masitinib (Masican)

Indications

• prevention of severe asthma exacerbation in patients with eosinophil count of >= 150/uL
• pending FDA approval for amyotrophic lateral sclerosis (ALS)
• may slow cognitive deterioration & improve function in Alzheimer's disease

Dosage

• start: 4.5 mg/kg/day PO
  • maximum: 6 mg/kg/day PO (no benefit of 6 vs 4.5 mg/kg/day for AD)

Adverse effects

• gastrointestinal disorders
• edema
• rash

Mechanism of action

• macrophage colony stimulating factor 1 receptor (CSF1R) inhibitor
• targets mast cells & macrophages including neurotoxic aberrant glial cells (resident macrophages in the brain) via CSF1R inhibition
• provides a neuroprotective effect & attenuates neurodegeneration Clinical trials
• in phase 3 trial
• phase 2 Alzheimer's disease: 24 weeks, patients already taking cholinesterase inhibitor &/or memantine for at least 6 months
  • measures: ADAS-Cog score & ADCS-ADL scale

Notes

• Cost: $288 for 200 mg.
  • at a dose of 4.5 mg/kg/day, cost for a 70 kg person would be $450/day

More general terms

• small inhibitory antineoplastic agent (ib drug)

Database

• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=10074640
• PubChem: http://pubchem.ncbi.nlm.nih.gov/summary/summary.cgi?cid=25024769