Latest Research News
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.
BrainStorm Cell Therapeutics announce 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 thought 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.
Top-line results from HEALEY Platform trial for pridopidine announced
23 February 2023
Top-line results from the pridopidine regimen of the HEALEY Platform trial have been announced. The primary endpoint of the regime was not met. This meant that participants randomised to pridopidine did not have a statistically significant slowing of disease progression as measured by ALSFRS-R over 24 weeks compared to participants randomised to placebo. However, pridopidine was found to have a potential greater benefit for those who were within 18 months of symptom onset with a definite or probable ALS diagnosis.
The mechanism of action of the drug suggest it may have benefits for people living with MND with bulbar and speech related symptoms. The trial found improvements in speaking rate and articulation rate for those who took pridopidine compared to those who took placebo. You can read more about the results in a press release.
The HEALEY Platform trial is designed to accelerate the process of drug development and the path for effective treatment for MND by testing multiple drugs simultaneously and adaptively.
Study shows differences between female and male motor neurones
17 February 2023
Researchers have found features that are different in female and male motor neurones. Skin cells from people living with MND and people without the disease were taken and reprogrammed into motor neurones (known as iPSCs). The team analysed the motor neurones to try and detect any patterns or differences between the two groups. They expected to see differences between the groups but the changes were very subtle. However, they did find that male MND samples showed changes compared to both those without the disease and the female MND samples. The researchers conclude that they have found a way to distinguish between male and female motor neurones, which could help to further our understanding of the disease and help explain why more men are diagnosed with MND. Find out more here.
Study provides new insights into why motor neurones are vulnerable to MND
8 February 2023
A recent study characterised neurones and other cells inside the human spinal cord in order to see if they could understand why motor neurones are more vulnerable to MND. They found that motor neurones, compared to other cells, had higher levels of genes which have been linked to the development of MND. They also found that motor neurones had higher levels of genes related to the cytoskeleton and neurofilament. These are important for the shape and size of cells, especially with providing support for large cells such as motor neurones. As our motor neurones rely on these specialised structures to support them, the researchers suggested that it could make them more vulnerable to damage in MND. You can read more here.
New MND Pre-Fellowship Scheme created as part of the virtual MND Institute
1 February 2023
The MND Association and MND Scotland have announced the creation of four new MND pre-fellowships, as part of a collaborative scheme at the new virtual MND institute. These pre-fellowships are designed to attract and retain early career researchers wanting to establish a career in MND translational research and become MND research leaders of the future. Before applying for fellowships, such as the MND Association Lady Edith Wolfson Fellowship Programme, researchers require initial data to show the potential of the research. A funding gap currently exists which can make it difficult to obtain this initial data and these pre-fellowships are designed to bridge this gap.
This funding call is designed to promote collaboration across the MND Research Institute network. With this in mind, the funding call has been a collaborative effort, with funding from the MND Association and MND Scotland providing the necessary administrative support.
Applications for the pre-fellowship scheme are now being accepted and those wishing to apply can find out more information here.
New study reveals more about possible links between cholesterol and prognosis in MND
27 January 2023
A recent publication by researchers from the University Medical Center Utrecht in the Netherlands have found evidence to suggest that increased levels of High-density lipoprotein (HDL), more commonly known as ‘good’ cholesterol, are linked to poorer survival in people with MND. The population-based study also found that levels of total cholesterol and low-density lipoprotein (LDL), known as the ‘bad’ cholesterol, were lower in people who had more advanced MND. This may mean that blood cholesterol levels could help to predict disease severity and prognosis. You can find out more in a publication here.
£1 million awarded to research into cutting-edge therapies for MND
24 January 2023
LifeArc, MND Association and My Name’5 Doddie Foundation have jointly awarded £1 million to researchers at University College London to progress two promising new therapies. The projects have been awarded as part of the £1.5 million joint Motor Neuron Disease Translational Fund to support research into MND. This marks the first two research grants awarded to projects aiming to develop innovative gene and cell therapies for MND. You can read more in a press release here or find out more about the projects funded over on our blog.
