Guest blog

Blog – UK Motor Neuron Disease Research Institute

Blog from Dr Jane Haley

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In the future, I’d like to think that 2023 will been seen as a pivot point at which the hunt for a disease-modifying treatment for motor neuron disease (MND) took shape and took off in the UK. As the year in which a vision to realise significant, effective and life changing treatments for MND by the end of a decade was enabled. The year that the UK MND Research Institute (UK MND RI) was born.

MND is an almost uniquely unpleasant terminal condition – rapidly robbing people of their ability to walk talk, eat and, ultimately, breathe. One in 300 people in the UK will die of MND and average survival is just 18 months after diagnosis. It also inflicts a high burden on those who love the person affected, as they care for someone whose physical abilities are challenging and rapidly changing. There is currently only one licensed treatment in the UK, Riluzole, and this only extends life by 2-3 months. Transformation of treatment is essential, and quickly because people with MND do not have time.

There are many commonalities between neurodegenerative conditions which makes MND relevant to dementia researchers and even a possible pathfinder condition. Indeed, some people with MND also experience frontotemporal dementia (FTD) and this association is particularly found in people with a mutation of the C9orf72 gene (1).

We know that MND is a 6-step process, in which a combination of genetics and other factors combine to trigger neurodegeneration (2). For some people MND is inherited and large effect gene variants can be identified in up to 20% of patients. These gene variants lower the age of onset, could worsen prognosis, and might be associated with other diseases such as frontotemporal dementia, Parkinson’s disease or schizophrenia.  Environmental risk factors are more difficult to demonstrate with certainty, but high athletic ability and previous deployment to a war zone have been associated and with MND (3).

Other commonalities between many neurodegenerative conditions are practical. This includes the need for decent diagnostics so that earlier diagnosis can be achieved – currently it can take months to get a diagnosis of MND because, in addition to their being no definitive test, the ramifications for the person affected are so profound that all other conditions need to be ruled out. There is also a need for clear biomarkers that can be used to track disease progression – to broaden and better quantify measured outcomes in clinical trials, but also to gather mechanistic information from people that can then be fed back into fundamental research.

This week is Global MND Awareness Week so I thought dementia researchers might be interested in how and why the UK MND RI has come into existence, and what it plans to deliver.

The UK has a richness of talented, high-quality researchers, including those who are clinically qualified and usually have more direct and insightful experience of the conditions that are their focus. And this is as true for MND as for other research areas, such as dementia. Translation from fundamental research to treatment in neurodegenerative conditions is, however, notoriously difficult so, if there is to be a transformation in treatment, things needed to be done differently.

Working in partnership can be a very powerful tool for progress and to do things differently we need an integrated research community. We also need to address the translation end of the treatment pipeline so that when promising new targets are identified (which is now starting to happen for MND thanks to improved mechanistic understanding) they have a smooth journey, generating high-quality data, through the pipeline and into clinical trials. This approach can only be achieved with people working together, willing to combine ideas and resources in order to create a sum that is greater than its parts.  This is the concept behind the new UK MND Research Institute (UK MND RI).

The gestation of the UK MND RI has been rapid – during 2020 a vision was co-developed by patient campaigners and researchers to unlock the MND research potential that exists within the UK and accelerate the pathway to new treatments. They wanted to specifically target that hard-to-bridge gap between fundamental research and clinical trials – a gap that is hard to fund and has, rather tactlessly, been dubbed ‘the valley of death’ (4). They calculated that £50 million of ‘pump priming’ funding by the UK Government would help create a UK wide virtual research institute

Supported by the MND Association, My Name’5 Doddie Foundation and MND Scotland, the United to End MND campaign was created and is driven by patient campaigners ( This coalition worked relentlessly throughout 2021 to raise public awareness of MND, and campaign for £50 million from the UK Government to realise their collective vision.

In the meantime, rather than wait, the charities and LifeArc (a large biomedical research funding charity) were joined by the MRC and NIHR and they decided collectively to commit £4.25 million of their own funds into getting things started and creating a firm governance platform for the hoped-for £50 million from the government to land – which it did in November 2021 (and, although a year of uncertainty followed, the funds are now starting to be released).

