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.
Researchers find a new drug that could be a future treatment for MND
06 July 2026
Researchers from the University of Arizona have discovered a drug that could be tested as a future treatment for MND. The drug, called XL20, has been tested in the laboratory and was found to protect motor neurones from damage in MND. XL20 is designed to reduce the activity of a protein that becomes faulty and toxic in MND, called TDP-43. This protein is found to be faulty in around 97% of people with MND.
The team identified a part of the TDP-43 protein they say is responsible for causing damage to motor neurones in MND. XL20 is thought to stick to this part of TDP-43 thereby blocking the harmful effects to reduce the damage that it causes, while still allowing the protein to carry out it’s usual job. Researchers tested the drug in mouse models of MND and found the drug reduced muscle weakness and extended survival of the mice by about one week.
While these results are promising, the drug is still in very early stages of development and hasn’t been tested in people yet. XL20 needs further testing in the laboratory to see if it should be taken forward for testing in clinical trials in the future. Read more in the press release.
New results announced from previous phase 2b trial of PrimeC
30 June 2026
Neurosense therapeutics has announced new analyses from its phase 2b trial including a follow up extension of drug PrimeC. In the trial, participants were given either the drug or a placebo (dummy drug) for six months, followed by an open label extension (the follow up phase where everyone on the trial gets the treatment) for 12 months. New results suggest treatment with PrimeC significantly reduced the amount of a protein called TDP-43 found in the blood of participants on the drug vs placebo at the end of the 6 month trial and end of the 12 month open label extension.
In 97% of people with MND this protein, TDP-43, becomes faulty and no longer works properly within the neurones causing them to die. The faulty TDP-43 proteins form clumps that become toxic and damage the cells. While we know faulty TDP-43 plays a role in the disease for most people, there is not yet robust scientific evidence that reducing TDP-43 levels in the blood improves the health of motor neurones or is linked to slower disease progression. Measuring levels of TDP-43 in the blood is not a standard measure used to determine how well a drug works in clinical trials.
These results demonstrate PrimeC is acting on MND biology but this doesn’t mean that this treatment is effective. This new analysis has not yet been published which means no peer reviewed data is available. Without peer reviewed data, we cannot be sure results are reliable nor can we draw conclusions. This phase 2b trial was not designed to assess whether the treatment is effective and a larger phase 3 study will be needed to determine if PrimeC could be a treatment for MND. A phase 3 trial of PrimeC is currently in preparation and we welcome the further testing of this drug for MND. PrimeC has not been approved as a treatment for MND and is not available outside of a clinical trial. Read more in the press release or find out more about PrimeC.
New drug targeting UNC13A moves into clinical trials in the UK
24 June 2026
Trace Neuroscience has announced its drug, TRCN-1023, has received approval to begin clinical testing in the UK and the Netherlands. The drug will be tested in a small Phase 1/2 clinical trial called FUNCTION ALS, which forms part of a wider global programme including the LAUNCH-ALS trial in China. TRCN-1023 targets the gene UNC13A that provides vital instructions for making a protein that allows motor neurones to communicate with other nerve cells and muscles.
This trial follows on from original research, carried out by a team at UCL led by Professor Fratta, funded by the MND Association and Medical Research Council through a Lady Edith Wolfson Fellowship. This work led to the discovery that UNC13A levels are associated with disease severity and TDP-43, a protein that doesn’t work as expected in 97% of people with MND and can form harmful clumps inside nerve cells. When TDP-43 stops functioning normally, it can affect the UNC13A gene. This reduces the production of healthy proteins, which cells need to communicate effectively with other nerve cells and muscles. TRCN-1023 is designed to fix the UNC13A gene and help restore the production of functional proteins. TRCN-1023 is entering it's first stage of testing in humans and has not yet been shown to be safe or effective for people with MND. The trial is not yet open in the UK and is still in the set-up stages. We will continue to monitor progress and share updates. Read more in the press release.
New research sheds light on the body’s nerve repair system
01 May 2026
A new study, led by the UK Dementia Research Institute (UK DRI) at University College London, and Kings College London, has found out more about how a natural substance in the body, called Brain Derived Neurotrophic Factor (BDNF), is in involved in repairing axons (also known as nerve fibres). Researchers have known for a long time that BDNF supports the health and survival of brain cells, but it has not been clear how it does this. The researchers used stem cells to grow motor neurones in the laboratory to investigate how BDNF supports the survival and repair of axons in damaged cells.