FDA set date for Advisory Committee Meeting to discuss approval of Tofersen
23 January 2023
Biogen have announced that the Food and Drug Administration (FDA) have decided to hold an Advisory Committee Meeting for the approval of Tofersen. The Advisory Committee Meeting will be held virtually on 22 March 2023. Advisory committees are made up of a panel of experts who discuss the New Drug 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.
Tofersen has been found to slow the progression of the disease in around 2% of cases of MND, those living with the SOD1 mutation. You can read more about Tofersen here or read the results from the Phase 3 trial here.
Mature neurones grown in the lab for first time
12 January 2023
Researchers from Northwestern University have, for the first time, created highly mature neurones. Previous research has shown that stem cells (iPSCs) can be used to grow neurones. However, the current techniques result in limited maturation which reduce their use in neurodegeneration studies. In this new study, the researchers used a new technique called “dancing molecules” which resulted in more mature neurones. These neurones also demonstrated enhanced signalling capabilities and greater branching ability which are essential for their function. Researchers have hinted that these lab-grown neurones could one day be transplanted into people living with neurodegenerative diseases, such as MND, to replace lost or damaged neurones.
As part of this research study, the researchers also created these mature motor neurones from skin cells from a person living with MND. They found that using these motor neurones allowed them to look at biological mechanisms, such as protein clumping, known in MND, for the first time. You can find out more about here.
Type A meeting granted for NurOwn
27 December 2022
BrainStorm Cell Therapeutics have announced that the U.S. Food and Drug Administration (FDA) have granted a Type A meeting, which will be held on 11 January 2023. In November 2022, the FDA declined to consider the approval of NurOwn as a treatment of ALS. A Type A meeting will allow BrainStorm and the FDA to discuss the content of the refusal to file letter and potential next steps. You can read more here and find out more about NurOwn here.
Probiotic found to have neuroprotective properties in MND
20 December 2022
Researchers at Canada's CHUM Research Centre have found evidence which suggests a probiotic, known as HA-114, has neuroprotective properties and helps to suppress the progression of motor neurone degeneration, in animal models. The researchers hypothesised that HA-114 helps to restore balance to impaired pathways that make energy for cells (known as energy metabolism), which is crucial to keep motor neurones functional and alive. They suggested that once this pathway was restored, it led to a decrease in neurodegeneration. Further work will now start to further investigate HA-114 in a clinical setting. You can read more here.
Additional analysis announced from NP001 trials
14 December 2022
New analysis from two clinical trials of NP001 provided evidence to suggest that NP001 may slow progression of MND by stopping a process known as Microbial Translocation. Microbial translocation is a process where bacteria from the gut leaks into the bloodstream, leading to inflammation which has been known to be linked to MND.
Results from the NP001 trials found that a subset of people aged 40-65 years with elevated baseline levels of inflammation had a slower decline in disease progression, as measured by ALSFRS-R. This new analysis of the trial also looked for inflammatory biomarkers (unique biological signals) of the disease. They found that several blood biomarkers which indicated a reduction in microbial translocation and inflammation in those who were treated with NP001 compared to those who received placebo. You can read more here and find out more about NP001 here.
£29.5 million released from the Government for the United to End MND campaign
12 December 2022
The Government has announced that it will accelerate the distribution of the £50 million it pledged to invest in targeted MND research last year. Secretary of State for Health and Social Care Steve Barclay revealed a plan to ensure £29.5 million of the funding was available for MND research in the coming months. You can read a statement from the United to End MND coalition here.
More information from the Government on the £50 million can found here.
BrainStorm Cell Therapeutics have requested a Type A meeting with the FDA to discuss NurOwn
12 December 2022
BrainStorm Cell Therapeutics have announced that they have submitted a Type A meeting request to the U.S. Food and Drug Administration (FDA). In November 2022, the FDA declined to consider the approval of NurOwn as a treatment of ALS. A Type A meeting would allow BrainStorm and the FDA to discuss the content of the refusal to file letter. BrainStorm also intend to discuss a path to an FDA Advisory Committee Meeting in this meeting. Advisory Committee Meetings involve getting independent advice from outside experts. The Type A meeting is expected to occur within 30 days of the FDA's receipt of the meeting request. You can find out more here.
