Summary
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]
Details
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)