Here you can find the most up to date research news. Stay tuned to find out about clinical trial outcomes, breakthroughs in the lab, interesting research papers and more.
MND-SMART stops trialling trazodone and memantine due to lack of benefit
28 September 2023
The independent trial committee for the MND-SMART clinical trial recently reviewed the stage 2 interim analysis for memantine and trazodone. It was decided testing should not continue for either drug as it is highly unlikely that the either drug will show benefit for people living with MND. This decision was based on the fact that there was no significant improvement in the rate of change in the ALSFRS-R scale compared to the placebo – indicating no change in the rate of progression of the disease. As a platform trial, MND-SMART allows for drugs to be removed quickly if they are not showing any benefit. Platform trials also allow for the easy addition of potential new drugs into the trial. There are plans to add an additional three drugs into MND-SMART over the next couple of years.
MND-SMART is still trialling amantadine, which was introduced into the trial in April 2023. There will be an opportunity for people who have been on the trial to re-join MND-SMART and be randomised into the other arms of the trial. You can find out more on MND-SMART’s website.
FDA Advisory Committee vote against the approval of NurOwn in the US
28 September 2023
BrainStorm has announced the outcome of the Food and Drug Administration (FDA) Advisory Committee meeting on NurOwn as a treatment for MND. Overall, when asked the question “Does the data presented demonstrate substantial evidence of effectiveness for the treatment of mild to moderate ALS?” the panel answered no. The panel voted 17 to 1 (and 1 abstain) that the evidence presented did not demonstrate that NurOwn is an effective treatment of mild to moderate ALS. You can read more about the Advisory Committee vote here.
Although the FDA considers the recommendations of its advisory committees, the recommendations of the panel are non-binding. The final decision regarding the approval of the Biologics License Application for NurOwn will be made by the FDA by 8 December 2023. You can read more about NurOwn here.
New funding scheme aiming to accelerate MND research launched
28 September 2023
A new programme aiming to accelerate the pace of research into MND has been launched today by Dementias Platform UK. The MND Translational Accelerator Programme aims to help develop discoveries from the laboratory into potential new treatments, also known as translational research. Researchers can apply for a slice of £6 million funding as part of the Programme when applications open in October 2023. This £6 million investment, funded by the Medical Research Council and the National Institute for Health Research (NIHR), is part of the government’s Department of Health and Social Care’s £50 million commitment to research in MND. Head to the website to learn more about the funding and how to apply.
New investment into MND Association funded research could help take new potential treatment into clinical trial
27 September 2023
Research that was initiated by a research grant funded by the MND Association and is currently being supported through a joint project with LifeArc has led to a spinout company from the University of Sheffield. This spinout company, called Crucible Therapeutics, has recently received £5 million investment from Northern Gritsone and Argobio Studio. Crucible Therapeutics aims to develop new potential gene therapies for the most common genetic form of MND and FTD, known as C9orf72.
Funding from the MND Association and LifeArc is currently supporting the development of the gene therapy at the University of Sheffield. The therapy aims to reduce the effect of a protein, known as SRSF1, which has been found to cause toxic RNA molecules to move to the wrong place within the cell. It is hoped that preventing the RNA molecules from moving to the wrong place will have a therapeutic effect in people with MND/FTD. Crucible Therapeutics will work in collaboration with the University of Sheffield and the Cell and Gene Therapy Catapult (CGT Catapult) in the development of this gene therapy. The new investment aims to help push this work into early clinical trials. You can read more about the research involved in this project here or more about the new investment here.
Additional data from CNM-Au8 clinical trials suggests potential survival benefit
27 September 2023
Clene Nanoscience has reported additional data from both clinical trials of CNM-Au8, including the RESCUE-ALS trial and the HEALEY ALS Platform. The additional data from the RESCUE-ALS trial includes long-term follow-up (2 years) from the open-label extension part of the trial. The open-label extension is where everyone involved in the trial gets access to the treatment, regardless of whether they received the placebo in the randomised part of the trial. This data suggested a significant average survival benefit of 19.3 months for those who initially received the treatment compared to placebo. The full results are expected to be published soon. You can read more about the RESCUE-ALS results here.
