FAQs and Latest Update
An update on the #United2EndMND MND Research funding, new Institute & FAQs
***** Please note that this page is being updated as you read! – check back soon for the latest FAQs and answers – 07/04/2023 *****
There has been a lot going on and around the formation of the new National Institute for MND Research.
We have produced a series of questions that you might just have, which you can jump to right now by clicking here – Take me to the FAQs.
In our June update, we outlined how the scientific partnership had reached an understanding on the way the UK government funding of £50m will be released through a limited number of funding applications. We also received in September 2022, a very welcome and encouraging governmental statement by the then outgoing Health Secretary, Steve Barclay.
Since his reappointment in October 2022, progress has moved ahead with some pace, culminating in the exciting announcement made on 12/12/2022.
At the same time, our scientists have been busy with the work funded by the award of £4.25m from the UK government, MND Association, My Name’5 Doddie Foundation, MND Scotland and LifeArc made earlier this year. Although this award is separate to the £50m, the research community and government are using it to lay the vital and key foundations for the Institute that will now be built with the £50m, including the £30m just announced.
The new Institute will see six of our leading Universities – Liverpool, Sheffield, King’s College London, Edinburgh, UCL and Oxford and
22 other research centres working together closely to accelerate the development of treatments. These world-leading scientists are putting aside normal academic competition to work together for the benefit of patients. It is unprecedented and we are grateful for their dedication.
Some of the exciting actions already underway
- Work that aims to develop a minimum of three new drug candidates for UK trials within the first 3 years.
- Searching for new biomarkers to make drug trials more reliable, robust, potentially shorter and with smaller placebo groups.
- Developing a new disease progression assessment tool, building on the ALSFRS-Rfunction scale, so that scientists and patients can be more confident that a drug works.
- Establishing common stem cell models for all laboratories to use so that they can work seamlessly together, speeding up discoveries.
This is all complementary to other projects and trials, such as Biogen’s Tofersen, to treat SOD1 genetic MND – which has now been submitted for drug approval to the USA FDA, the soon to be reported MIROCALS (Interleukin 2) trial and numerous other studies in the UK today.
The details of UK Clinical trials can be found here
You might also find the Clinical Studies Group website
useful as well.
These offer continued hope and further advancement towards treatments. It really feels like we are turning a corner and that cusp we speak about is about to be breached.
Frequently Asked Questions
1. When will the researchers receive the first funds from the £50 million government commitment? Will they receive any of the funds in 2022? When will the researchers begin their work?
Researchers are already undertaking the work outlined in their scientific plan, building the foundations of the institute. This is being enabled through the LifeArc administered £4.25m funding, mentioned above. They are in the process of applying for the £50m funding now, but it is unlikely, given the necessary processes (including peer review) that any will be released in 2022. However, given the considerable work already underway, we do not think this will have a significant impact on the timing of the work. At present, scientists are putting the foundation blocks in place for the institute and their collaborative work – this is essential to accelerating progress. LATEST UPDATE – on 12/12/2022 the U.K. Government announced the immediate availability of £30m for the Institute! This announcement will significantly reduce the burden on our researchers.
2. What is the schedule for receiving the £50 million commitment over the five years?
It is now envisaged that 2 or 3 significant grants will release the funds per year over the first 5 years. We also believe that the £50m will be only the start of government commitment and that it will be accompanied by significant charity and industry investment. The scientists currently intend to submit applications in the region of £5m to £6m a year, followed by a more significant renewal of the institute foundational funding within 3 years. This is, of course, subject to change as the project progresses.
3. Is additional investment likely to be forthcoming and who will it come from?
There are indications that £50 million will be the minimum government commitment. We also believe there will be continuing contributions from the major charities as well as considerable investment from the international pharmaceutical and medical research industries. The recently allocated £4.25m grant from the newly formed partnership with LifeArc (translational science specialists), MND Association, My Name’5 Doddie Foundation and MND Scotland included a contribution from UK government of £1m. These projects are foundational for the National Institute for MND Research and have already started.
4. When will the first drugs be trialed? Who will be eligible?
After the preparatory work, we expect new trials will start to appear within 18 months. A key, challenging and game-changing aim of the project is to move towards offering every patient, on diagnosis, a place on a trial. Currently, finding a trial is challenging and is a cause of deep frustration within the patient and carer community. Our neurologists are similarly frustrated and wish to speed the process up by attracting international pharmaceutical companies to run more clinical trials in the UK on the world leading platforms they are developing, including TRICALS, MND-SMART. TRICALS and MND-SMART are already hosting several trials but have been hampered by the pandemic. It is hoped that access to such trials will be eased as things improve. Historically, trials have only been available for patients within 2 years of diagnosis, often with other exclusion criteria. As the project progresses, the scientists’ aim is to offer every patient, including those already diagnosed, a place on a trial through a combination of more trials, personalised trials and innovative trial protocol improvements.
