In the last decade, the MND Association has invested millions in research within the UK and across the world. We are a leader in the funding and promotion of cutting-edge MND research and, with over 30 years experience of identifying the most promising projects, we only fund and support scientific and medical research of the highest quality and relevance to MND.
And the great news is, we are not the only ones!
The International Alliance of ALS/MND Associations has 54 member institutions, in 40 countries around the world – from Mongolia to Mexico, Malta to Malaysia – who are supporting, funding, collaborating in and carrying out MND research, and/or offering much needed care and support to people with MND and their families.
All the institutions listed by the Alliance are shown on the map above. If you want to take a look at some of these, they are easy to access through the International Alliance website. Some of the websites are not in English but you can use the Google Translate Web tool to translate the entire site into English (or any other language).
So let’s take a whistle-stop tour and explore some of the latest research and support projects that other institutions around the world are involved in. The institutions I mention are shown on the map with a yellow pointer.
A closer look at the work of research institutions around the world
The ALS Therapy Development Institute in Boston, Massachusetts have developed AT-1501, a new antibody which acts in a highly targeted, disease specific way to ‘damp down’ the immune system. Thirteen years in development, AT-1501 is planned to move into human clinical trials later this year.
Research would suggest that about 70% of people with MND have an immune response at some point during their disease. Targeting this immune response in people with MND is important in developing effective treatments. In pre-clinical testing in a SOD1 mouse model, AT-1501 extended life span significantly, delayed disease onset, improved body weight, maintained muscle function and decreased indications of inflammation in nerves and spinal cord time after time. You can find out more about antibodies on our blog.
MND Australia are funding a project led by Dr Mary-Louise Rogers, from Flinders University, that aims to develop an MND therapy based on delivering factors specifically to motor neurones that have been shown to improve the health of the motor neurones in a pre-clinical mouse model of MND. They will use a urinary biomarker, p75, to determine if treatment is working. The study will inform the researchers if this therapy has the potential to be developed as a valid treatment for MND.
ALS Canada is funding a project headed by Dr Turgay Akay from Dalhousie University which is investigating why some mice are able to walk almost normally despite significant loss of motor neurone function. To understand how the nervous system enables movement, Dr Akay is studying cholinergic-boutons (C-boutons). These are specialised synapses (the junction between two nerve cells) that are connected to motor neurones. Previously, Dr Akay noticed that C-boutons were responsible for changing the excitability of motor neurones (how the neurone reacts to its environment) in mice. The level of excitability depended on what the mice were doing – swimming produced a more excitable response and therefore higher muscle activation than walking. In his work with mice, Dr Akay has observed that they can still move quite well despite a significant loss of motor neurones and thinks that C-bouton activity is responsible for this. He believes that C-boutons increase in size and number to compensate for the loss of motor neurone function. He will examine mice with severe motor neurone loss to see if this effect slows or accelerates MND progression. He will also test drugs known to change C-bouton signalling to see if motor function can be preserved in mice. This may pave the way for future human studies to find new ways to improve mobility and possibly slow disease progression.
ALS Canada is also promoting two clinical trials. The first is a phase 1 trial and is testing the safety and tolerability of a new oral medicine called GDC-0134 in people with MND. This has been designed to block the activity of a protein called DLK, which is found in nerve cells in the brain and spinal cord. Previous studies have shown that activation of DLK may cause these nerve cells to die when they are stressed. Tests in animal models have shown that when DLK is blocked it may help to delay motor neurone death. Seventy people living with MND will be enrolled onto the study which will enable researchers to test whether the drug is safe, determine dosage and learn more about how the body breaks down the drug internally. Successful completion may lead to a larger Phase 2 trial.
The second is a phase 2 trial which is looking at the drug pimozide, which was originally used to treat schizophrenia. In MND loss of muscle function may occur because the muscles and nerves are not able to communicate any more. This communication happens at the neuromuscular junction and pimozide has been shown to enhance communication at this location in animal studies. This trial will investigate if treatment with pimozide slows the progression of MND in people, using the ALSFRS.
In France, ARSLA is co-funding the PULSE Protocol. PULSE stands for Prognostic Value of Biomarkers in Amyotrophic Lateral Sclerosis & Endophenotypic Study. With data collected from 1000 people with MND over the course of their disease, and matched controls, using clinical, radiological and genetic biomarkers, PULSE will establish precise criteria for prognosis, and the collaborative efforts of researchers across the country will use the data collected from participants to better characterise the mechanisms of the disease and guide new therapeutic concepts.
