Jane Haley, PhD, MND Scotland, Glasgow, UK, shares an overview of the groundbreaking MND-SMART platform trial underway in the UK, which aims to expedite the discovery of effective treatments for motor neuron disease (MND). The charitably-funded Phase II/III trial has a unique, multi-arm, adaptive design that enables the agents being studied to change as new discoveries are made. Several agents will be studied at once, in comparison to a single placebo group, maximizing the proportion of patients receiving an active intervention. In this way, treatments that do not prove to be effective can be dropped and new ones introduced, without the time and cost limitations of launching a whole new study. The agents currently being studied are repurposed agents – memantine, amantadine, and trazodone – reducing the risks of adverse events and avoiding lengthy approval processes if proven effective. MND-SMART will run continuously for years to come, with nearly 500 patients recruited so far. This interview took place at The BNA 2023 International Festival of Neuroscience in Brighton, UK.
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Transcript (edited for clarity)
Well MND Scotland took a leap of faith back in 2018 and provided the funding to set up MND Smart, which is the first platform trial in the UK for a neurological disorder and it’s purely focused around, well at the moment, trialing repurposed drugs to try and find things that will improve life expectancy for people with MND. So it’s been hugely successful. Although it received funding in 2018 from us, it was it actually launched in 2020 right as the pandemic hit...
Well MND Scotland took a leap of faith back in 2018 and provided the funding to set up MND Smart, which is the first platform trial in the UK for a neurological disorder and it’s purely focused around, well at the moment, trialing repurposed drugs to try and find things that will improve life expectancy for people with MND. So it’s been hugely successful. Although it received funding in 2018 from us, it was it actually launched in 2020 right as the pandemic hit. And so it had a slight hiatus in recruitment. But it has been amazing that despite the fact we’ve had a global pandemic, it has recruited according to its schedule and it’s just about to recruit its 500th participant. It’s currently in 19 or 20 sites across the UK. It’s no longer just in Scotland and it’s bringing hope to people where there was none before.
So in the in the ten years prior to this trial, less than ten people in Scotland had been able to participate in a clinical trial. And now we have nearly 500 people participating across the UK and many, many of them are in Scotland. So it’s transformed the landscape for people with MND who want to participate in clinical trials. And more to the point it’s been taking a model from another area. So platform trials have been very common in cancer research and they have transformed the landscape there because what it does is instead of looking at one drug, ramping up a clinical trial, looking at one drug and then closing the clinical trial down, analyzing the results, starting a new clinical trial with a new drug which takes years and years and years. It builds a platform and you build new arms of treatment into that so you can start a treatment arm and you can end it and move people over to a different treatment arm, start a new treatment arm, all keeping the infrastructure in place that allows that. It’s much, much more efficient way of undertaking clinical trials, which also makes it more cost effective.
This clinical trial has been solely funded by donations from people. It’s been funded by MND Scotland and then other charities have come on board to also assist with funding so it doesn’t have government funding. So it needs to be run in a way that actually can be funded by charities at the level that they can afford. And it’s also setting a model for other neurological diseases within the UK. It’s part of the ACCORD group which is trying to encourage this kind of platform trial design amongst other conditions. And I know that Parkinson’s is one area, multiple sclerosis has just launched a platform trial as well, the Octopus trial. So it is gaining traction and I’m very, very proud that MND Scotland was a core initial funder that set that entire project up and that will continue. Platform trials are not short-term things they are enduring by their very nature. So I’m very, very proud that we are a funder and continue to be.