Here you can find the latest MND research news from around the world. Stay tuned to find out about clinical trial outcomes, breakthroughs in the lab, interesting research papers and more.
MIROCALS trial of IL-2 results are published
09 May 2025
Full results from the MIROCALS trial, testing an existing drug called IL-2, have been published in the Lancet. Overall, the trial failed to meet it’s primary aim of showing that IL-2 is an effective treatment for MND for the whole trial population. When the whole trial population was analysed, treatment with IL-2 showed a modest increase in survival (12%) for those on the treatment during the study, but this was found not to be statistically significant.
However, the researchers have suggested that it may be beneficial for a subgroup of people with MND. This subgroup was based on levels of a marker of disease activity called neurofilament. Those with low neurofilament levels , suggesting that they had slower disease progression, were found to be most likely to benefit from IL-2 treatment. The researchers found that those with low neurofilament and taking IL-2 were 18% more likely to be alive at the end of the trial compared to those with slow progression who were on placebo. They also reported a 23% decrease in the rate of change in ALSFRS-R between those with low neurofilament and on IL-2 and those with low neurofilament on placebo. The researchers suggest that IL-2 is not an effective treatment for all people with MND, but that it may have a modest benefit for some people with MND who have slower disease progression, as measured by levels of neurofilament.
You can read more about the results in our blog, find out more IL-2 on our website or read our latest update.
New research identifies CREB3 protein as a possible target for new treatments
10 April 2025
Researchers from the University of Strasbourg have uncovered a possible new target for future potential treatments. They suggest that a rare gene change in the CREB3 gene might be linked to a reduced risk of developing MND, and that people with MND who have this gene change have much slower disease progression and survive for longer than those who don’t. The gene change is thought to lead to very high activity of the CREB3 protein, which is involved in controlling which genes are switched on and off inside cells.
The researchers looked at genetic data from 1,873 people with MND and 3,926 healthy controls. They found that the rare gene change in CREB3 was associated with a 39% lower risk of developing MND. The researchers also found that those with MND who had the rare gene change live an average of a year longer than those without it and have significantly slower disease progression. This suggests that increasing the activity of the CREB3 protein may be a good target for developing new potential treatments for MND. This research is in the very early stages and more research is needed to understand more about the role that CREB3 plays in cells in the disease and the effect that increasing it’s activity may have on neurons in MND. You can read the publication here.
Lighthouse II trial of Triumeq is terminated
07 April 2025
TRICALS have announced that the Lighthouse II phase 3 trial of Triumeq has been stopped. The trial was designed to include an interim analysis after a certain amount of time to assess whether the drug was showing signs of benefit. Unfortunately, the interim analysis found no difference in survival measures between those taking the drug and those on placebo (dummy drug). This suggests that Triumeq is not beneficial for people with MND. Following these results, the decision was made to stop the trial and participants on the trial have been asked to stop taking the drug immediately. You can read more about this in a press release.
Lighthouse II was testing Triumeq which is an existing drug used to treat HIV. It recruited over 400 participants across Europe, the UK, Australia and New Zealand. The MND Association co-funded this study. You can find out more about the trial on our website.
New research suggests that Neurofilament light chain could cause inflammation in the brain
19 March 2025
New research from scientists in San Francisco has found that a marker of disease activity, called Neurofilament Light chain (NfL), could play a role in disease progression in MND. NfL is a protein which is released into the blood and spinal fluid when neurons are damaged and die. This study has suggested that the release of NfL may trigger the action of brain immune cells called Microglia. Microglia are cells which usually scan the brain for signs of infection and injury and cause inflammation to help to remove the threat. Previous research has suggested that these cells become overactive in MND and cause inflammation which contributes to the progression of the disease.
The researchers found that completely removing NfL in a mouse model of MND led to reduced activity of microglia, lowered inflammation and delayed the onset of symptoms. This research suggests that NfL could play a role in disease progression and symptom onset when it is released from neurons. Further research is needed to uncover more about how NfL activates microglia and to investigate whether NfL may be a good target for the development of new potential treatments. You can read the publication here.
