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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Tissue biomarkers: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

Around the globe, teams are working to find tissue biomarkers for MND and there some promising candidates coming through, some of which were explored at session 8C of the Symposium: Tissue Biomarkers.

We first heard from Rebekah Ahmed (C83), who presented her data on possible neuroendocrine and metabolic biomarkers. Ahmed and her team analysed several proteins related to these systems in the blood of 127 people with either MND or MND with FTD and compared them to controls. The Neuropeptide Y protein (NPY), which is related to eating habits and food intake, was found to be higher in people with MND and MND-FTD, and lower in people with FTD, shining the light on a possible biomarker. All patients also had an increase in leptin and insulin levels, highlighting the need to examine how these neuroendocrine changes affect underlying disease pathology.Read More »

Psychological and emotional wellbeing: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

Written by Kaye Stevens and Rachel Boothman

At the 2018 International Symposium on MND in Glasgow, it was positive to see an increase in the number of studies about the psychological and emotional impact of MND/ALS. Read about our highlights below.

From the expected to the unexpected, such as studies which considered the effect of gut health on brain and mood.  (C2) J Cryan – As stress and other factors such as medications can affect gut bacteria, there is a need to maintain a healthy microbiome. This led to a recommendation for sharing refined human poo. Coming your way soon could be ‘Crapsules’ and supplements such as ‘Poopulate’.

(C40) Jane Parkin Kullmann – In other work on stress, researchers in Australia found that stress is not necessarily a risk factor in the development of MND/ALS, indeed it appears that people with the disease may actually be more resilient. Further study is ongoing to determine whether this might indicate a genetic difference.Read More »

Technology and MND: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

Where to start on a subject as wide and varied as technology and MND?

Indeed, this problem is not just limited to a simple blog post, it is a challenge for us as an MND charity faced with a proliferation of potentially beneficial technological developments in smartphones, wheelchairs, and exoskeletons to name but a few.

Fortunately, there is a fundamental question that can help us make sense of it all and it is a question that stems from our Association values – What does this mean for people with MND? I’ll be trying to answer this question as part of my summary of technology talks from our 29th International Symposium.

Much of the content that was presented related to the use of technology in clinical trials, so let’s start by considering clinical trials and what we want from them:

  • We want them to be efficient and report results quickly – this means they will be cheaper, so we can do more of them and secure a cure or effective treatment for MND more quickly.
  • We also want the trials to be reliable and give accurate results whilst allowing as much patient participation as possible.
  • Above all, we want trials to translate into tangible change such as clinical developments that improve quality of life or the introduction of an effective treatment for MND.

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Clinical trials: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

In the Clinical trials and trial design (4B) session we heard from two speakers looking at ways to improve current design of clinical trials. In his plenary talk, Mahesh Parmar (C20) provided his perspective on the necessity of changes from his experience working on cancer trials, highlighting that any efforts to improve clinical trials should be focused on Phase 3 where the most money and time is spent. One solution that stuck with a lot of clinicians attending Prof Parmar’s talk was the design used in the STAMPEDE trial, a large clinical trial assessing effectiveness of new treatments for people affected by prostate cancer, which has been running since 2005. The innovation of this approach is the ongoing protocol that allows to test multiple treatments within the same established clinical trial, allowing new drug candidates to be tested (relatively) straight away, avoiding the creation of a brand new clinical trial. This design improves efficacy of testing new treatments, systematic approach to testing, and access to a large pool of participants who could take part in multiple treatment trials over time.

Brian Dickie, the Director of Research Development at the MND Association said: “Prof Parmar’s presentation generated a lot of interest amongst clinicians who are regularly involved in MND trials and there was a strong feeling that this is the direction that we need to be taking with MND as it could increase the efficiency and reduce the cost. That said, it will take a while to put the building blocks in place and we certainly wouldn’t want to hold up trials that are already in advances stages of planning, so I would expect to see a gradual introduction of changes to trials design over the coming years.”

You can learn more about the multi-arm multi-stage trial design from Prof Parmar here.

