We have announced the launch of projects aimed at identifying repurposed drugs to treat motor neuron disease (MND), a devastating, life-shortening condition.

The four projects will test whether drugs approved for other conditions could be used to treat MND, helping to identify which treatments should be further tested in clinical trials.

The majority of MND clinical trials, including those using repurposed drugs, fail due to lack of effectiveness. This initiative aims to change this by improving the early research in drug repurposing and ensuring the testing being done is as rigorous as possible.

This targeted programme, part of our MND & Rare Dementia Translational Challenge, assessed proposals from all over the world with a focus on those with the most compelling evidence that their drug candidate could be effective. 

“MND is a devastating condition and there have been very few breakthroughs for people living with it. This is why we are hopeful this truly global search for the most promising ideas, attracting interest from researchers in 31 countries across five continents, give us the best possible chance of finding safe and effective treatments for MND. We need to do better for people living with this disease as they don’t have time to wait.”

Paul Wright, Head of MND & Rare Dementia at LifeArc

The four projects being funded are:

• University of Munich in cooperation with the University of Ulm and the Istituto di Ricerche Farmacologiche Mario Negri IRCCS in Milan – Prof Paul Lingor, Prof Alberto Catanese and Prof Valentia Bonetto exploring whether an existing drug, already approved for other conditions, could be used to help protect nerve cells by targeting a key process known to drive MND

• University of Sheffield – Prof Richard Mead Investigating whether drugs called JAK inhibitors, used to treat conditions like arthritis and inflammatory bowel conditions like ulcerative colitis, could slow progression of MND

• Massachusetts General Hospital – Dr Brian Wainger investigating whether the well-known heart medication, digoxin, can be used to protect neurons and slow progression of MND

• University of Sydney – Prof Adam Walker studying whether a drug being tested in cancer that stimulates the cell’s natural ‘clean-up’ process could remove toxic proteins that build up in people living with MND and so help slow the nerve damage caused by the disease

“Our project exemplifies how interdisciplinary collaboration and smart drug repurposing can transform existing medicines into new hope for people with MND. Our goal is to move rapidly from molecular understanding to tangible therapeutic progress, turning scientific discovery into real-world impact.” 

Prof Paul Lingor from University of Munich and one of the project leads

One person who knows the reality of life with MND and the lack of options is Chris, who was part of our MND patient panel, or Insights Group, who reviewed the research proposals. He was diagnosed with MND around 2 years ago and has recently been diagnosed with a rare form of dementia linked to MND called Fronto-Temporal Dementia.

Chris said, “MND has no cure, and virtually nothing can be done to slow progress. Looking at repurposing existing drugs, rather than ‘reinventing the wheel’, offers genuine hope that treatments may be found. While any breakthroughs will come too late for me, there may be hope for patients in the very near future, and MND may soon cease to be such a devastating diagnosis.”

MND is a devastating, life-shortening condition with very few treatments and no cure, with most people passing away within 3-5 years following diagnosis.

This programme is designed to generate the strongest possible research and identify which repurposed drugs should progress into clinical trials and established UK trial platforms such as the EXPERTS-ALS and MND-SMART studies. It will also help develop the next generation of scientists who can continue efforts to find a cure for MND.

The insights gained from this programme can inform not just MND research, but also drug development for other neurodegenerative conditions where repurposing could accelerate treatment discovery.