The data from the HEALEY ALS Platform trial also highlighted a potential survival benefit of CNM-Au8. This data is described as ‘post hoc’ which means this analysis was completed after the initial data from the trial was seen by the researchers. Post hoc analysis normally allows researchers to gain additional information which could then be further confirmed in another clinical trial. The researchers used the PRO-ACT database, which is a large database of information from people living with MND in the United States, and compared it to the survival data in the trial. This comparison showed a significant improvement in survival, with a 49% decrease in the risk of death. This means that more people were still alive at the end of the trial who received treatment compared to the survival data seen in the real-world database. You can read more about the HEALEY Platform trial results here.
CNM-Au8 is an oral-suspension of gold nanocrystals, which has been shown to improve motor neurone survival in laboratory studies. You can read more about CNM-Au8 here.
New phase 1 clinical trial tests a potential device which may slow progression of MND
14 September 2023
PathMaker has announced that the launch of a phase 1 trial to test their device called the MyoRegulator for the time in people with MND. This device aims to reduce hyperexcitability which is an abnormal and more frequent sending of electrical signals and this can cause damage to the neurons over time. It is thought that reducing hyperexcitability may help to preserve motor neuron health and slow disease progression. MyoRegulator is non-invasive and is delivered through the skin to send messages to neurons in the spinal cord.
The phase 1 trial is an open label trial, meaning that everyone in the trial will test the device, and will recruit people with MND in the US to test the safety of this device and see whether it may be practical to use and beneficial for people with MND. You can read more here.
Researchers awarded first funding from the MND Association's Pre-Fellowship scheme
12 September 2023
Two UK researchers have been announced as the first to receive funding through the MND Association's new Pre-Fellowship scheme. The first-of-its-kind Pre-Fellowship Scheme for MND is funded by the MND Association and administered by MND Scotland. Dr Alannah Mole, University of Sheffield, and Dr Emily Carroll, University of Oxford have been selected to receive the funding. The scheme provides 12-18 months of 'pump priming' funds to bridge a funding gap and aims to help retain early career researchers who have chosen to investigate MND. This scheme will allow the award holders to gather data needed to show the potential of their research, which could lead to longer-term fellowships. Dr Mole's research will examine the sequence and timing of events that ultimately lead to nerve cell failure in MND. While Dr Carroll will look at existing drugs, which are currently used to treat other conditions, to see whether they could be effective in the treatment of MND. You can read more about the research projects here.
New treatment candidate shown to reduce inflammation in MND
7 September 2023
A new potential therapy called AB126, being developed by Aruna Bio, is designed to reduce inflammation and promote motor neuron protection and survival. This therapy consists of exosomes which are small packets of proteins and other compounds needed for cells to communicate with each other. The AB126 exosomes are made from brain stem cells and is thought to increase the growth and repair of neurons.
In models of MND, AB126 was found to reduce inflammation and a biological marker of neuron damage known as Neurofilament light chain (NfL). This early testing shows promising results for continuing to develop and test AB126 to fully understand whether it may be beneficial as a treatment for MND. You can read more here.
Replacement motor neurones could restore muscle function in MND
22 August 2023
Researchers from University College London have been able to use replacement motor neurones in mice with nerve injuries. These replacement motor neurones can then be activated by light and improve muscle function. Researchers found that the replacement motor neurones needed regular activation with the light to help start to improve muscle function. This research suggests that damaged motor neurones can be bypassed, and muscles can be activated by using replacement motor neurones. The researchers state there are still many challenges to be overcome before this approach could be used in people with MND, including whether attaching the replacement motor neurones would work with human motor neurones. You can read more here.
