eople with motor neurone disease (MND) could be offered drugs to treat the disease faster under a new initiative.
The scheme across the UK will screen candidate drugs rapidly in a bid to identify which should be tested in larger clinical trials.
The study, called Experimental medicine Route To Success, or Experts-ALS, will take people at the point of diagnosis with MND and offer them the opportunity to take one of three drugs being tested to treat the condition.
Known as “repurposed drugs”, the medicines on offer are already be licensed for use on other diseases, but will have some data suggesting they could also treat MND.
Biomarkers is patients’ blood, known as neurofilament light chain (NFL), will then be tested at intervals over three to six months, with continuous monitoring at group level.
According to researchers, blood NFL level is “the most promising biomarker in MND to date”.
If the overall group NFL is not changing significantly or rising in response to the drug, researchers will assume it is not slowing the disease.
However, if the biomarker is found to the falling, then the medicine will be prioritised for larger clinical trials so the potential to treat MND can be fully tested.
It is hoped patients can begin taking part in the study by summer 2024.
As well as identifying new treatments, the initiative, which involves 11 MND centres across the UK, will also help develop the next generation of experts in the field.
Motor neurone disease has a devastating impact on people’s lives, and I continue to work with leading scientists to cut red tape and accelerate research
The Experts-ALS research team is led by Professor Martin Turner at the University of Oxford and UK MND RI co-director Professor Chris McDermott at the University of Sheffield.
Prof McDermott said: “MND is a cruel and devastating disease and we need new approaches to identify more effective treatments to help patients.
“Experts-ALS is a pioneering project to prioritise the drugs which have the best chance of success in halting the progression of this terrible degenerative disease.
“Over five years, we will be able to screen drugs faster, on a larger scale and identify which ones should proceed into phase three trials based on signals found in people living with MND.”
It comes after the Government announced that £36.5 million of its £50 million, five-year commitment to MND research had been allocated to projects.
It said it hopes the cash “will enable faster progress towards treatments” by backing cutting-edge research.
Health Secretary Steve Barclay added: “Motor neurone disease has a devastating impact on people’s lives, and I continue to work with leading scientists to cut red tape and accelerate research.
“I want this vital funding to get to where it needs to be as quickly as possible so early phase studies can lead the way in helping us find new and effective treatment for those people who are living with this debilitating disease.
“We know we are not there yet, but we will find a cure and we’re doing everything we can to make it happen.”
In December 2022, the Department of Health and Social Care (DHSC) was accused of withholding research funding for MND.
It had initially announced its £50 million commitment in 2021 but, after the death of former Scotland rugby international Doddie Weir last November, the Motor Neurone Disease Association said none of the money was in the hands of researchers.
Rugby star Kevin Sinfield also hit out at the Government on Twitter, saying he “can’t see any reason” why scientists could access the funding, while his former Leeds Rhinos team-mate Rob Burrow, who has MND, accused ministers of holding back the cash.
