research nurse doing a breath test with a patient



An innovative drugs trial designed to speed up the time it takes to find medicines that can slow, stop, or reverse the progression of, MND.

Watch our animation to find out about MND-SMART and how the trial works. 


MND-SMART is a pioneering clinical drugs trial in its design and reach. It will allow hundreds of people living with motor neuron disease (MND) across the UK to take part in tests of potential treatments. 

The trial is a charitably-funded university and NHS initiative that has been developed by a range of MND and clinical trial experts from across the UK, working closely with people with MND. It is led by the Euan MacDonald Centre for MND Research based at the University of Edinburgh, alongside colleagues from University College London and the University of Warwick.

Trial design

MND-SMART stands for Motor Neuron Disease – Systematic Multi-arm Adaptive Randomised Trial. 

Multi-arm means that, unlike typical clinical trials which test a single treatment, MND-SMART will test more than one at the same time. Trial participants taking the different treatments will be compared with a single group who receive a dummy drug, called a placebo. This means that people in MND-SMART are more likely to receive an active treatment when compared to standard clinical trials where half of participants receive the placebo and half the active treatment.

The trial is also adaptive so that researchers can change the drugs being tested according to emerging results. This means that new medicines can be added once a trial has started, while treatments that do not prove effective can be dropped. This is a phase 2 / 3 trial which means if drugs appear to be effective, they will seamlessly transition into phase 3 without the need for additional permissions or trial participants needing to complete a phase 2 trial and join a separate phase 3 trial. 

The range of experts involved in designing the trial are from many different clinical and academic backgrounds including MND consultants and nurses, statisticians and lab scientists.

We’ve involved and listened to people with MND throughout the trial design process and so:

  • we’ve made the clinical trial design that is open to as many people with MND as possible with very few exclusion criteria
  • the study drugs are being given as liquids which will make them as easy as possible for people to swallow
  • we’ve introduced the option of video calls for some appointments to reduce the number of times people have to travel to a Clinic
  • we’ve made the language we’ve used in the trial materials as easy to understand as possible

The drugs

The trial will initially test medicines that are already approved as treatments for other diseases. This means there is a reduced risk of adverse effects in trial participants. Repurposing drugs in this way also avoids some of the lengthy approvals processes needed for new drugs and could cut years off the time taken for the medications to become more widely available.

To find out more about the drugs currently being tested in MND-SMART, click here.

MND-SMART is a long-term study that will test medications for many years to come. We want to enable people with MND to live longer and enjoy a better quality of life.


We are extremely grateful to the individuals and organisations who fund MND-SMART. This comprises donations to the Euan MacDonald Centre for MND Research, as well as research grants from MND ScotlandAlan Davidson FoundationMy Name'5 Doddie Foundation and Motor Neurone Disease Association