Update from clinical trial investigating daily dose of Oral Edaravone
7 August 2023
Mitsubishi Tanabe Pharma America have announced that the Phase 3b clinical trial investigating a new dosing regime for Oral Edaravone (MT-1186) will be discontinued. Oral edaravone is already approved for the treatment of MND in the US with a on/off dosing regimen administered in 28-day cycles. This trial was designed to determine if taking a daily dose of oral edaravone would be more beneficial for people with MND. Pre-planned interim analysis from the trial showed that if the trial continued there is low chance that the daily dose would be more beneficial than the on/off 28-day cycle dose. The trial used the ALSFRS-R scale to measure the efficacy of the drug. While the trial investigating this dosing regime has been stopped, preliminary findings suggest that the efficacy of the on/off 28-day cycle dose is consistent with the Phase 3 trial which supported the FDA approval. You can read more here or find out about the Phase 3b trial here.
In Europe, a Phase 3 trial (ADORE) is testing a different formulation of Oral Edarvone, known as FNP122. This trial is also testing a once-daily dose of the potential treatment. The trial has closed for recruitment in 2022 and topline results are expected in 2024. You can read more about the ADORE trial here.
Immune cells could be a target for MND therapy
2 August 2023
Researchers have found that a protein called alpha-5 integrin is found at consistently high levels in immune cells found close to damaged motor neurones in people with MND. When researchers used an antibody to block the protein, in mouse models, they found it slowed disease progression and improved motor function.
Immune cells are essential for maintaining the health of the brain and spinal cord, but can be found to be overactive in MND, causing inflammation and damage to motor neurones. The researchers found that the immune cells which had high levels of alpha-5 integrin also produced more inflammatory molecules. Integrins are found on the surface of cells and are involved in helping cells attach to nearby cells. They also receive and transmit signals that tell the cells how to behave. The researchers proposed that the immune cells, known as macrophages, with high levels of alpha-5 integrin may recruit other immune cells and worsen inflammation. Further work is now needed to gather more evidence on the benefit of the antibody. You can read more here.
New Research Project to Investigate Link Between Traumatic Brain Injury and Increased MND Risk
26 July 2023
The MND Association, along with MND Scotland and My Name’5 Doddie Foundation is funding a new research project exploring whether traumatic brain injuries, including those that may occur in sport, can lead to an increase in the risk of developing MND. This new project will be led by Professor William Stewart and team at the University of Glasgow.
Almost 4 million existing and anonymised electronic health records from a UK population database (the Clinical Practice Research Datalink) will be analysed to understand whether there is a relationship between traumatic brain injury and MND. Understanding more about the risk factors for MND could help identify strategies to reduce the risk of developing MND and ways to detect it earlier. You can read more here.
Experimental drug candidate shown to target key mechanisms in ALS/FTD
24 July 2023
Athira Pharma has announced that their experimental drug candidate, ATH-1105, shows promise in reducing inflammation, neurodegeneration, and target key drivers of ALS/FTD in early laboratory research. ATH-1105 activates the HGF-MET system, which helps to protect neurones from damage by promoting growth, repair and survival.
In models, ATH-1105 was shown to protect neurones from damage, which was highlighted by the reduction in neurofilament light chain, a marker of neuronal damage. A key driver of ALS/FTD is the buildup of a protein called TDP-43, which happens in around 97% of all ALS cases. ATH-1105 was also found to help reduce the buildup of TDP-43. This research shows promising results in early laboratory work, but further work is now needed to understand how this could translate into people living with MND. You can read more here.
Researchers find more molecules in unexpected locations in MND
21 July 2023
Researchers at the Francis Crick Institute and University College London have found hundreds of molecules in the wrong place in MND nerve cells. The molecules affected are mRNA, which are the instructions to make proteins, and proteins, which play a vital role in tasks in every cell in the body. Previous research has shown that a few proteins, including one called TDP-43, are found to relocate from the control centre of the cell (nucleus) to the body of the cell (cytoplasm) in MND nerve cells.
This new research looked at two cell models with genetic changes associated with MND (TARDBP and VCP). The researchers found hundreds more molecules that relocate in the disease. The relocated molecules were also found to interact with each other more, compared to those in the right place, with the researchers suggesting that the molecules may drag each other with them, creating a domino effect.
