The charity has helped launch four projects, which are aimed at identifying the most promising repurposed drug candidates to treat motor neurone disease.
London-based medical research organisation LifeArc has helped launch four projects aimed at identifying the most promising repurposed drug candidates to treat motor neurone disease (MND).
This targeted programme, part of the charity’s MND & Rare Dementia Translational Challenge, has focused on generating preclinical evidence to identify the most promising repurposed drug candidates for testing in clinical trials.
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 majority of MND clinical trials, including those using repurposed drugs, fail due to a lack of effectiveness.
“This was a truly global search for the most promising ideas, attracting interest from researchers in 31 countries across five continents,” said Paul Wright, head of MND and rare dementia at LifeArc.
“We believe these projects give us the best possible chance of finding safe and effective repurposed treatments for MND,” he added.
Move into clinic trials
The four projects being funded are a project from the University of Munich in cooperation with the University of Ulm and the Istituto di Ricerche Farmacologiche Mario Negri IRCCS in Milan which explores 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; a project from the University of Sheffield which is investigating whether drugs called JAK inhibitors, used to treat conditions like arthritis and bowel conditions like ulcerative colitis, could slow progression of MND; one from Massachusetts General Hospital which 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; and finally a project from University of Sydney which is 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.
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.