The researchers found that BDNF activates multiple different repair pathways in the brain cells at the same time, coordinating the body’s built in repair systems to work together. The study suggests that BDNF switches on genes that are important for repair. This controls proteins that help to rebuild the cell’s structure and guide axons to grow back properly. Through this study, the researchers discovered how BDNF sends the signals to activate these repair pathways. Uncovering how BDNF switches on these repair pathways could lead to future research into drugs that target these switches and activate the repair pathways in neurones. You can find out more in the press release.
International research study finds new genetic links to MND
01 April 2026
A major new international study, led by researchers at the University of Utrecht, has uncovered several new gene changes linked to MND. Most gene studies in MND have looked into common gene changes, but research has suggested that some people with MND may have rarer changes. Rare gene changes are harder to find because researchers need to look at genetic information from thousands of people to find them. Researchers in this study created the largest collection of MND genetic data ever to study rare changes, which included using data collected from ProjectMINE which was funded by the MND Association.
The researchers studied the genetic makeup of nearly 18,000 people with MND and over 200,000 people without the disease and identified new genes (called YKT6, HTR3C, GBGT1 and KNTC1) that might be changed in people with MND. The research also added to existing evidence that changes in four other genes are linked to MND (called ARPP21, DNAJC7 and CFAP410). The researchers showed that more people with MND may have gene changes than previously thought, even if the disease does not run in their family. This research highlights the importance of genetic testing in MND and, how studying rare gene changes can open doors to new MND treatments in the future. You can find out more in the paper.
Head of Research at the MND Association, Dr Nick Cole, said:
'We are pleased to see more discoveries that deepen our understanding of the causes of MND, which remains a devastating disease. This work is important because each genetic link identified represents a new potential target for the development of future treatments. We also acknowledge that the generosity of people with MND and their families—who have provided samples for research—has played an essential role in making these discoveries possible.'
Researchers develop new research tool for MND
19 March 2026
Researchers at the University of Essex, funded by the MND Association, have developed antibodies to target disease-related proteins inside cells. Antibodies are proteins that can identify and bind to other proteins inside the body, and where these are faulty, help them to be removed by the immune system. Normally, antibodies are too large to enter cells, so the researchers have created a smaller simplified version called intrabodies. These intrabodies can easily enter cells and stick to key proteins linked to MND and other neurodegenerative conditions. The researchers were able to design 672 intrabodies that can exist within cells.
These intrabodies could be a powerful tool to research disease related proteins that are known to cause damage in MND and will be made available for other scientists to use in their research. They may also be useful in developing and testing new gene therapies as future treatments for MND. The researchers are continuing to test these intrabodies to identify which may show the most promise as future treatments for MND. You can read more about this in a press release or find out more about the study on our website.
Raya Therapeutic announce the name of the drug to be tested in the EXPERTS-ALS platform
09 March 2026
Last year, Raya Therapeutic announced that a drug they are developing had been selected to be tested as part of the EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) platform. They have now announced the name of this drug. RT1999, also known as Smilagenin, has previously been tested for Parkinson’s disease and is known to be safe. Previous studies in cell and animal models of MND suggest that the drug could help control the production of proteins which are protective to motor neurones in MND. Testing this drug in EXPERTS-ALS will help to determine if these promising signs seen in the lab are also seen in people with MND. RT 1999 is expected to be added to the EXPERTS-ALS trial by the end of 2026. You can read more about EXPERTS-ALS here or the press release here.
MND-SMART team announce results for Amantadine drug
03 March 2026
The MND-SMART team have announced that the drug amantadine will be discontinued from testing in the platform trial. The latest review of data collected so far, conducted by independent committees, found no significant improvement in the rate of change in ALSFRS-R for those on the drug compared to those on placebo. This suggests the drug did not slow the progression of MND. The MND-SMART trial is continuing and is currently testing a drug called Tacrolimus. You can read more about this announcement in our news story or find out more about MND-SMART on our website.
Interim results announced for ANQUR, a phase 1/2 clinical trial of QRL-201
24 February 2026
QurAlis have announced interim results from their proof-of-concept phase 1/2 trial of QRL-201. In the trial, participants were given either the drug in one of two doses, or a placebo (dummy drug). The interim results suggest that QRL-201 was safe and well tolerated. The press release also suggests that the ALSFRS-R score declined at a slower rate after six months of treatment for some of the participants, who were those who had a slower baseline rate of disease progression.