In January 2023 the first phase of the UK MND RI started work. It is led by Co-Directors Professor Ammar Al-Chalabi (King’s College London) and Professor Chris MacDermott (University of Sheffield) and this initial phase consists of 6 core academic centres around the UK but, as it develops, more researchers are joining and the intention is for it to be an inclusive and welcoming environment for MND researchers.

The timing of this initiative is important as, for the first time, there is now sufficient knowledge of MND disease causes and mechanisms to design rational and coordinated approaches to therapy, and enough knowledge of how to measure patient outcomes and how to carry out trials in a patient friendly efficient design.

The initial phase of the UK MND RI will focus on establishing the coordination of the research effort, which is pivotal to delivering maximum impact for people with MND. This includes establishing appropriate governance as well as a website presence etc.  The scientific focus will initially be on a few key areas which have been identified as blockages to progress but will expand as funds are released and new projects launched.

Firstly, the lack of adequate disease measurements will be addressed since this impacts on both clinical trial design and interpretation. Using collection of biological samples (e.g. blood, urine, saliva) from 1000 people with MND across the UK and combining this with data held in the national registries in England/Wales/Northern Ireland and in Scotland (which established the first MND registry in the world) the aim is to identify and characterise new and existing diagnostic and disease progression biomarkers. These will then be tested in human-based cell models developed within the institute.

The lack of a coherent national pipeline to test therapeutics or characterise biomarkers using human-based cell models is another block to progress. Working toegther, researchers within the institute will set up infrastructure that will enable them to develop a UK MND cell network. This will facilitate the sharing of resources and expertise across centres so that small molecules and genetic therapies can be tested using multiple approaches and validated independently

Understanding and properly exploiting MND-relevant data is also another area where progress could be unlocked. To this aim, just 2 weeks ago, there was an MND-focused data workshop in London that brought together MND Researchers and data scientists from many areas, to explore where the gaps in our knowledge were and what data and steps were needed to address that.

The institute is also embedding training into it plans, with a new Pre-Fellowship scheme has been established to bring early career researchers into the MND space– in another example of partnership working it is funded by the MND Association and administered by MND Scotland.

This is an exciting time to be working in MND, progress in understanding the mechanisms that underly the condition is now starting to identify possible targets and generate new treatments for testing. Clinical trials such as MND-SMART are being established that are open to more people with MND regardless of location where, in the past there was limited access. Now, with the establishment of the UK MND RI a community of talented and committed researchers is coming toegther with common purpose – to find a cure for MND. Because it is about time there was a cure.


  • Balendra R, and Isaacs AM. C9orf72-mediated ALS and FTD: multiple pathways to disease. Nat Rev Neurol. 14 (2018): 544-558. doi: 10.1038/s41582-018-0047-2
  • Al-Chalabi A, et al, Analysis of amyotrophic lateral sclerosis as a multistep process: a population-based modelling study. Lancet Neurol. 13 (2014): 1108-1113 doi: 10.1016/S1474-4422(14)70219-4
  • Chapman et al. Physical activity as an exogenous risk factor for amyotrophic lateral sclerosis: a review of the evidence. Brain. 146 (2023) :1745-1757. doi: 10.1093/brain/awac470
  • Seyhan, A.A. Lost in translation: the valley of death across preclinical and clinical divide – identification of problems and overcoming obstacles. transl med commun 4, 18 (2019). doi: 10.1186/s41231-019-0050-7

Dr Jane Haley Profile Picture

Dr Jane Haley


Dr Jane Haley MBE is Director of Research for MND Scotland. Jane is respondiable for developing and delivering the MND Scotland research strategy and overseeing their wide portfolio of grant funding. Outside the office Jane is a Girlguide leader, Treasurer and Trustee for Girlguiding Midlothian. Her tip for ECRs? Talk to people and willingly collaborate. You won’t regret it.


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