Top-line results from the MIROCALS clinical trial announced at 33rd International Symposium on ALS/MND
6 December 2022
Results from the MIROCALS (Modifying Immune Response and OutComes in ALS) clinical trial, a randomised placebo-controlled trial investigating the safety and efficacy of low dose interleukin 2 in people with ALS, were announced at the 33rd International Symposium on ALS/MND. You can read more in a press release or on our blog.
IL-2 was found to be safe and well tolerated, side effects were minimal across both treatment and placebo. When the whole trial population was analysed, treatment with IL-2 showed a modest decrease in risk of death for those on the treatment over the 21 months of the study, but this was found not to be statistically significant. However, when the survival analysis was adjusted - as planned in advance - by subgrouping people using a particular biomarker (a biological signal of damage to neurones) they found a larger and statistically significant effect on survival for those who received the treatment. This effect equated to an over 40% decrease in the risk of death at 21 months for a large proportion (80%) of the overall trial population who received IL-2.
Further analysis of the results is being conducted to understand how IL-2 affects the underlying biology of the disease. The lead researchers on the trial are prioritising discussions with drug regulatory bodies to determine next steps for IL-2 in the treatment of ALS.
You can find out more about IL-2 and the trial here.
European Medicines Agency accept the Marketing Authorisation Application for Tofersen
5 December 2022
Biogen have announced that the European Medicines Agency (EMA) have accepted the Marketing Authorisation Application for Tofersen. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP). You can read more in a press release.
Tofersen is an experimental drug which has been designed specifically for people living with MND with an SOD1 mutation. The results showed that Tofersen was found to slow the progression for these people over a period of 12 months. You can find out more about Tofersen here and read about the results of the study here.
New study uncovers more about SOD1 genetic variants
16 November 2022
A new study published in Nature Communications has found that some mutations associated with SOD1-MND can predict age of onset, some can predict disease progression rate, and some predict both. This means that the risk of developing MND and the clinical progression of the disease may be influenced by different processes.
Discovering more information about the genetic links to MND will help in the development of new gene therapies and increase understanding of why some treatments may benefit some more than others.
You can find out more information about Tofersen, a potential new gene therapy for SOD1-MND here.
FDA declines to consider the approval of NurOwn
14 November 2022
BrainStorm Cell Therapeutics have announced that they have received a refusal to file letter from the Food and Drug Administration (FDA) in the United States for the New Biologics License Application (BLA) for NurOwn as a treatment of ALS. This means that the FDA have decided to not consider the approval of NurOwn based off the information BrainStorm have supplied them through the BLA application. The FDA have indicated that BrainStorm can request a meeting (known as a Type A meeting) to discuss the content of the refusal to file letter. You can read more in a press release.
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.
Oral Edaravone has been approved by Health Canada
9 November 2022
Mitsubishi Tanabe Pharma Canada have announced that Health Canada has approved the oral formulation of RADICAVA (edaravone) in Canada for the treatment of ALS. You can read more in a press release.
The oral formulation of RADICAVA has also been approved in the United States, with results showing oral edaravone has a similar efficacy to the IV formulation of the drug. The IV formulation of the drug is already approved in Japan, South Korea, United States, Canada, Switzerland and China. You can read more about oral edaravone here.
A trial in the UK investigating a different formulation of oral edaravone (FNP-122) is currently recruiting. You can find out more information and where it is recruiting here.
New research shows evidence of MND-associated proteins in the gut
17 October 2022
A new study published in The Journal of Pathology: Clinical Research has found that the same protein, pTDP-43, that are linked to motor neurone disease can be found in the gut years before any MND symptoms occur. The study found pTDP-43 to be present in the gut, skin and lymph nodes often years before the patients presented with symptoms of MND. The researchers hope that this could help in the early detection of MND. You can read more about the research in a press release here.
You can also read more about this research paper over on our blog.
FDA extends review date for Tofersen
17 October 2022
Biogen have announced that the Food and Drug Administration (FDA) have extended the review period for the New Drug Application for Tofersen. The approval decision was originally expected to be by 25 January 2022, but this has now been extended to 25 April 2023. You can read more about Tofersen here or read more about the approval decision extension in a press release.