Denali Therapeutics announce further results from the trial of DNL343
06 March 2025
Earlier this year, Denali Therapeutics released top-line results from the DNL343 arm of the HEALEY Platform trial. Top-line results revealed that the drug did not meet it’s primary or secondary endpoints of the trial. Following these results, Denali has further analysed the data from the HEALEY Platform trial and an open label extension, where everyone in the trial got the chance to take the drug for 28 weeks. They looked into whether the drug showed signs of benefit in subgroups of people with MND and if it had any effect on the level of a marker of disease activity called Neurofilament (NfL), as well as if taking the drug for longer might make a difference. The results of the additional analysis have found that there was no effect on the levels of NfL during both the trial and open label extension. This suggests that the drug isn’t able to slow the progression of MND. The open label extension of DNL343 will now be stopped and Denali have said that the full data from this analysis will be presented at a later date. You can read more about this in a press release.
New research sheds light on how ‘healthy fats’ are affected in MND/FTD
25 February 2025
A new study, led by the UK Dementia Research Institute (UK DRI) at University College London, and the UCL Institute of Healthy Ageing, has found that levels of healthy fats such as omega-3 are reduced in MND and FTD linked to a change in the C9orf72 gene. The researchers looked at fruit fly models, cell models and tissue from people with MND/FTD with a change in the C9orf72 gene. These reduced levels are thought to be due to changes in the way that healthy fats or fatty acids are produced and processed in cells in the disease. The researchers also found evidence to suggest that these changes may be present at the very early stages of disease.
The study also showed that increasing two types of fatty acids in the diets of fruit flies with C9orf72 linked MND/FTD led to a small improvement in survival. Survival was found to improve further when the researchers used gene editing to increase the activity of some genes involved in producing and processing fatty acids in neurons. This increased levels of healthy fats in neurons and helped to reduce damage to the cells. This research is promising, early stage research but further testing is now needed to determine which fatty acids should be focused on, in what amounts they could be effective and how they can be delivered to the neurons. You can find out more in our news story.
Results announced from Phase 2 trial of Trimetazidine
08 February 2025
Researchers have published their findings from a Phase 2 trial, called MetFlex, which tested Trimetazidine in people with MND. Trimetazidine is an existing drug that is used to correct changes in metabolism in angina. The trial, which tested the drug in 21 people over 12 weeks, showed that Trimetazidine is safe and well tolerated by people with MND. The researchers also found that markers of oxidative stress (which contributes to cell damage) and resting energy expenditure (the energy our bodies use when resting) were significantly decreased during the trial. This suggests that the drug worked as the researchers thought within the body. The MetFlex trial was a small, open label trial meaning that everyone on the trial took the drug and there was no placebo group. The findings are promising and demonstrate the need for a larger, placebo controlled trial to test whether Trimetazidine could slow down the progression of the disease. You can read more in the publication.
InFlectis BioScience announce results of Phase 2 trial of IFB-088
06 February 2025
Results have been announced for a Phase 2 trial testing IFB-088 in 51 people with bublar- onset MND, where initial symptoms are changes in speech and difficulty swallowing. Participants were given either IFB-088 alongside riluzole or a placebo (dummy drug) alongside riluzole for six months. The trial found that IFB-088 was safe and well tolerated by people with MND and the company have suggested that the drug also shows signs of benefit for people with the disease. InFlectis BioScience said that trial showed signs of slowing functional decline, with a significant difference in ALSFRS-R scores between those on the drug and those on the placebo. Biomarkers measured in the trial also helped to confirm that the drug works as thought within the body. These results pave the way for a larger clinical trial to test IFB-088 in more people with MND. You can read more in the press release.
IFB-088 is a new drug which is designed to boost the cell’s integrated stress response that acts like a shield against things that cause the cell stress. It is also thought to reduce the production of toxic chemicals that cause damage to the cell, which may help to protect the neurons from damage and death.
