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Clinical trial results suggest new drug masitinib may slow MS progression

Results from 300 people with progressive MS showed disability got worse more slowly for people taking a drug called masitinib, compared to those taking a placebo.

The drug-maker AB Science announced the results last weekend at a leading MS research conference.

301 people with MS took part in the trial. They all had either primary progressive MS or non-active (i.e. not experiencing relapses and no new lesions on an MRI) secondary progressive MS.

Researchers compared disability progression in people taking masitinib with people taking a placebo (dummy drug). They measured disability progression by seeing how people’s scores on the EDSS test increased over time. A higher score on the EDSS indicates more severe disability.

People taking masitinib showed a slower worsening of disability than the placebo group. This was true for both primary progressive and secondary progressive MS.

A scientific breakthrough for treating MS

Currently, only two drugs are licensed for certain types of progressive MS: ocrelizumab for primary progressive MS and siponimod (siponimod is not currently recommended by NICE or the Scottish Medicine Consortium for use on the NHS) for active secondary progressive MS.

If masitinib were licensed it would offer a new hope for thousands of people with progressive MS who currently have no treatment options.

Trial investigator Professor Patrick Vermersch from Lille University in France, described the results as a “scientific breakthrough”. He said, “these data are extremely encouraging, and may provide new hope for progressive MS patients.”

A different mechanism to target progression in MS

Like all currently available treatments for MS, masitinib works by targeting the immune system which misbehaves in MS. But there is a key difference.

The existing medications licensed for progressive MS target cells in what’s called the ‘adaptive’ immune system. But masitinib targets cells in the ‘innate’ immune system.

These two systems work together to keep us safe from invaders like viruses. The innate immune system springs into action at the first signs of danger, fighting anything that doesn’t belong. The adaptive immune system remembers previous invaders and targets them specifically for destruction.

This is the first time a drug targeting the innate immune system has shown an effect on MS progression. It adds to the growing body of evidence suggesting these cells make a major contribution to what goes wrong in MS, and that targeting them could be a promising treatment avenue for progressive MS.

What’s next?

Some outstanding questions remain. The trial included another group who took a higher dose of masitinib. These results did not show a significant difference between the drug and the placebo. But we don’t yet know why that might be.

And, as always, any positive trial results can only make a real difference to people with MS once a regulator like the European Medicines Agency concludes there is sufficient evidence the drug is safe and effective. The trial investigators intend to confirm their results with another, larger study looking at both EDSS and MRI. We will keep you updated on their progress.

You can read read the full abstract on the MS Virtual 2020 conference website.

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