New study investigates link between rugby and MND
4 October 2022
A new study published in the Journal of Neurology, Neurosurgery and Psychiatry has found that playing rugby at international level could increase the risk of developing MND. The study looked at the health data from Scotland International rugby players by examining the types of health conditions they have experienced and their causes of death. It found that, while they lived slightly longer than the average population, they appeared to have an increased risk of developing neurodegenerative diseases, such as dementia, Parkinson’s disease and MND.
While the study is interesting and raises important questions, the amount of data included in the study was very small and results should be viewed with caution. To find out more and for some FAQs head over to our blog.
Top-line results from HEALEY Platform trial for CNM-Au8 announced
3 October 2022
Top-line results from the CNM-Au8 regiment of the HEALEY Platform trial has been announced. The primary endpoint of the regime was not met. This meant that participants randomised to CNM-Au8 did not have a statistically significant slowing of disease progression as measured by ALSFRS-R over 24 weeks compared to participants randomised to placebo. However, CNM-Au8 was found to show a potential survival signal, with those randomised to 30 mg of CNM-Au8 demonstrating a >90% reduction in risk of death. The potential survival signal is consistent with results from the Phase 2 RESCUE-ALS study which also demonstrated a reduction in the risk of death. You can read more in a press release or find out more about CNM-Au8 here.
The HEALEY Platform trial is designed to accelerate the process of drug development and the path for effective treatment for MND by testing multiple drugs simultaneously and adaptively.
FDA announces the approval of AMX0035
29 September 2022
The Food and Drug Administration (FDA) have announced that they have approved AMX0035 for the treatment of MND in the United States, as RELYVRIO™. This approval is based off the data from the Phase 2 CENTAUR trial. Two countries have now approved AMX0035, with Canada conditionally approving the drug in June 2022. The FDA approval in the US is a welcome step forward in the fight against MND and further highlights the commitment and dedication of the research community in finding effective treatments. You can find out more about the approval in a press release. You can also find out more about what this approval means to people living with MND in the UK on our blog and also read some FAQs.
The Phase 3 clinical trial, called PHOENIX, is still recruiting in the UK. To find out where it is recruiting head to our AMX0035 webpage.
Top-line results from the HEALEY Platform trial for Verdiperstat announced
29 September 2022
The HEALEY center announced the first top-line results from the HEALEY Platform trial for the Verdiperstat arm. The primary endpoint of the regime was not met. This meant that participants randomised to Verdiperstat did not have a statistically significant slowing of disease progression as measured by ALSFRS-R over 24-weeks compared to participants randomised to placebo. There were also no statistically significant benefits for other measures including muscle strength, respiratory function and survival. Full study results are expected to be released later in the year. You can read more in a press release.
The HEALEY Platform trial is designed to accelerate the process of drug development and the path for effective treatment for MND by testing multiple drugs simultaneously and adaptively.
New study suggest changes are needed in genetic testing guidelines for MND
27 September 2022
Current guidelines in the UK state that only those diagnosed with MND who have a family history or have a young onset (under 40 years old) should be offered genetic testing. New research, published in Brain, found that under these current guidelines many people who have a genetic link to MND are not being offered genetic testing and their genetic link remains undiscovered. This means that these people would also not have access to gene therapies that are starting to emerge for MND. The researchers say that genetic testing and counselling should be offered to everyone who is diagnosed (or may be) with MND, regardless of age and family history. You can read more about this research over on our blog.
CNM-Au8 continues to show potential survival benefit in RESCUE-ALS open-label extension
25 September 2022
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. Whilst the trial failed to reach its primary endpoint of reducing disease progression, the trial has continued to show a potential survival benefit over 144 weeks. This additional data is consistent with the findings which were announced in June and suggest that participants who received CNM-Au8 at the start of the trial had a 70% lower risk of death compared to those who received CNM-Au8 36-weeks later in the open-label extension. You can read more about these results in a press release from Clene or in a poster Clene presented at the 2022 AANEM Scientific Conference.