EXPERTS-ALS Drug Screening platform opens for recruitment
31 January 2025
The EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) drug screening platform has begun recruiting participants. The platform, which aims to rapidly test promising treatments for MND, is now open at 6 sites across the UK and it is hoped that 5 more sites will open throughout the year.
The platform is designed to rapidly test promising treatments in people with MND to see if they show signs of benefit. It will initially test repurposed drugs (drugs that are already used for other conditions) to see if levels of a marker of neuron damage, called neurofilament light chain, decrease with treatment. EXPERTS-ALS will help to quickly prioritise which drugs are the most promising and should be tested in larger phase 3 clinical trials to test whether they are beneficial for people with MND. EXPERTS-ALS is funded by the MND Association, National Institute for Health & Care Research (NIHR), MND Scotland, My Name’s Doddie Foundation and LifeArc. You can find out more about EXPERTS-ALS and how to take part on our website and blog.
FDA have lifted clinical hold on phase 1 trial of AMX0114
22 January 2025
Amylyx Pharmaceuticals have announced that the Food and Drug Administration (FDA) have lifted the clinical hold they placed on AMX0114, a potential treatment for MND. The clinical hold meant that the FDA had issued an order to pause the start of a trial of as they required additional information from Amylyx about the therapy.
This hold has now been lifted, meaning that the FDA have given the go ahead for a trial to begin. Amylyx have said they are working to open sites in the US for a phase 1 trial of the potential treatment. This Phase 1 trial, called LUMINA, will test AMX0114 in 48 people with MND to see whether it is safe, works in the body as designed and test different doses of the therapy. This trial will be recruiting in the US and Canada. You can read more about this in a press release.
AMX0114 is a potential gene therapy for MND and is an anti-sense oligonucleotide which targets the instructions for a protein called calpain-2. Calpain-2 has been found to be increased in people with MND and this increased activity is thought to be involved in causing damage to neurons. AMX0114 is designed to decrease levels of calpain-2.
Top-line results announced for the DNL343 and ABBV-CLS-7262 arms of the HEALEY Platform trial
6 January 2025
Top-line results from both the DNL343 and ABBV-CLS-7262 regimens of the HEALEY Platform trial have been announced.
In the DNL343 arm of the trial, top-line results showed that it did not meet it’s primary or secondary endpoints. This suggests that there was no significant change in ALSFRS-R scores (from baseline to 24 weeks) for those who received the treatment compared to those who received placebo (dummy drug). The secondary endpoints of muscle strength and respiratory function were also not found to change significantly. You can read more about this in a press release.
The ABBV-CLS-7262 arm tested two different doses of the drug (primary dose and experimental high dose) and found that the primary dose did not meet it’s primary or secondary endpoints. At the high dose the primary endpoint of change in ALSFRS-R scores (from baseline to 24 weeks) was not met, but there were signs of possible benefits for secondary endpoints of muscle strength and respiratory function. You can read more in the press release.
New compound shows early positive effect in lab cells
03 January 2025
Ellorarxine is a drug compound developed by Nevrargenics, a UK based neuroscience company. Ellorarxine is not yet a medicine and has not been tested in humans. The compound targets a physiological process called retinoid signalling, which plays a critical role in brain development and repair, though it’s role in neurodegenerative diseases, including motor neurone disease (MND) is not fully understood. The research published and presented by the company in October 2024 suggested the drug demonstrated a number of potentially positive effects on cells grown in the laboratory, including protective antioxidant and anti-inflammatory responses. There is no published data to show whether the drug has the same effect in humans or, indeed, whether it is safe for use in people. So while these early stage results are interesting, further research is needed. Nevrargenics has sought advice from the MHRA about clinical trial design. We are in touch with the team at Nevrargenics and will of course share updates with our community in the future.
