Amiloride

Amiloride is a licenced medication in the treatment of heart disease. It is also currently being tested in a phase 2 clinical trial called MS-SMART in people with secondary progressive MS.

Current phase of trial: Phase 2

Type of MS: Secondary progressive MS and optic neuritis

How does amiloride work? How is amiloride taken?

Latest Research

Earlier Research

What are the side effects of amiloride?

How does amiloride compare with current therapies?

When is amiloride likely to become available?

How does amiloride work?

Amiloride is a sodium channel blocker. A build-up of sodium in the brain is thought to be linked to nerve damage in MS, and based on existing research, amiloride is thought to be neuroprotective through its action in stopping sodium entering cells. The theory is that it could therefore be effective in the treatment of MS.

How is amiloride taken?

Amiloride is taken as an oral pill.

Latest Research

MS-SMART

MS-SMART is a three-year trial that will test the safety and effectiveness of three different drugs - amiloride, fluoxetine and riluzole – against a placebo treatment in 440 people with secondary progressive MS. Recruitment is taking place throughout 2015 and the trial is expected to be completed in 2017. For more information you can visit the MS-SMART website http://www.ms-smart.org/.

ACTION trial

We funded a clinical trial to look at the neuroprotective potential of amiloride in a phase 2 trial involving 46 people with acute optic neuritis.

This trial examined the effect of taking amiloride on the nerves at the back of the eye (retinal nerve fibre layer) compared to a dummy (placebo) drug. Initial results of this trial were presented at a scientific conference in September 2016. Researchers found that amiloride did not protect nerves from damage in acute optic neuritis.

Earlier Research

In 2007, the MS Society set up the UK MS Clinical Trials Network (CTN): a group of MS experts tasked with developing and producing clinical trials for progressive MS. The group, funded by the MS Society, carried out £500,000 worth of underpinning research that was needed in order to develop the MS-SMART trial.

Through the work of the CTN, a number of drugs were established as having the potential to be neuroprotective, of which amiloride scored particularly highly.

 

One trial has tested the safety and effectiveness of amiloride in 14 people with primary progressive MS. The treatment was deemed safe, and a significant reduction in brain atrophy was observed.

What are the side effects of amiloride?

The side effects associated with amiloride in people with MS are currently unknown, although side effects reported in the early small stage trials included 2 out of 14 patients stopping treatment, due to worsening bladder problems.

How does amiloride compare with current therapies?

Amiloride hasn’t yet been directly compared with other therapies for progressive MS.

When is amiloride likely to become available?

The MS-SMART trial is due to finish in 2017 and if the results are positive amiloride would then need to be tested in larger phase 3 trials before it could be licensed.

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