LifeArc launches projects to identify motor neuron disease repurposed drug candidates

Four projects aimed at identifying the most promising repurposed drug candidates to treat motor neuron disease (MND) have been launched by the self-funded medical research organisation, LifeArc.

The projects are part of the organisations’ MND & Rare Dementia Translational Challenge, which has focused on generating preclinical evidence to identify the most promising repurposed drug candidates for testing in clinical trials. 

The majority of MND clinical trials, including those using repurposed drugs, fail due to lack of effectiveness. 

To improve clinical trial success rates, LifeArc’s funding call focused on ensuring that only the projects with the most robustly supported repurposed drug candidates progress to clinical studies. 

This means strengthening early-stage decision-making with high-quality preclinical data, transparent methodologies, and independent replication of results. 

 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 are 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 bowel conditions like ulcerative colitis, could slow progression of MND.
• Massachusetts General Hospital – Dr Brian Wainger is investigating whether the well-known heart medication digoxin, used to treat abnormal heart rhythms, 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 cells’ 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

Paul Wright, Head of MND & Rare Dementia at LifeArc, said:

This was a truly global search for the most promising ideas, attracting interest from researchers in 31 countries across five continents. The quality of applications was incredible, and after many conversations, including valuable insights from our patient representatives, we agreed on four outstanding projects. We believe these projects give us the best possible chance of finding safe and effective repurposed treatments for MND.

One person who knows the reality of life with MND and the lack of options is Chris, who was part of LifeArc’s 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.

 Prof Paul Lingor from the University of Munich said:

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 Richard Mead from the University of Sheffield said:

The idea for our program came from Dr Peter Richardson, an outstanding scientist who developed MND later in his career. Peter is no longer with us which is just one indicator of the urgency with which we need to find new treatments. Repurposing is one option and the program LifeArc have put together is designed to address the substantial scientific, regulatory and commercial hurdles to advance high quality repurposing candidates to the clinic.

Beyond funding, LifeArc’s science team will work closely with each of the awardee project teams throughout their research. This includes providing scientific advice, supporting the rigorous design of experiments, and facilitating independent replication of results to confirm findings. 

The funded teams will also become part of a new collaborative research network designed to encourage collaboration and knowledge-sharing between researchers. This approach aims to accelerate progress and highlight best practices to strengthen the global approach to drug repurposing in MND. 

This programme is designed to generate the strongest possible evidence on which repurposed drugs should progress into biomarker-guided clinical studies and clinical trial platforms such as EXPERTS-ALS and MND-SMART in the UK and the Healy ALS platform in the US. 

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 such as rare dementia.