MND-SMART team announces interim analysis results of Amantadine
18 December 2024
The MND-SMART team have announced that the drug amantadine will continue to be evaluated as part of the platform trial. This interim analysis of the latest data, conducted by independent committees, found no safety concerns for amantadine. This means the drug will continue to be tested in the trial to determine whether it is of any benefit to people with MND. MND-SMART has also confirmed the launch of a fourth arm in Edinburgh in January 2025. You can read more about this announcement in our press release or find out more about MND-SMART on our website.
New online platform improves access to participate in UK MND research studies
12 December 2024
Researchers from Sheffield have announced the launch of a new remote monitoring platform that is designed to make it easier and quicker for people with MND across the UK to take part in MND research. The platform, called Telehealth in MND-Research or TiM-R, will bring all of the UK MND studies into one place for people with MND to see what studies are happening and contact researchers to register interest in taking part.TiM-R will give users updates on how studies are progressing and allow participants to complete questionnaires and submit data remotely, rather than having to travel to the clinic. MND researchers can also use this information to understand what studies users may be eligible for and engage with potential participants.
This platform, funded by the MND Association along with LifeArc, My Name’5 Doddie Foundation and the National Institute for Health and Care Research (NIHR), is part of the work of the UK MND Research Institute (UKMNDRI) to accelerate the search for more effective treatments for MND. You can read more about TiM-R in this press release or sign up for the platform here.
Dazucorilant clinical trial does not meet primary endpoint
11 December 2024
Corcept Therapeutics reported that the phase 2 clinical trial of Dazucorilant (DAZALS) did not meet its primary endpoint. This suggests that there was no significant change in ALSFRS-R (from baseline to 24-weeks) for those who received the treatment compared to those who received the placebo (dummy drug). The open label portion of the trial will continue until March 2025 to assess long-term effects of the drug. Further details on the trial outcome and open label extension are expected in the coming months. You can read more about the results in a press release or find out more about Dazucorilant here.
Top-line results announced from cardinALS Phase 2 trial of Utreloxastat
27 November 2024
PTC Therapeutics have announced the top-line results of the cardinALS Phase 2 trial testing Utreloxastat in people with MND. The trial did not meet primary or secondary endpoints. There was no significant change in ALSFRS-R (from baseline to 24-weeks) for those who received the treatment compared to those who received placebo (dummy drug). This suggests that Utreloxastat is not beneficial for people with MND. The data also showed no significant reduction in a marker of nerve damage called neurofilament light chain (NfL) when comparing Utreloxastat to placebo over 24-weeks. This drug was found to be safe and well-tolerated. You can find out more about the results here or read more about the cardinALS trial here.
PTC Therapeutics have announced that there are no plans for further development of Utreloxastat for MND.
NICE have announced they will change how Tofersen is assessed for access via the NHS
18 November 2024
The National Institute for Health and Care Excellence (NICE), who decide which treatments become available on the NHS, has announced that they have changed their decision and will now appraise Tofersen through the Highly specialised Technologies (HST) route.
Previously, NICE had decided to use the Single Technology Appraisal (STA) route to assess the drug, which meant that it was highly unlikely that Tofersen would become accessible to people with SOD1-MND through the NHS. This change means that Tofersen will now get a fair assessment to decide if it will be accessible through the NHS. You can read more about what this change means here.
Tofersen is a gene therapy for around 2% of people with MND who have a change in a gene called SOD1. You can read more about Tofersen here.
New research project investigates potential link between professional football and MND
13 November 2024
A new research project has been announced which is investigating whether there is a link between playing professional football and developing MND. The research, funded by the MND Association, My Name'5 Doddie Foundation and the Darby Rimmer MND Foundation, is a collaboration between researchers in the UK and Italy. Professor Ammar Al-Chalabi of King’s College London and Dr Elisabetta Pupillo of the Mario Negri Institute for Pharmacological Research in Milan will analyse death certificates from thousands of footballers who played in Series A and B in Italy and those who were part of the Professional Football Association in the UK to discover whether they had a higher risk of dying from MND than the general population. You can read more about this project on our website.