5. There are a lot of trials listed on the MNDA clinical trials page. Which of those trials are most likely to be successful? Will the trial drugs listed go on to the trial platforms that were spoken about during the campaign?
People living with MND and their families should discuss the most appropriate trials and how they might participate with their neurologist. If you need more information you should contact one of the major charities for advice. You can find some information on current trials here https://www.mndassociation.org/research/clinical-trials/treatment-trials/ You might also find the Clinical Studies Group website https://www.mndcsg.org.uk/clinical-research/what-and-where useful as well. It is difficult to say which trials are likely to be successful for which people. All trials are by definition, experimental. All the therapies mentioned on this page have shown some hints of efficacy at an early stage of research, but they need to complete the trial process. Robust protocols and regulations are in place to ensure confidence in the outcome. Often, seemingly promising candidates can flounder at a later stage.
6. How will scientists decide upon the most likely therapies for trial?
A drug discovery and development programme will be established which will aim to deliver a continuous stream of new targeted medicines for testing in clinical trials. Key to selecting the most likely drug candidates will be robust pre-clinical testing (prior to human), matched to the growing number of disease targets. Truly building on both the most recent discoveries and the enormous scientific legacy of the last 30 years of MND research it is hoped that the most promising candidates will progress more rapidly to pivotal in-human studies.
7. You have said that the government investment is for targeted translational research. How will research for care be funded going forward?
Care funding is of course vital for the MND community. MND is an almost uniquely challenging disease when it comes to the demands of healthcare. Both care research and care will continue to be funded by government through unique applications to the NHS and charities.
8. Who are LifeArc? What is their role in this?
LifeArc is a self-funding charity. Its specialism is in selecting promising drugs and accelerating their progress from discovery and basic science through to patient trials. This is known as translational science, taking research from the laboratory to use in the clinic. LifeArc has achieved a lot in this area and has significant cash reserves from its proven track record in cancer. With this new and exciting partner, the #United2EndMND coalition hopes to bring similar success to MND.
9. Why is this partnership being formed now?
The rapid increase in MND research activity globally and the rate at which “targets” (the genes or processes in the human body which appear to be involved in causing MND) are being found, necessitates this partnership. Traditionally, trials are set up and then ripped down, making for a hugely inefficient and costly process. With a single efficient infrastructure and network dedicated to translational research, we can learn from mistakes made and obstacles found in the process and develop a much-needed faster trial process.
10. Previously you have mentioned an MND Research Institute – is this still a possibility?
Yes, it is. The goal of our scientists is to establish such an entity to provide even more focus on MND. The £4.25 million investment announced in June and the associated work are the foundation stones of the Institute.
11. Are UK Scientists collaborating globally on this project and/or other MND Research endeavours?
Compared to even 5 years ago the world of MND Research, and in fact all medical research, is all about global collaboration. Although there are different regulatory systems throughout the world, patient advocacy is driving change. Trials are collaborated on globally with many being operated in several regions of the globe. There are also unique trials within regions, including here in the UK. At the centre of MND Research are our leading MND/ALS global Associations and charities.
12. What can I, or families affected by MND, do to help the ongoing campaign?
We, as just 5 people living with MND, are all aware of the deep emotional desire to do anything that can help. We welcome all suggestions, but two things jump out to us:
1) We would like the wider medical professional community to know about the endeavour. Your local neurologist/consultant/GP will not necessarily be in the picture. Please tell her/him about United2EndMND and how you would like to make sure they are up-to-date, so they can direct patients to appropriate trials when they come on stream. Please point them towards the MND Association, My Name’5 Doddie Foundation and MND Scotland. But also direct them to the MND Clinical Trials websites of the MND Association and the Clinical Studies Group https://www.mndcsg.org.uk/clinical-research/what-and-where
2) Please keep your MP updated with progress on the project and its funding, especially the £50 million promised government investment. We have every confidence that it will come in a timely manner, but we must monitor the situation and make sure it is in line with our scientists’ needs. You can keep up-to-date on progress and any necessary action here on our website.
13. I am interested in taking part in a clinical trial, but I am confused on choosing and how to enrol
Yes it can be confusing. A very good reference is the MND Association webpage https://www.mndassociation.org/research/clinical-trials/treatment-trials/. Details, including contact names are included. We suggest, in the first instance, talking with your neurological consultant and/or GP. Why not send them the webpage link?
If you are interested in a specific clinical trial simply email the leader contact on the trial details page.
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