Published results from extended clinical trial of Tofersen shows promise in slowing MND progression
21 September 2022
Results from the Phase 3 clinical of Tofersen have been published in the New England Journal of Medicine. Tofersen is an experimental drug which has been designed specifically for people living with MND with an SOD1 mutation. The results showed that Tofersen was found to slow the progression for these people over a period of 12 months. Additionally, the drug was found to hit its therapeutic target by reducing the levels of toxic SOD1 protein. Researchers also saw a lowering in a protein called neurofilament light chain, which is a potential marker of nerve damage and could be an indicator of decline in disease progression. You can read more about the results of the trial over on our research blog.
Got a question about Tofersen? Read some FAQs or email the research team at research@mndassociation.org
Phase 2 clinical trial results of RT001 announced
15 September 2022
The results have the Phase 2 clinical trial for RT001 has been announced by BioJiva. RT001 is a investigational fatty acid and is expected to help make cells less vulnerable to damage and protect from degeneration. The 6-month placebo-controlled trial enrolled 43 people living with MND and was followed by a 6-month open label phase where everyone received the drug. The results showed a small slowing in disease progression as measured by ALSFRS-R. The results also suggested participants with more severe disease (defined as those who had an ALSFRS-R score of less than 41) had a larger slowing in disease progression. You can find out more about the results in a press release from BioJiva.
Whilst these results are promising it is important to note that this was a small trial with a small number of participants and the results will be need to be supported by a larger clinical trial.
Amylyx releases additional preclinical data for AMX0035
13 September 2022
Amylyx Pharmaceuticals announced a new publication showing preclinical data comparing AMX0035, which is the combination of Sodium Phenylbutyrate and TUDCA, to the individual components. In this preclinical data, they tested the combination of AMX0035 versus the individual components in ALS-derived cells. They found that AMX0035 changed the activity of many more genes involved in ALS-relevant pathways compared to either of the individual components alone.
AMX0035 has been tested in a Phase 2 clinical trial and is currently under review by the FDA in the US and a decision is expected by 29 September 2022. A Phase 3 clinical trial is also underway and is recruiting in the UK. Top-line results are expected in 2024.
TUDCA is currently being evaluated in a Phase 3 clinical trial. The trial is expected to read out top-line results in late 2023.
FDA Advisory Committee Vote for the approval of AMX0035 in the US
8 September 2022
Amylyx Pharmaceuticals have announced the outcome of the second FDA Advisory Committee meeting on AMX0035 as a treatment for MND. Overall, the panel voted for the approval of AMX0035 when asked whether the data from the Phase 2 Centaur trial and the additional information that has been published by Amylyx since March 2022 establishes that the drug was an effective treatment for people with MND. This is the second time that the panel has come together to vote about the approval of AMX0035, with the panel voting against the approval in the first meeting in March 2022. Since this first meeting Amylyx has released additional data from the Centaur trial and open label extension. 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 AMX0035 will be made by the FDA by 29 September 2022. You can read more about AMX0035 here.
Coya Therapeutics announces publication of phase 2a clinical trial results for COYA 101
01 September 2022
Coya Therapeutics have announced the results of a phase 2a trial of COYA 101 which show that the treatment is safe and well tolerated in people with ALS. This trial and open label extension also suggested that most participants given COYA 101 had a reduced decline in ALSFRS-R score and an increase in regulatory T-cell activity, which helps to reduce excessive inflammation. You can read more about this here.
Whilst these are promising results, the phase 2a trial only enrolled a very small number of participants and a larger trial is needed to fully assess the efficacy and long-term safety of COYA 101.
AB Science announces it has filed an application for conditional Marketing Authorisation to the EMA for Masitinib
25 August 2022
AB Science have filed an application to the European Medicines Agency (EMA) for a conditional Marketing Authorisation for Masitinib in the treatment of ALS. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP) with a decision to be made whether to approve or not is aimed to be in 210 days. The application is based on the Phase 2/3 data from the AB10015 study. A conditional Marketing Authorisation means if the drug is approved, additional data will be needed from the ongoing Phase 3 trial to maintain the approval of the drug. You can read more here and find out more about Masitinib here.
