Fourth drug added to MND-SMART

24 June 2025

A fourth drug, tacrolimus, has recently been launched in MND-SMART.

MND-SMART is the largest MND clinical trial in the UK. The trial is designed to test multiple drugs at once so that researchers can get definitive answers about the efficacy of new drugs rapidly. Drugs that are found to be ineffective can be removed from the trial and new drugs can be added once the trial is already running.

We are one of the partners who have funded MND-SMART, alongside the Euan MacDonald Centre for MND Research, MND Scotland, MND Association, the Alan Davidson Foundation and Baillie Gifford.

Tacrolimus has recently been launched as the fourth drug to be tested as part of the clinical trial. This is a medication already approved for use in a range of conditions to suppress the body’s immune system, including to prevent rejection after organ transplants.

Dr Madina Kara, our interim Director of Research said: "Alongside other funding partners, we have supported MND-SMART to enable smart and efficient research to accelerate the development of new treatments. We are pleased to continue supporting clinical studies through funding many teams across the UK through our Clinical Research Infrastructure Awards, to increase the opportunities for people with MND to be recruited to MND-SMART and other clinical trials."

Co-designed with people living with MND, MND-SMART incorporates features to ease the burden of participation, such as at-home assessments, drugs couriered to participants, remote follow-up using teleconferencing, and more regular contact with healthcare professionals.

Steve Barrett, MND-SMART participant and a member of the patient advisory group, said: "I have found it hugely empowering being involved in MND research and I have a real sense that something transformational will appear because of this work within my lifetime. Without research programmes such as MND-SMART, those who are diagnosed with this disease would be treated in ways that have never brought positive outcomes, and a predominant sense of hopelessness would perpetuate. From an emotional, physical, and learning perspective, MND-SMART is absolutely mission critical for what we are trying to achieve, which is a better suite of outcomes for people diagnosed with MND, and their loved ones who are also affected by the condition.”

Promising research carried out in laboratories at the UK Dementia Research Institute at the University of Edinburgh has shown tacrolimus may be able to slow the progression of MND by stopping abnormal protein clumping and reducing inflammation in the nervous system, positioning it as a strong candidate for the trial.

Professor Siddharthan Chandran, MND-SMART Chief Investigator, Director of Euan MacDonald Centre and of the UK Dementia Research Institute, said: “It is encouraging that our pipeline of laboratory research including human-based drug screening is yielding exciting new candidate drugs such as tacrolimus with a compelling evidence base to justify definitive testing in clinical trials.”

MND-SMART is enabling hundreds of people living with MND to take part in tests of potential treatments. Almost 1000 people have been enrolled at 23 sites across the UK since the trial launched in 2020.

Professor Suvankar Pal, MND-SMART Co-Lead Investigator, UK Dementia Research Institute Clinical Group Leader and Euan MacDonald Centre, University of Edinburgh, said: “The addition of tacrolimus into MND-SMART is a positive step for people affected by MND. We already know the drug is safe, and it has shown real promise in preliminary testing in the lab. In just five years, we’ve expanded UK trial infrastructure for MND to 23 sites in all four nations of the UK, in both rural and urban areas. There is a strong sense of hope that comes from our participants, and we are proud to be changing the conversation for people living with MND from managing symptoms to actively participating in research that could help themselves and those diagnosed with MND in the future.”

For further information about this latest update, please visit the MND-SMART website or, if you are enrolled on the trial, please speak to your site team.