A potential drug candidate that may help stop the molecules from ending up in the wrong place was also found, but further work is needed to understand the impact this could have on the disease. You can read more here.
Researchers find using a hybrid design in clinical trials could reduce number assigned to placebo
11 July 2023
Researchers at University Medical Centre Utrecht have found using a hybrid clinical trial design can help to lower the number of participants assigned to the placebo (dummy drug) in MND clinical trials. Hybrid designs use a placebo group alongside data from a carefully selected group of people who are not taking part in the clinical trial. This could allow for less people to be assigned to the placebo group, which can be considered unethical.
The researchers used a previous clinical trial to test the feasibility of the approach. When comparing data from the hybrid design to the standard design, the researchers were able to determine that the treatment had the same effect, but with more precision. This means that the same outcome from the trial was found using less people within the trial, including less people who received the placebo. This approach could help to accelerate the development of new treatments, but further work is needed to check this approach can be used across a variety of different clinical trial scenarios. You can read more here.
New project announced to test combinations of existing drugs for the treatment of MND
30 June 2023
A new project, led by Professor Siddharthan Chandran through the UK Dementia Research Institute (UK DRI), aims to identify combinations of existing drugs that could be used together to treat MND. As MND is a complex disease, it is thought that a variety of drugs targeting multiple biological pathways may be needed to reduce, and potentially stop, damage to neurones. This project will use artificial intelligence and models of MND to identify and test promising drug candidates. The top candidates will then be tested in pairs in models of the disease. It is hoped that this approach will lead to testing the most promising combinations of drugs in clinical trials, such as MND-SMART. The £3.3 million project is funded by medical research charity LifeArc. You can read more here.
AMX0035 receives negative opinion from European Medicines Agency Committee
23 June 2023
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has announced a negative opinion for the conditional approval of AMX0035 (Albrioza). The CHMP panel reviews the scientific evidence and forms an opinion on whether new drugs should be approved in Europe. The committee based their review on data from the phase 2 CENTAUR trial. Amylyx, the pharmaceutical company behind AMX0035, have announced that they will request a re-examination. This process will take approximately 4 months. You can read more in a press release.
The EMA determines approvals for the EU. Since Brexit, a process (known as the European Commission Decision Reliance Procedure) has been put into place so that the regulator for Great Britain (England, Wales and Scotland), known as the Medicines Healthcare products Regulatory Agency (MHRA) may rely on a decision taken by the EMA on the approval of new treatments. This is the regulatory pathway Amylyx is currently pursuing for Great Britain.
A phase 3 confirmatory trial of AMX0035, called PHOENIX, is currently underway. Topline results are expected mid-2024. You can find out more about AMX0035 and PHOENIX on our website.
New programme announced that aims to move potential MND drugs into clinical trials faster
21 June 2023
A new experimental medicine programme, called EXPErimental medicine Route To Success in ALS (EXPERTS-ALS), designed to rapidly test promising treatments has been announced. It is a flagship programme of the UK MND Research Institute (UK MND RI). EXPERTS-ALS will quickly identify the most promising drugs to go forward to be tested in larger phase 3 clinical trials to assess whether they are beneficial for people with MND.
The platform will initially test repurposed drugs (drugs that are already used for other conditions) in small groups of people with MND for up to 6 months to see if levels of a marker of neurone damage, called neurofilament light chain, decreases with treatment. It is currently being set-up and the team hope to begin testing drugs in the Summer of 2024. The EXPERTS-ALS programme will also help to develop the next generation of MND researchers and will be led by Professor Martin Turner (University of Oxford) and Professor Chris McDermott (University of Sheffield).
The MND Association intends to help to fund this programme along with the Department of Health and Social Care (through the National Institute for Health and Care Research), MND Scotland, My Name’s Doddie Foundation and LifeArc. Find out more about EXPERTS-ALS on our blog.