New publication finds a new key deficit in MND
22 August 2022
MND Association funded Lady Edith Wolfson Fellow Dr Andrew Tosolini recently published a new paper which found selective deficits in a key transport system known as axonal transport. The paper highlighted that this transport system behaves differently in motor neurones in MND compared to healthy motor neurones. This work adds another crucial piece to the puzzle of what goes wrong in neurones in MND and will help in the development of therapies to slow MND in the future. You can read more here.
We sat down with Dr Tosolini to discuss this new research in more detail and you can read our chat and find our more about this research in a blog post here.
Real-world data analysis of survival with RADICAVA (edaravone) announced
15 August 2022
Mitsubishi Tanabe Pharma America have announced the publication of a paper which discusses the analysis of people living with MND taking Radicava (edaravone) in a real-world setting rather than that of controlled clinical trial. The data suggests that continued treatment with Radicava in people living with MND was associated with prolonged survival of an average of 6 months compared to those not treated with the drug. You can read more here.
Whilst real-world data is useful to continue to support approval of a new drug and bridge the gap between clinical trials and everyday practice, it is important to note that the data may be subject to bias since it is no longer under the randomised control of a clinical trial.
BrainStorm have announced they will submit a BLA for NurOwn
15 August 2022
BrainStorm have announced today that they will submit a Biologic License Application (BLA) seeking approval from the U.S. Food and Drug Agency (FDA) for NurOwn. You can read more here.
BrainStorm also announced a correction has been made to the Muscle and Nerve publication from December 2021. Analysis in the original publication used an incorrect model. This correction had an impact on one of the secondary endpoints, average change from baseline in ALSFRS-R, in the pre-specified subgroup of participants with a baseline score of at least 35. The data showed a statistically significant treatment difference of more than 2 points of those on NurOwn compared to those on placebo for that subgroup. You can read the correction here.
Encouraging laboratory results lead to feasibility trial to test Terazosin in MND
11 August 2022
Encouraging results from the laboratory have led to a small feasibility trial to test Terazosin in MND. The study, which has already opened for recruitment, will recruit 50 participants from the Oxford MND Care Centre. Terazosin is already approved to treat high blood pressure and benign prostatic hyperplasia, making this another example of how existing drugs could be repurposed for MND. Early research in the laboratory has shown that Terazosin protected motor neurones in models of MND by increasing energy levels. You can read more here and find out more about the early laboratory research in a publication here.
UK sees more MND clinical trials open for recruitment
05 August 2022
Over the past couple of months an additional four clinical trials have opened for recruitment in the UK. There is now a range of MND clinical trials that are open for recruitment including trials for people living with MND who have a specific gene mutation (such as FUS, C9orf72 and SOD1), as well as trials for those who don't or do not know. You can find out more about which trials are now open for recruitment and which sites are recruiting here.
MND Association holds inaugural MND EnCouRage UK event
28 July 2022
The MND Association recently held the inaugural MND EnCouRage UK event aimed at recognising, retaining and encouraging the important work of early career researchers and providing them with an opportunity to spend time with people living with MND. You can find out all the highlights in a blog post here.
FDA grants priority review for Tofersen for SOD1 MND
26 July 2022
Biogen have announced the Food and Drug Administration (FDA) have accepted the New Drug Application for Tofersen. They have also granted a priority review with a decision expected by January 25 2023. Tofersen has been designed for people living with MND with an SOD1 mutation which is known to affect about 2% of people living with MND. You can find out more here or read more about Tofersen here.
CNM-Au8 receives positive EMA opinion on Orphan Drug Status
22 July 2022
Clene have announced that CNM-Au8 has received a positive opinion from the European Marketing Agency (EMA) for their Orphan Drug Status submission. You can read more here or find out more about Orphan Drug Status here.
You can also find out more about CNM-Au8 here.
Viral protein HERV-K ENV is implicated in MND
21 July 2022
Ancient viruses (retroviruses), such as HERV-K, have left genetic material in our DNA and it is thought that the reactivation of this genetic material could cause MND. HERV-K contains the blueprint (genetic code) to make the HERV-K ENV protein which may be activated in people with MND.