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Lifestyle & environment: Highlights from Glasgow

This blog is part of the ‘Highlights from Glasgow’ collection of articles, where you can read about the content of some of the talks and posters presented at the 29th International Symposium on ALS/MND.

In the Epidemiology session (5C), several talks focused on the risk associated with various lifetime events, and the demographics of people who develop MND categorised by onset at various body regions. Susan Peters (C37) and her colleagues studied a group of 1,500 people with MND and 3,000 control participants, and found that people who had suffered head trauma after the age of 55 had an increased risk of developing the disease compared to those without this type trauma. They further found reduced risk in people currently/recently taking antihypertensive and cholesterol-lowering medication, but this risk was significantly increased in people who were taking these medications earlier in life. These findings now need to be explored further to investigate the underlying mechanisms that would explain these differences.Read More »

It’s not just about the neurones

Long before the latest wave of cellular and molecular biology advances started to give us new information on what was going on at the cellular level in MND, some doctors had observed that if the disease started in one particular part of the body, it would be neighbouring parts that became affected next.  This suggested that the disease usually starts in a single part of the brain or spinal cord before spreading further, like ripples in a pond.

How this happens is not well understood. It is likely that there are a number of processes going on, but they can broadly be divided into two theories. One of these is that damaged proteins can leak out of sick neurons and ‘infect’ their neighbours – a subject we have discussed at previous international Symposia.Read More »

Catch up on Symposium…focus on causes and treatments

Following on from our previous catch-up blog on clinical management talks presented at the Symposium, here is a continuation that looks at talks focusing on treatment therapies and causes of MND.

RNA Binding & Transport

RNA is the lesser-known ‘cousin’ of DNA – it contains copies of genetic instructions sent out from the nucleus – the ‘control hub’ of every cell. This RNA is carried out of the nucleus by lots of different proteins, including the RNA-binding proteins TDP-43 and FUS, which act as ‘couriers’ dropping off their RNA at the right part of the cell and then returning to the nucleus for the next package.

These binding proteins both play an important role in motor neurone health. In motor neurones affected by MND, the TDP-43 and FUS seem unable to make their way back to the nucleus so they form clumps in other parts of the neurone. How and why this happens is not really understood and several presentations on the first day of the Symposium provided insight into what might be going wrong.  Dr Brian Dickie, Director of Research Development at the MND Association, summarises these presentations in his blog Libraries, Doormen and Harry Potter. You can also hear Brian talk about RNA proteins on the Symposium website.Read More »

Catch up on Symposium…focus on ‘clinical management’

From abstracts to posters, pushpins to ribbons, it takes a whole year to get to this day – no, not Christmas, but the 28th International Symposium on ALS/MND. In this and the following ‘catch-up’ blog we will summarise what went on at the Symposium and where you can find out more information. To begin with, you can read about what goes into organising the biggest meeting of its kind on our blog:  It’s that time of year again … #alssymp.

Because of the diversity of the talks presented at the Symposium, we categorised them into five key themes that follow the timeline ‘from bench to bedside’; biomedical research, diagnosis and prognosis, causes of MND, clinical trials and treatments, and improving wellbeing and quality of life. You can read more about each of these themes on our Symposium LIVE webpages.Read More »

Lessons learnt in the past year…update on clinical trials

Looking for a treatment for MND is the ultimate goal of the whole MND community. Unfortunately, as MND is a very complicated disease, it is not as easy as it may sound. Setting aside the sheer cost of running trials, researchers have to look at all the possible causes of MND (the genes, lifestyle and environment) and then target these with specific compounds and hoping that this strategy won’t be halted by a different biological process. This is made even harder by the large number of possible combinations of these causative factors and the many different ways these can interact.

Thankfully, lots of research groups across the world are doing their best to tackle the adverse disease mechanisms, which is why we heard lots of results of early as well as late stage clinical trials, new strategies to design better treatments in the future, and lessons learnt from previous studies.

While there was much more to hear and read at the Symposium, here we summarise the Clinical trials session (4B), where five presenters reported results and analyses of the treatments they have been investigating.Read More »