MND Association invests in MND-SMART clinical trial
19 June 2023
The MND Association has committed £500,000 to MND-SMART, the UK’s first platform trial. The trial is designed to run for several years and give more people with MND a chance to take part in a trial. The funding from the MND Association is in partnership with MND Scotland which has contributed a further £2 million bringing a combined investment from both charities of £2.5 million. This additional investment from both charities will help maintain the infrastructure to enable MND-SMART to test new drugs for years to come.
MND-SMART has an innovative trial design which allows more than one drug to be tested at the same time, speeding up progress and reducing the number of people assigned to the placebo (dummy drug). The trial has already recruited over 500 participants and is currently testing 3 potential drugs. You can read more about MND-SMART and how to get involved on our blog or on the MND-SMART website.
Additional results from CNM-Au8 arm of HEALEY Platform trial announced
16 June 2023
Clene Nanomedicine has announced additional results from the HEALEY Platform trial. A statistically significant reduction of a marker of nerve damage, called neurofilament light chain (NfL), was observed for those who received 24-weeks of treatment with CNM-Au8 compared to placebo. The reduction in NfL may indicate a possible reduction in nerve damage. However, comparisons to baseline levels (at the start of the trial) of NfL have not been reported. Previous results from the CNM-Au8 trials have also highlighted a potential survival benefit for those taking the drug compared to placebo. Further analysis of NfL, other biomarkers and long-term survival are underway. You can read more in a press release or find out more about CNM-Au8 on our website.
Date for FDA Advisory Committee Meeting set for NurOwn
6 June 2023
BrainStorm Cell Therapeutics have announced that the Food and Drug Administration (FDA) Advisory Committee Meeting, which will discuss the approval of NurOwn in the United States, has been scheduled for 27 September 2023. Advisory Committee Meetings involve getting independent advice from outside experts who then make a recommendation to the FDA. Although the FDA considers the recommendations of its Advisory Committees, the recommendations of the panel are non-binding. The final decision regarding the approval of the Biologics License Application (BLA) for NurOwn will be made by 8 December 2023. You can read more in a press release or find out more about NurOwn on our website.
NICE provides update on the regulatory process for Tofersen
2 June 2023
The National Institute for Health Care Excellence (NICE) has published details of its findings for the next stage for Tofersen in the regulatory process. While it has recommended Tofersen should have a technology appraisal NICE has said it does not meet all the criteria for the Highly Specialised Technology route. This is a decision which the MND Association completely disagrees with and that we find extremely frustrating. We are very concerned that this will seriously affect the accessibility of the drug for people with MND in the UK. We are working with the pharmaceutical company Biogen which is in on-going conversations with NICE. This includes submitting evidence from both neurologists and people with MND who will benefit from this drug. We will continue to keep you updated on this page. You can read more about Tofersen on our blog.
News from MND clinical platform trials running in the UK
1 June 2023
Both MND clinical platform trials running in the UK, MND SMART and MAGNET, have provided updates to the MND community. Platform trials aim to accelerate the development of new potential treatments for MND by testing multiple drugs simultaneously. MND SMART, the first UK platform trial, has added a third drug to the trial, called amantadine. They also announced that over 500 participants from 20 UK sites have been recruited. You can find out more on the MND SMART website.
MAGNET is an European and Australian platform trial and part funded by the MND Association. The trial has just opened for recruitment in the UK. MAGNET is currently only testing lithium carbonate in people with UNC13A MND, but additional drugs will be added in the future. You can read more about the MAGNET trial on our blog.
Amylyx provides an update on ongoing regulatory review of AMX0035 in Europe
30 May 2023
Amylyx have provided an update on the ongoing review of the Marketing Authorisation Application (MAA) by the European Medicines Agency (EMA) for AMX0035 for the treatment of people with MND. Following a meeting in May, Amylyx have been informed that the Committe for Medicinal Products for Human Use (CHMP) is trending towards a negative opinion on the application for conditional approval of AMX0035. A formal opinion will be given at the next CHMP meeting, held on June 19-22. Amylyx have stated that they will request a formal re-examination if a negative opinion is received. You can read more in a press release.
