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Amiloride is a drug already used to treat heart disease. Unfortunately, results from a mid-stage clinical trial called MS-SMART have shown that it is not an effective treatment for MS.

Current phase of trial:
Phase 2

Find out more about amiloride

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. Scientists predict amiloride could protect nerves by stopping sodium entering cells.

How is amiloride taken?

It's a tablet

Latest amiloride research


MS-SMART was a phase 2 trial that tested the potential of three different drugs - riluzole, amiloride and fluoxetine - in 440 people with secondary progressive MS.

The top-line results of MS-SMART were announced in October 2018.

Unfortunately, none of the three drugs that were tested showed the potential to slow disability progression in MS.

Read more about MS-SMART on the project page.

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. Scientists examined the effect of taking amiloride on the nerves at the back of the eye (retinal nerve fibre layer) compared with a dummy (placebo) drug.

Initial results of this trial were presented at a scientific conference in September 2016. Researchers found amiloride did not protect nerves from damage in acute optic neuritis.

Earlier research

In 2007, we set up the UK MS Clinical Trials Network (CTN): a group of MS experts tasked with developing and producing clinical trials for progressive MS.

We funded them to carry out the £500,000 worth of underpinning research 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.

What are the side effects of amiloride?

The side effects associated with amiloride for people with MS will be tested as part of the MS-SMART trial.

In the early trial two out of 14 patients stopping treatment due to worsening bladder problems.

Commonly reported side effects from people using it to treat other conditions include nausea, vomiting, abdominal pain, headache and dizziness.

How does amiloride compare with current therapies?

There are no treatments licensed to treatment secondary progressive MS yet.

When will amiloride be available?

The MS-SMART trial finished in 2018. The results showed that amiloride is not an effective treatment for MS. The trial was extremely well conducted, meaning these results are robust and we therefore now know not to pursue these drugs any further.

This means that amiloride will not be taken forward to further clinical trials, and will not become available as a treatment for MS.

Read more about MS-SMART on the project page.

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