Masitinib treatment in patients with progressive multiple sclerosis: a randomized pilot study
Patrick Vermersch1, Rabah Benrabah, Nicolas Schmidt
1Department of Neurology, University of Lille Nord de France (EA2686), Hôpital Roger Salengro, CHU de Lille, Lille cedex, 59037, France. Patrick.VERMERSCH@CHRU-LILLE.FR
BMC Neurology
|June 14, 2012
Summary
Masitinib showed potential therapeutic benefits for progressive multiple sclerosis (MS) patients by improving functional scores. Further research is warranted to confirm these findings for primary progressive MS (PPMS) and secondary progressive MS (SPMS).
Area of Science:
- Neuroimmunology
- Clinical Pharmacology
- Drug Development
Background:
- Progressive multiple sclerosis (MS) lacks effective treatments.
- Mast cells contribute to MS pathogenesis by releasing inflammatory mediators.
- Masitinib, a tyrosine kinase inhibitor, targets mast cell activity.
Purpose of the Study:
- To assess the safety and clinical benefit of masitinib in treating primary progressive MS (PPMS) and relapse-free secondary progressive MS (rfSPMS).
Main Methods:
- A multicenter, randomized, placebo-controlled, proof-of-concept trial.
- Masitinib was administered orally for at least 12 months.
- The primary endpoint was the change in the Multiple Sclerosis Functional Composite (MSFC) score.
Main Results:
- Masitinib was relatively well-tolerated, with similar overall adverse event rates to placebo.
- Masitinib treatment showed a positive trend in improving MSFC scores compared to placebo.
- 32% of masitinib-treated patients achieved a clinical response versus 0% in the placebo group.
Conclusions:
- Masitinib may offer therapeutic benefits for PPMS and rfSPMS patients.
- This study supports further investigation into masitinib for progressive MS.
- Masitinib represents a potential novel treatment for progressive MS.