AMX0035 is a combination therapy, with a dual mechanism of action which is hoped to improve the survival of neurones. AMX0035 has already been approved in Canada and the United States. A phase 3 trial is currently ongoing, with topline results expected in 2024. Find out more about AMX0035 here.
Apellis announce topline results from the MERIDIAN trial of pegcetacoplan
26 May 2023
Apellis have announced the topline results for the MERIDIAN trial, which was investigating pegcetacoplan as a possible treatment for MND. The drug was designed to inhibit the activity of a protein called C3, which is involved in a specific immune response called the complement system. The MERIDIAN trial was assessing the safety and efficacy of pegcetacoplan to see whether it was beneficial for people with MND when compared to a placebo. While the treatment was found to be safe and well tolerated, the trial did not meet it’s primary or secondary endpoints which suggests that it did not have any clinical benefit for people with MND. Apellis have made the decision to discontinue the development of pegcetacoplan for MND. You can read more in a press release or find out more about pegcetacoplan on our website.
Topline results from the FOCUS-C9 study of WVE-004 announced
23 May 2023
Wave Life Sciences have announced the topline results for the FOCUS-C9 trial, which is investigating the use of WVE-004 in people living with with the most common genetic form of MND and FTD. WVE-004 has been specifically designed for people with MND/FTD who have a mutation in the C9orf72 gene. The FOCUS-C9 trial looked at different dosages and dosing schedules of WVE-004 and compared these to placebo. WVE-004 was found to be safe and well-tolerated and levels of a key biomarker, known as poly(GP) was reduced for those taking WVE-004. This lowering of Poly(GP) indicated that WVE-004 lowered levels of toxic proteins which are thought to play a role in C9orf72 MND/FTD. Unfortunately, WVE-004 was not shown to have any clinical benefit for people living with MND or FTD. No significant difference was observed by ALSFRS-R between those who took WVE-004 and those who took the placebo. Wave Life Sciences has now decided to discontinue the development of WVE-004. You can read more in a press release or find out more about WVE-004 on our website.
New MND and Sport Expert Working Group formed
16 May 2023
A new group, convened by MND Scotland, has been formed in response to scientific publications which indicate there may be an increased risk of developing MND among people who participate in elite-level sport, including football and rugby. Dr Nick Cole, our Head of Research, represents the MND Association at this new group which will work to explore the potential link between sport and MND. The group also includes experts in sport and MND from around the world, people living with MND, MND Scotland and My Name'5 Doddie Foundation. The aim of the group is to better understand the possible link, determine what research questions need to be asked to identify any underlying causes, as well as determine what resources are available, or required, to answer them. You can read more on MND Scotland's website. You can also read about the latest research into MND and rugby over on our blog.
LifeArc and PrecisionLife form new partnership to accelerate the discovery of targeted treatments
15 May 2023
LifeArc and PrecisionLife have formed a new partnership which aims to accelerate the discovery and development of targeted treatments for people living with MND. This collaboration will help to develop findings by PrecisionLife (working alongside the MND Association, PrecisionLife, King's College London and University of Sheffield) who are using artificial intelligence to identify subgroups and potential new treatments which could lead to more personalised therapies. This will work alongside new biomarkers, founded by PrecisionLife, which could be used to identify which drugs may work better for different people. LifeArc will lead and coordinate the drug discovery research part of the project, with PrecisionLife supporting them to select the most promising drug targets. You can read more in a press release.
Registration for the 34th International Symposium on ALS/MND now open
9 May 2023
Registration for the 34th International Symposium on ALS/MND has now opened. The Symposium, which is the first to go back in-person since 2019, will be held in Basel, Switzerland. Since the COVID-19 pandemic, the Symposium has been held virtually, and one of the advantages of holding it online is the ability for those unable to attend in person (particularly people living with ALS/MND) to participate in the event. The MND Association are keen to continue this aspect of inclusivity and have included a 'online' registration. This will include access to selected live-streamed sessions, the ePoster hall and post event, on-demand content. You can find out more and register on the Symposium website.
