June 26, 2014

Phase II trial shows promising results for Masitinib for progressive multiple sclerosis

Results from a randomized, placebo-controlled, Phase II trial investigating the safety and efficacy of Masitinib, a selective oral tyrosine kinase inhibitor, for treatment of progressive MS were published in BMC Neurology and indicate a promising area of treatment for progressive MS. [Vermersch P, Benrabah R, Schmidt N, Zéphir H, Clavelou P, Vongsouthi C, Dubreuil P, Moussy A, Hermine O. BMC Neurol. 2012 Jun 12;12(1):36. Epub ahead of print]

Mast cells (immune cells that mediate inflammatory response) are implicated in the pathology of MS because of their ability to sustain CNS inflammation. Previous animal model studies have demonstrated that Masitinib, a selective oral tyrosine kinase inhibitor can effectively inhibit the survival and activity of mast cells. Thirty-five individuals with primary progressive MS and relapse-free secondary progressive MS were randomly selected to receive Masitinib orally at 3 to 6 mg/kg/day or placebo for at least 12 months to evaluate change as compared with baseline in the multiple sclerosis functional composite score (MSFC). An improvement on MSFC scores was observed in MS-related impairment in the Masitinib group as compared with those who received placebo.

Although Masitinib was generally well tolerated the most common side effects experienced included weakness, rash, nausea, edema (swelling), and diarrhea.

This study suggests that Masitinib may be of therapeutic benefit to individuals with primary progressive and relapse-free secondary progressive MS however further evidence from larger placebo controlled trials are warranted.

MSFC source: National MS Society (USA)