We have a clear mission: to accelerate the discovery of effective treatments for motor neuron disease (MND). To do this, we’re pursuing a range of scientific strategies, including one that may sound simple but holds great potential: repurposing existing drugs.
Repurposed drugs, already approved for other conditions, are being explored to potentially bypass early hurdles in drug development and move swiftly towards testing treatments that could bring hope to those affected by this condition.
Why repurposed drugs?
Drug development is a long, complex and costly process. It can take more than a decade and, on average, £1 billion to bring a brand-new drug from concept to clinic.
For MND, that timeline is too long and repurposed drugs can offer a smarter, faster path.
Given that these drugs have already been approved for other diseases, we have a wealth of knowledge about their safety, mechanisms of action, and how the body metabolises them.
For instance, Amantadine is a strong example. First used as an antiviral, it was later successfully repurposed to help manage symptoms in Parkinson’s disease, showing how existing drugs can find new uses in treating other conditions.
A strategic part of our research portfolio
Repurposed drugs are not a shortcut but a strategic, evidence-based approach to accelerate progress.
Many of the most promising clinical trials in MND today are testing repurposed drugs, often supported by insights from stem cell models, genetic studies, or biomarkers that point to relevant disease pathways.
As part of our coordinated research programme, we’re investing in the infrastructure, tools and expertise needed to run high-quality clinical trials of repurposed drugs.
These include platform trials such as EXPERTS-ALS and MND SMART, which test multiple treatments simultaneously. MND SMART is the most inclusive platform trial, and almost all patients are eligible to participate. EXPERTS-ALS has a groundbreaking approach to testing drugs that could significantly accelerate the discovery of effective treatments for MND.
Our recent blog explains why we’re investing in platforms like EXPERTS-ALS, and how this approach could help fast-track more options into trials for people with MND.
A united response
By working together across the UK and with international partners, we are creating the conditions for faster progress, from lab to clinic.
Our co-director, Professor Ammar Al-Chalabi, emphasised why researching repurposed drugs is so important: “MND is a complex disease, and there will likely never be a single silver bullet. That’s why we support a broad research pipeline, one that includes drug repurposing and cutting-edge investigations into the biology of the disease.”
Repurposed drugs are a vital part of the search for effective MND treatments. They offer a faster route to trials, grounded in evidence and guided by collaboration. We remain committed to supporting this work and to keeping the MND community informed every step of the way.
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