Apellis announce that the open-label phase of the MERIDIAN trial of pegcetacoplan has ended
27 April 2023
Apellis have announced that the open-label phase (where everyone in the trial now receives the treatment) has ended earlier than planned. This decision was made following analysis of unblinded data and was made by an independent data monitoring committee. The committee recommended not to continue treatment. This decision was not based on any safety concerns. Full analysis of the randomised part of the trial will now be completed, with top-line results expected in mid-2023. You can read more in a press release.
Genetic testing guidelines for MND in the UK changed
26 April 2023
The UK guidelines (UK National Genomic Test Directory criteria) have been updated to state that there is no limit on age or family history for routine genetic testing. This change in guidelines now allows more people living with MND the option of having genetic testing. With the rise of potential genetic therapies, which require a confirmed genetic mutation, these changes could allow more people living with MND access to therapies in the future. While we welcome these changes, we are aware that there are significant delays with accessing genetic counselling, testing and results. The MND Association is working to consider how we can help remove barriers to ensure more people are able to access genetic testing and appropriate counselling should they wish to.
If you have a question about genetic testing, please see our FAQs.
FDA announces the approval of Tofersen for the treatment of SOD1 MND
25 April 2023
The Food and Drug Administration (FDA) have announced that they have approved Tofersen (Qalsody) for the treatment of SOD1 MND in the United States. This approval is based off the data from the Phase 3 VALOR trial. This data included evidence that Tofersen reduced neurofilament light chain levels, which is a marker of nerve damage. SOD1 MND is where there is a mutation within the SOD1 gene and accounts for around 2% of all MND cases. The FDA approval in the U.S. is a welcome step forward in the fight against MND and further highlights the commitment and dedication of the MND community in finding effective treatments. You can find out more about the approval in a press release.
Tofersen is currently not approved in the UK. However, tofersen is being reviewed by the European Medicines Agency to see if the treatment could be approved in the UK and Europe. To find out what this approval means for people living with MND in the UK, head over to our blog.
Study provides new insights into the link between the brain immune cells and motor neurone death
21 April 2023
Researchers from the University of Edinburgh and the University of Dundee have found that an essential function within brain immune cells is faulty and can lead to motor neurone death in C9orf72 MND. Researchers used stem cell methods to create brain immune cells (in a dish), known as microglia, from people with C9orf72 MND. They found that the essential waste clearance function of these microglia was faulty. The faulty waste clearance led to a build-up of toxic chemicals within the immune cells which spread to the cellular environment around motor neurones. This led to motor neurone death.
Researchers were able to boost the faulty waste clearance in microglia, using a compound known to activate waste clearance, and prevent motor neurone death. The researchers suggest with further research this work could lead to the development of a potential new therapy for MND. You can read about more in the paper published in Science Advances.
UK MND Research Institute now operational
13 April 2023
The new UK MND Research Institute, set up after the successful United 2 end MND campaign, is now operational. The Institute, which includes over 20 Universities and Centres, aims to accelerate the search for treatments and improve access to clinical trials. Scientists who are part of the Institute have been submitting applications for the £50 million funding that has been ring-fenced by the Government for MND. You can get the latest updates from the Institute here.
Cytokinetics announce that the COURAGE-ALS trial of Reldesemtiv will be stopped due to futility
31 March 2023
Cytokinetics have announced that COURAGE-ALS, a phase 3 trial of a potential treatment called Reldesemtiv, will end earlier than planned. An interim analysis of data from the trial found no differences in ALSFRS-R score or any key secondary endpoints between those on placebo and those on the drug. There was no evidence that Reldesemtiv has a beneficial effect for people with MND based on data collected at the 24-week assessment and, due to this, the COURAGE-ALS trial and open label extension will be concluding. Further analysis of the data will be conducted to understand these results. You can read more in a press release here. You can find out more about Reldesemtiv here.
