People with progressive forms of MS (primary and secondary progressive MS) have long been concerned about the lack of medications that might help slow the progression of the disease.
While there are many medications now that have some modest effect in relapsing-remitting MS, there has been little progress in developing any medication that might help progressive MS. This new pilot study, from Lille in France, offers hope that a treatment may eventually prove to be somewhat effective.
This was a preliminary study, without adequate numbers to prove a benefit, but designed to see if the drug masitinib did affect the course of progressive MS in a small sample of people with that disease. Masitinib is a drug that is taken by mouth and inhibits an enzyme called tyrosine kinase, leading ultimately to the inhibition or death of mast cells, certain cells in the immune system intimately involved in the progression of MS.
The researchers randomly allocated 35 people with progressive forms of MS to the treatment group (27) or placebo (8). While there were side effects in those treated, there was also a substantial improvement in their functional scores compared to when they started the trial, as opposed to a worsening of the functional scores in those receiving placebo.
There were some serious adverse reactions to the drug, including marked reductions in the white cell count, predisposing these people to the potential for serious infections, but these disappeared rapidly on discontinuing the drug.
While there are clearly problems related to this toxicity to be worked through yet, this is the first significant hope of a drug treatment for progressive forms of MS, and is likely to be welcome news to many people with this form of the disease. This study paves the way for a larger randomised controlled trial of masitinib.