Masitinib is an oral immunomodulatory drug (one that changes the activity of the immune system), which is being developed as a possible treatment for progressive forms of multiple sclerosis (MS) and other diseases, such as amyotrophic lateral sclerosis (ALS). Its developer, AB Sciences, is currently testing its safety and effectiveness in clinical trials for a number of conditions. The drug has been designated an Orphan Drug by the U.S. Food and Drug Administration (FDA) as a potential ALS treatment.
How masitinib works
Also referred to as AB1010, masitinib belongs to a class of chemical compounds known as tyrosine kinase inhibitors (TKI). Tyrosine kinases are enzymes responsible for the activation of proteins vital to several cell signaling processes related to the growth, division, and proliferation of cells in the body. Masitinib is a selective TKI, which blocks the activity of tyrosine kinases and reduces inflammation. Mast cells (immune cells highly involved in allergic and anaphylactic reactions) are the main targets of this drug.
According to AB Sciences, “through its activity on mast cells and microglia, and consequently the inhibition of the activation of the inflammatory process, masitinib can have an effect on the symptoms associated with some inflammatory and central nervous system diseases and the degeneration of these diseases.”
History of masitinib
A pilot, 18-month Phase 2 study in 35 patients with primary or secondary forms of progressive MS was initiated in 2011. The results did not show a statistically significant difference between masitinib and the placebo, but there were promising indications that justified its continued testing, the MS Society reported.
A Phase 2b/3 double-blind, randomized, placebo-controlled study (NCT01433497) is assessing the safety and effectiveness of masitinib in people with primary progressive or relapse-free secondary progressive MS. The trial was expected to run for 96 weeks and finish in December 2015.
The main objectives were to assess the effectiveness of masitinib via changes in MSFC (Multiple Sclerosis Functional Composite). MSFC is a three-part assessment system that measures changes in leg function and walking ability, arm and hand function, and cognitive function.
Changes in the MSQOL-54 (Multiple Sclerosis Quality of Life 54 items), which is a quality of life measure, as well as changes in EDSS (Expanded Disability Status Scale), which is a used for quantifying disability in multiple sclerosis and monitoring changes in the level of disability over time, were also going to be assessed. This study intended to enroll 450 participants.
The recruitment status of this study is currently unknown and although the completion date has passed, the study’s status has not been updated or verified in more than three years.
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