BrainStorm Cell Therapeutics have announced an FDA Advisory Committee Meeting for NurOwn
27 March 2023
BrainStorm Cell Therapeutics have announced that the Food and Drug Administration (FDA) will hold an Advisory Committee Meeting for the approval of NurOwn. The date for this is still to be announced. Advisory committees are made up of a panel of experts who discuss the application and then make a recommendation. Although the FDA considers the recommendations of its advisory committees, the recommendations of the panel are non-binding. You can read more in a press release here.
NurOwn is a potential therapy which purifies mesenchymal stem cells from bone marrow extracted from the patients hip bone. The stem cells are then matured so they produce neurotrophic factors which are though to protect and promote the health of neurones. These cells are then injected back into the patient in their spinal fluid. You can find out more about NurOwn here.
FDA Advisory Committee outcomes are announced for Tofersen
23 March 2023
Biogen have announced the outcome of the FDA Advisory Committee meeting on Tofersen as a treatment for SOD1 MND. The panel members unanimously agreed that reductions in levels of Neurofilament light chain, a biomarker of disease, are likely to predict clinical benefit for people with SOD1 MND. Overall, the panel voted no when asked whether the data from the Phase 3 Valor trial and open-label extension provide substantial evidence of the effectiveness of Tofersen for people with MND, who have changes in the SOD1 gene. However, ultimately the panel concluded that the benefits of Tofersen outweigh the risks. You can read more about the Advisory Committee vote here.
Although the FDA considers the recommendations of its advisory committees, the recommendations of the panel are non-binding. The final decision regarding the approval of the New Drug Application for Tofersen will be made by the FDA by 25 April 2023. Tofersen is also still being reviewed by the European Medicines Agency for approval in Europe. You can read more about Tofersen here.
Study suggests pathway in the immune system could be linked to MND
13 March 2023
Researchers from Harvard Medical School and Boston Children's Hospital have identified a new pathway in the body's immune system which plays a role in neurodegeneration. The immune system is vital for helping cells fight off danger, such as infections. One type of immune molecule called gasdermin E has been found at very high levels in neurons of people living with MND. The researchers also found that when neurons detect danger, gasdermin E causes damage to the powerhouse of the cell, known as mitochondria, and axons. When the researchers inactivated gasdermin E in models of MND, the mitochondria and axons were protected from damage. These findings suggest that gasdermin E could be a potential target for therapeutic intervention in the future. You can read more here.
Further results from the CNM-Au8 trial reported
7 March 2023
Clene Nanomedicine have announced additional data from the open-label extension of the RESCUE-ALS trial which is testing CNM-Au8 in people living with MND. The trial failed to reach its primary endpoint of reducing disease progression over 36-weeks. However, further results from the trial have now shown potential benefits in disease progression, as measured by ALSFRS-R, over 48-weeks. They found participants who were originally randomised to the treatment had a 2.6 point difference on the ALSFRS-R compared to those who were originally randomised to the placebo.
They also looked at data between week 60 and week 120 on the open-label extension. These weeks were chosen as it gave participants who were originally randomised to placebo 24-weeks to get to a steady state on the treatment. They found that there was a 6.0 point difference in ALSFRS-R for those who were originally randomised on the treatment compared to placebo. You can read more about these results in a press release and find out more about CNM-Au8 on our website.
Researchers say nasal spray could be developed to treat C9orf72 MND/FTD
1 March 2023
Researchers at the University of Sheffield’s Institute of Translational Neuroscience have found that using a small molecule, known as a peptide, could help prevent the death of nerve cells in C9orf72 MND/FTD. The peptide is made up of a short chain of amino acids which are the building blocks of proteins. This peptide has been designed to stop the faulty instructions (RNA) used to make toxic repeat proteins (known as DPRs) being transported outside of the control centre of the cell. Blocking the production of toxic DPRs, in animal and cell models, was found to prevent the progression of MND/FTD and restore the function to affected nerve cells. The researchers also hinted that the peptide, with further studies, could be developed into a nasal spray. You can read more in the press release or find out about the early research into this area on our blog.