Closer to home
The Irish Motor Neurone Disease Association research team is headed by Professor Orla Hardiman at Trinity College, Dublin. One of the projects they are involved with is called Euro-MOTOR: European multidisciplinary ALS network identification to cure motor neurone degeneration. Through the collection of patient samples and detailed lifestyle questionnaires in Ireland, Italy and The Netherlands, it is hoped the project will help to further understand the causes of MND which, in turn, will allow the development of new therapies.
Researchers at MND Scotland are investigating the impact of specific types of apathy on people with MND and their families. Apathy is a lack of motivation that can affect people in three ways: being unmotivated to plan, organise or finish tasks; no enthusiasm to think of new ideas; being emotionally indifferent to what is going on around them.
The team have developed the Dimensional Apathy Scale (DAS) which assessed these different types of apathy independent of physical disability. Through repeated interviews over the course of a year, the team will assess how apathy impacts on quality of life and caregiver burden. Determining how motivation changes over time, together with establishing how it relates to quality of life and burden, will help guide intervention and symptom management techniques to improve the care and the lives of people with MND and their families.
Good news stories
The Asha Ek Hope Foundation for MND/ALS in India runs a riluzole programme that ensures that riluzole (known in India as Rilutor) is available, free of charge, to people with MND all over the country. This is supported by the generosity of the Sunpharma Company who provides the tablets, at no cost, to the Foundation who then send them where they are needed.
The Asha Ek Hope Foundation is the only MND support organisation in India, and there are around 65,000 people living with disease at any one time. In a country where around 30% of the population of over 1.3 BILLION people live on less than $2 a day, this programme is a great benefit, providing medication and support where it is needed most.
Asociación ELA Peru have co-ordinated an agreement with a company to provide non-invasive ventilation (NIV) equipment at a discounted rate to it’s members. This is great news as NIV can help manage symptoms and may allow independent breathing for longer.
The Taiwan MND Association organises the ‘Colourful Youth Life Camp’, which is a free two-day camp for children who have a parent with MND. Through group and creative activities, the children are given the opportunity to share their experiences, explore ways to communicate with their parents throughout their illness and establish a support network of peers.
Just the tip of the iceberg
The projects covered here represent only a ‘taster’ for each organisation. Researchers around with world (and there are many more outside the International Alliance) are investigating MND from every angle, trying to identify what causes the changes in motor neurones that ultimately lead to their death, and find therapeutic responses to this. Institutions are also working tirelessly to identify ways in which the quality of life for people who are living with the disease now might be improved, and providing care and support for everyone affected by MND.
Although the projects and trials listed in this article are not open to people from the UK to take part in, their results may have far-reaching implications for people living with the disease now and in the future, and pave the way for even more research until, one day, a cure is found. You can find details of projects the MND Association funds and clinical trials that you can take part in on our website.
Use these links to go directly to the research and support projects mentioned in this article.
ALS Therapy Development Institute, USA – AT-1501
MND Australia – p75
ALS Canada – C-boutons
ALS Canada – GDC-0134 trial
ALS Canada – pimozide trial
ARSLA, France – PULSE protocol (this is in its original language, use the Google Translate Web tool)
Irish MND Association – Euro-MOTOR
MND Scotland – Apathy in MND
Asha Ek Hope Foundation, India – riluzole (Rilutor) programme
Asociación ELA Peru – NIV assistance (this is in its original language, use the Google Translate Web tool)
Taiwan MND Association – Colourful Youth Life Camp
Perhaps you might explain then why it is that only Riluzole has been approved for use – and that was 22 years ago?
Thank you for your comment, my sincere apologies for the delay in replying. MND is an extremely complex disease, as I am sure you are aware. There are several drugs that are currently undergoing clinical trials to see if they are suitable to use as a treatment for MND. However, the complexity of the disease makes this a very difficult process. Successes in the lab do not always carry forward when the drug is tested in humans and we see this when clinical trials do not produce the results the researchers are hoping for. Unless a trial is completed successfully the drug will not meet the stringent licensing rules we have in the UK. However as a new, and better, treatment is desperately needed researchers continue to develop compounds that will hopefully, one day, translate into a successful treatment. Please be assured that this research is on-going and will not stop until better treatments are found.
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