Fourth investigational arm launched in MND-SMART clinical trial
Jun 2025: A fourth drug, tacrolimus, has recently been launched in MND-SMART.
MND-SMART uses a multi-arm, multi-stage, adaptive design which means we can test multiple drugs simultaneously; discontinuing those that are shown to be ineffective and adding new drugs as evidence emerges. This approach reduces the time it takes to get definitive results. Tacrolimus has recently been launched as the fourth drug to be tested in MND-SMART.
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.
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.
"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."
Prof Siddharthan Chandran, MND-SMART Chief Investigator, Director of Euan MacDonald Centre and UK Dementia Research Institute
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.
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.
“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.”
Steve Barrett, MND-SMART participant and patient advisory group member
“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.
Prof Suvankar Pal, MND-SMART Co-Lead Investigator, UK Dementia Research Institute Clinical Group Leader and Euan MacDonald Centre, University of Edinburgh
Click below to hear more about the launch of tacrolimus:
MND-SMART is led by the Euan MacDonald Centre. The trial is a flagship project of the UK Dementia Research Institute.
The trial is funded by the Euan MacDonald Centre for MND Research, MND Scotland, MND Association, the Alan Davidson Foundation, My Name’5 Doddie Foundation, Baillie Gifford.