Researchers have now found, in cell models, that treating neurones with HERV-K ENV led to a reduction in overall neurone count and the surviving neurones were damaged. The researchers also found that treatment with GN-K01, which is designed to bind to the protein, helped to prevent the toxic effects in cell models. You can read more here.
Further data reported from the RESCUE-ALS Open Label Extension Trial
14 July 2022
Clene Nanomedicine has announced additional survival data from the open label extension trial of CNM-Au8, known as RESCUE-ALS. The additional results suggested participants who were originally randomised to CNM-Au8 lived longer than those who received CNM-Au8 9 months later. Analysis from the trial has suggested a 70% decreased risk of death for participants originally randomised to CNM-Au8, but further studies are needed to investigate this further. You can read more here or find out more about CNM-Au8 here.
CNM-Au8 is also being investigated in the HEALEY Platform Trial, which investigates the treatment for a larger cohort of participants. Results from this trial are expected in the next couple of months.
FDA plans to reconvene Advisory Committee for approval of AMX0035
5 July 2022
Amylyx announced the Food and Drug Agency (FDA) have decided to reconvene the Advisory Committee to discuss the approval of AMX0035. The Advisory Committee originally met in March 2022 but voted against the approval. This new meeting, which is planned for 7 September 2022, will focus on the additional results recently published by Amylyx. You can read more here and find out more about AMX0035 and the additional results here.
MND Association funded researcher Tatyana Shelkovnikova receives prestigious fellowship from UKRI
22 June 2022
MND Association fellow Tatyana Shelkovnikova has received a prestigious fellowship from UK Research and Innovation (UKRI). The fellowship is known as the Future Leaders Fellowship scheme and rewards outstanding researchers and innovators. You can read more here.
MND Association helps fund new £4.24 million grant to kick-start UK-wide collaborative research effort
21 June 2022
A group of charities and government research organisations have awarded £4.25 million to MND experts at six UK universities to kick start efforts to end MND. This new ‘MND Collaborative Partnership’ brings together people living with MND, charities MND Association, LifeArc, MND Scotland and My Name’5 Doddie Foundation, government bodies Medical Research Council (MRC) and National Institute for Health and Care Research (NIHR), and researchers from King’s College London, University of Sheffield, University of Liverpool, University College London, University of Oxford and University of Edinburgh. The partnership team will work together to find solutions to address problems currently hindering MND research and aims to discover meaningful MND treatments within years rather than decades. You can read more about this here or on blog post here.
MND Association Research Team give their highlights from the 2022 ENCALS meeting
17 June 2022
Members of the research recently attended the 20th European Network for the Cure of Amyotrophic Lateral Sclerosis (ENCALS) meeting in Edinburgh, Scotland, on the 1-3rd of June 2022. Here you can listen to them chat with Head of Research Dr Nick Cole about the meeting, being back in-person and their research highlights.
Health Canada approves ALBRIOZA (AMX0035) for the treatment of ALS
13 June 2022
Health Canada has approved ALBRIOZA (AMX0035), with conditions, for the treatment of ALS. This approval is subject to conditions, including being dependent on the safety and efficacy data from the ongoing Phase 3 PHOENIX trial. Topline results from the PHOENIX trial are expected in 2024. You can read more about the approval here.
The Food and Drug Administration (FDA) in the US and the European Medicines Agency (EMA) in the EU are both still reviewing AMX0035 for approval. The FDA approval decision is expected by 29 September 2022 and the EMA approval decision is expected in early 2023. You can find out more about AMX0035 here.
The Phase 3 trial, called PHOENIX, is currently underway in the US and some of Europe. The trial is currently 'in preparation' in the UK and we will update the community with more information when we have it.
Registration for the 33rd International Symposium on ALS/MND opens
6 June 2022
Registration for the 33rd International Symposium has opened. The event will be held online from Tuesday 6 December 2022 to Friday 9 December 2022. Registration costs £60. You can find out more and register here.
Are you a researcher and want to present your work? Abstract submission is also now open, so make sure you submit your abstract before 12 July. You can find out more information here.