QRL-201 aims to restore STATHMIN-2 levels in people living with MND. STATHMIN-2 is a protein that is important for neurone repair and stability of axons, which carry the electrical impulse from the cell body to the end of the neurone. In almost all people living with MND, STATHMIN-2 is significantly decreased.
Following these results, QurAlis are looking to extend the trial in an open label extension, meaning that everyone who was part of the trial will receive QRL-201, regardless of whether they were receiving the drug or the placebo in the original trial. The open label extension has been approved in Canada, and is under review in the UK and the EU. The press release states that QurAlis are preparing to take the drug forward into a key phase 3 clinical trial. This will provide further evidence as to whether QRL-201 could be an effective treatment for MND.
Read more about the trial on our webpage, and more about the interim data in the press release.
Head of Research at the MND Association, Dr Nick Cole, said:
'For people living with MND, it’s natural to feel hopeful when new drugs show potential. The science behind targeting STMN2 is exciting and represents a new direction for ALS treatment. But these are very early days. Only 69 people have been involved in the trial so far, and the interim analyses include even smaller subgroups. We will need larger, longer, and properly powered trials before we know whether QRL 201 can meaningfully slow disease progression.
For now, these results are a positive step forward, and the continuation of the trial is good news, but it’s important not to draw conclusions too soon.
We remain cautiously optimistic and will continue to follow the progress of QRL 201 closely as more data emerges.'
CervoMed announce Neflamapimod to be tested in the EXPERTS-ALS platform
18 February 2026
Neflamapimod, a compound being developed by CervoMed has been selected to be tested as part of the EXPErimental medicine Route To Success in ALS (EXPERTS-ALS) platform. EXPERTS-ALS is a platform which aims to rapidly test drugs to assess their potential to slow MND progression, by measuring a marker of disease activity within the body, called neurofilament light chain (NfL). If the treatment is found to significantly reduce levels of NfL in the blood across the whole group of participants it suggests there are early signs of benefit and the drug should be priortised for further testing. Neflamapimod, which has shown promising signs in models of MND, has been tested in clinical trials for Alzheimer’s disease and a form of Dementia so is already known to be safe. It is thought that the compound could help to reduce inflammation in the brain and improve communication between cells, both of which are known to become faulty in MND. Testing this drug in EXPERTS-ALS will help to determine if promising signs are seen in people with MND. Read more in the press release.
First participant dosed in phase 1/2 trial of VTx-002 for MND
9 February 2026
VectorY Therapeutics have announced that they have dosed their first participant in the Phase 1/2 PIONEER-ALS trial. This is a first in-human, randomised study investigating the safety and tolerability of VTx-002 in people living with MND. VTx-002 is a gene therapy that is designed to target clumps of TDP-43 protein, which form in about 97% of people with MND, and kick start the body’s immune system to remove them from the neurones. The gene therapy delivers the instructions for our cells to make an antibody which sticks to the toxic clumps of TDP-43 and marks them for removal by the immune system. It is thought that a single treatment of this gene therapy could mean that the body produces this antibody for years.
The study will test two doses of VTx-002 in 12 people with MND, with participants being randomly assigned to a dose when they are enrolled into the study. Participants will be given a single injection of the gene therapy into a large fluid-filled space at the base of the brain. They will then be followed up through clinic visits, remote monitoring through calls, tests and assessments to monitor the ongoing effects of the drug. PIONEER-ALS is currently recruiting in the US and Europe (clinicaltrials.gov). Read more in the press release.
Researchers suggest a link between air pollution and MND
20 January 2026
A new study, led by the Karolinska Institute in Stockholm, Sweden, has suggested that long-term exposure to higher levels of air pollution could be linked to the development and progression of MND. The researchers studied clinical data from 1463 people with MND and 9078 healthy people in Sweden and air pollution levels across the country for a period of 10 years before people were diagnosed with MND.
In this study, the researchers found an association between long-term exposure to higher air pollution levels and a small increased risk of developing MND. They also found an association between long-term exposure to higher air pollution and faster disease progression. These results were not found to be consistent across all subtypes of MND and further research is needed to understand more about how these associations might contribute to the development and progression of the disease.
Previous research into air pollution and MND has found conflicting results, and while this study adds to the evidence, strong conclusions about whether air pollution is linked to MND still can’t be made. It is known that the majority of cases of MND are likely to occur through a combination of genetic, environmental and lifestyle factors and not any single factor alone. While exposure to high levels of air pollution may contribute, it’s unlikely to be the only factor involved in the development and progression of MND. You can read more about the results in the published paper.