Amiloride is already used to treat heart disease. It's being tested for people with secondary progressive MS in a phase 2 clinical trial called MS-SMART.

Current phase of trial: Phase 2

Type of MS: Secondary progressive MS and optic neuritis

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 scientists predict that amiloride could protect nerves by stopping sodium entering cells.

How is amiloride taken?


Latest research


We co-funded 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. The trial is expected to be completed in 2018. For more information visit the MS-SMART website.

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 that 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. 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.

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 in 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.

When will amiloride be available?

The MS-SMART trial is due to finish in 2018. If the results are positive amiloride would then need to be tested in larger phase 3 trials before it could be licensed.

We'll keep you updated on the progress of amiloride.

Back to top

What's new?