Energy and metabolism in MND cells answer the burning question

Despite the winter chill, there is a warm fuzzy feeling today with the news of a paper published in the journal ‘Brain’ by an MND Association funded Research Fellow, Dr Scott Allen. Based at the Sheffield Institute for Translational Neuroscience (SITraN), Dr Allen was awarded a Senior Non-Clinical Research Fellowship by the Association in 2016, and we are immensely proud to have been able to play a supporting role in his work.

Dr. Allen giving a platform presentation at the International Symposium on MND/ ALS in December.
Dr Allen giving a talk at the International Symposium on MND/ ALS in December 2018.

In his paper, Dr Allen and his colleagues took a novel approach to understanding how MND affects the pathways that are important for making energy in cells of the central nervous system (CNS), that are crucial to keep motor neurons functioning and alive. Specifically, his work has pinpointed a specific mechanism that is changed in MND. The team also demonstrated that there is the potential to tackle this issue by circumventing the problem in order to maintain a critical energy balance in the CNS, and therefore potentially identifying a significant new target in the development of future treatment.

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Cannabis-based products for medicinal use

In November 2018 the Home Office released a draft Guideline scope for Cannabis-based products for medicinal use in which they announced that specialist doctors (like consultant neurologists) on the Special Register of the General Medical Council will be able to prescribe cannabis-based medicinal products to some patients. Before this, the only cannabis-based medicines licensed for use in the UK were nabiximols (Sativex), used as a treatment for spasticity (where muscles are continuously contracted, causing stiffness or tightness of the muscles, interfering with normal movement and speech), in multiple sclerosis (MS).Read More »

What’s the story with CuATSM

There has recently been a flood of news stories on the outcomes of the Australian Phase 1 clinical trial investigating Copper ATSM (CuATSM) which is a small man-made compound that can selectively deliver copper to cells. The results were first presented at our International Symposium in Glasgow back in December.

MND is a terrible disease and anyone affected by it is looking for good news. We really hope that CuATSM will provide a new treatment for MND that is going to have a positive effect on people’s disease progression.

However, CuATSM is not yet at a stage where a clinician can prescribe it as a treatment. Drug development is a long journey, where any drug has to pass important rigorous checks before approval as a medicine. This trial is an important ‘first’ in the drug development process.

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New ALS review article available

ammar2.jpgLast week, The New England Journal of Medicine (NEJM) published a review article by Professors Ammar Al-Chalabi and Robert Brown, in which they looked at the up to date evidence on the incidence of ALS, pathological mechanisms of the disease, as well as genetics and therapeutic strategies.

We would very much like to thank the NEJM who kindly allowed us to share full text of this article on our website – this is now available to view here.

Edaravone (Radicava) approved to treat MND in USA – what does this mean for people with MND in the UK

On Friday 5 May in America, the FDA, the organisation that approves drugs, announced that they’d granted a licence for the drug known as a Edaravone (to be marketed as Radicava ) for the treatment of MND. It’s unexpected news and we’re currently working out what this means for people with MND in the UK. Below is more information on what we know so far:

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Funding for Gut-sy MND research announced

Yesterday the Reta Lila Weston Trust announced that they will be funding Dr Nikhil Sharma and colleagues at the Leonard Wolfson Experimental Neurology Centre (LWENC) to investigate whether the bacteria that live in our guts could alter the progression of MND. The grant is for £1.2 million over a period of four years. The LWENC is run jointly by the National Hospital for Neurology and Neurosurgery (NHNN) and University College London (UCL).

Incredibly, researchers have found a link between the bacteria that live in our guts and important cells called microglia. We know that microglia help regulate the function of the motor neurones. This study aims to find out whether the balance of gut bacteria in MND could be linked to changes in microglia.Read More »

Focus on the research presented in posters in Dublin

Over 100 talks were given at this month’s International Symposium on ALS/MND in Dublin. There were also over 450 posters of research being presented too. Time in the conference programme was allocated on Wednesday and Thursday evening (day 1 and day 2 of the 3 day conference) to visit the posters – you might think that scheduled at the end of the day they would be less well attended – but not a bit of it! It was an extremely loud and buzzy part of the conference.


Below is a brief round-up of some of the posters that caught my eye.Read More »

Prize winning posters in Dublin

As well as all the networking, debate and new information being shared, the International Symposium on ALS/MND is also a time to celebrate achievements by the giving of awards. The Biomedical and Clinical poster prizes are an opportunity to recognise and celebrate the excellent research and clinical practice being conducted by those early in their career.

Now in its fourth year we hope that the poster prizes will help give the winners career a boost, and give them the encouragement and motivation to continue in MND/ALS research.poster-prize-winners-low-res This year the Panel selected an international group of winners: Dr Albert Lee from Australia and Elsa Tremblay from Canada were jointly awarded the Biomedical poster prize and Ruben van Eijk from The Netherlands won the Clinical poster prize. Each winner received a certificate and a glass engraved paperweight.

The prize winning research ranged from understanding the consequences of a newly discovered gene mutation linked to MND, to why the junction between nerves and muscles is one of the earliest signs of motor neurone damage, to a new statistical analysis to make clinical trials quicker and more efficient. Below I’ve explained more about the research that the winners presented.Read More »

IPG Prize recognises young research talent

I firmly believe that the quality of research is only as good as the researcher doing it, which is why the MND Association places a lot of emphasis on providing opportunities to attract, train and retain the brightest and best investigators in the UK and Ireland to develop their careers in MND research. These range from our ‘entry level’ PhD Studentships through to our successful Clinical Fellowships (funded jointly with MRC) and our more recent Non-Clinical Fellowship programme, offering opportunities to outstanding young researchers at a variety of career stages.

It’s also one of the reasons why the Paulo Gontijo International Medicine prize, presented at the Symposium Opening Session, is always an early highlight for me.Read More »

New genetic discoveries tell us more about what causes MND – Part 2

Two sets of MND genetic results were published yesterday. One of these results was about the importance of a new gene called NEK1. The second highlighted the role of gene C21orf2 in MND – we wrote an article about this yesterday. Both sets of results were published in the prestigious journal Nature Genetics.

What are the results and what do they tell us?

Researchers found that variations in the NEK1 gene contribute to why people develop the rare, inherited form of MND. Variations in the NEK1 gene were also found to be one of the many factors that tip the balance towards why people with no family history develop MND.

NEK1 has many jobs within motor neurones including helping keeping their shape and keeping the transport system open. Future research will tell us how we can use this new finding to target drugs to stop MND.Read More »