation | Decrease mast cell-mediated inflammation | | PDGFR | Pericyte function | May improve blood-brain barrier |
Drug Properties
Molecular Formula: C₂₈H₃₀N₆O₂S
Molecular Weight: 498.65 g/mol
Half-life: ~30 hours
Bioavailability: ~70-90%
Metabolism: Hepatic (CYP3A4)
Drug-Drug Interactions: Substrate of CYP3A4; avoid with strong inhibitors/inducers
Side Effects and Safety
Common Adverse Events
Nausea and vomiting
Diarrhea
Rash
Asthenia (weakness)
Edema
Serious Adverse Events
Liver enzyme elevations
Neutropenia
QT prolongation (rare)
Contraindications
Known hypersensitivity to masitinib
Severe hepatic impairment
Pregnancy and lactation
Combination Therapy Potential
Masitinib may have synergistic effects when combined with:
Riluzole: Additional neuroprotective effects
Edaravone: Antioxidant and anti-inflammatory combination
Standard of care: May enhance overall treatment response
Research Directions
Biomarker development to identify responders
Optimization of dosing regimens
Combination therapy studies
Early intervention trials in prodromal disease stages
Background
The study of Masitinib For Neurodegenerative Diseases has evolved significantly over the past decades. Research in this area has revealed important insights into the underlying mechanisms of neurodegeneration and continues to drive therapeutic development.
Historical context and key discoveries in this field have shaped our current understanding and will continue to guide future research directions.
Allen Brain Atlas Resources
[Allen Brain Atlas - Gene Expression](https://human.brain-map.org/) - Search for gene expression data across brain regions
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