The FDA has extended the approval decision date for AMX0035 to the end of September
3 June 2022
The FDA has extended the approval decision date, otherwise known as the Prescription Drug User Fee Act (PDUFA) goal date from 29 June to September 29 2022. The FDA has extended the date to allow more time to review additional analyses of data. You can read more here.
Biogen announces 12 month data from VALOR Phase 3 trial
3 June 2022
Biogen have announced 12 month data from the open label extension of the Phase 3 trial of Tofersen (VALOR) at the 2022 ENCALS meeting. The additional results from the open label extension suggested participants who were originally randomised to Tofersen had a reduced decline in ALSFRS-R compared to those who received Tofersen 6 months later. You can read more here or access the presentation from ENCALS here. You can also find out more about Tofersen here.
Eledon announces topline results from Phase 2a trial of Tegoprubart
31 May 2022
Topline results from the Phase 2a trial of Tegoprubart have been announced, showing the drug to be well-tolerated. The trial also found that the drug demonstrated dose dependent target engagement and inflammatory biomarkers associated with ALS were observed and reduced. You can read more here and find out more about Tegoprubart here.
Find out about this weeks MND/ALS research publications
25 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on new potential treatments such as WVE-004, clinical trial designs, biomarkers, TDP-43, SOD1 and much more. You can read the full list here.
Gene therapy reduces toxic RNA in preclinical studies
23 May 2022
A gene therapy known as CRISPR, which can be used to alter the genetic sequence within a cell, has been shown to reduce the buildup of toxic RNA (RNA is the photocopy of DNA which is used to make proteins) molecules found in C9orf72 MND. The researchers suggested that the therapy only affected the toxic C9orf72 RNA, meaning that the C9orf72 RNA needed for the function of motor neurones could still be produced. You can read more here. You can also find out more about CRISPR on our research blog here.
Find out about this weeks MND/ALS research publications
20 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on gene therapies, biomarkers, TDP-43, SOD1 and much more. You can read the full list here.
Further results from the AMX0035 CENTAUR trial reported
16 May 2022
Further results from the AMX0035 CENTAUR trial have been reported, including data up to 35 months after initial randomisation. The results suggested that the risk of events such as, trachestomy, permanent ventilation and hospitalisation was reduced by 47% for those on the trial who were originally randomised to the AMX0035 group. You can read more here.
FDA approves oral form of edaravone for treatment of ALS
13 May 2022
The Food and Drug Administration (FDA) has approved an oral form of edaravone known as Radicava ORS in the US. Radicava ORS is an orally administered version of Radicava, which was approved by the FDA in 2017 as an intravenous infusion. Radicava ORS can be self-administered at home. You can read more about the approval here.
Radicava (edaravone) is currently not approved for use in the UK. However, a new trial, called ADORE, which is investigating an oral form of Edaravone (FAB122) recently starting recruiting in Europe. Trial sites in the UK are currently 'in preparation'. You can find out more here.
Find out about this weeks MND/ALS research publications
13 May 2022
Each week we will be adding a list of the latest research papers published relating to MND/ALS. This week includes papers on respiratory onset MND, glucose metabolism in MND, TDP-43, SOD1 and much more. You can read the full list here.
Further survival data reported from AMX0035 Centaur trial
6 May 2022
New statistical analysis of the data from the Centaur trial has suggested a survival benefit of 10.6 months for those who were originally randomised to AMX0035 and continued treatment in the open label extension compared to those who never received AMX0035. This analysis, know an “rank-preserving structural failure time model”, estimates what the survival difference would have been if those who had initially been given placebo had stayed on placebo throughout the study rather than switching to AMX0035 in the open-label phase. You can read more here and find out more about AMX0035 here.
WVE-004 demonstrates proof-of-concept in preclinical studies
5 May 2022
WVE-004, an experimental therapy for people living with ALS who have mutations in the C9orf72 gene has shown proof-of-concept in preclinical studies. Researchers found that WVE-004 reaches the tissues affected in C9orf72 ALS in mouse models. They also found that the therapy reduced levels of biomarkers, such as poly-GP. You can read more here. You can read more about the FOCUS-C9 trial of WVE-004 in humans here.
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