Paving the way towards better clinical trials

“The annals of ALS clinical trials is strewn with failed studies. Only two out of more than 70 clinical trials have been positive, and even these showed only very modest benefit.  Is this dismal record strictly due to the extraordinary complexity of neurodegenerative disease in general, and ALS in particular?  Or is it due to methodological flaws that could be repaired?” 

Robert G Miller, Professor of Neurology, Stanford University

Although there is not much we can do about disease complexity, improving the way treatments are trialed is something that can be achieved. Imagine a world without clinical trials, where independent companies or individuals would be allowed to sell their self-made ‘drugs’ without any evidence that they were ever used on anyone with the disease, let alone that they would improve one’s condition. No one would know what the drug is (which could simply be a water solution), how it works and whether as soon as the drug is taken, we would be poisoned.

Thankfully, this is not the case and clinical trials, although not perfect, are considered the gold standard for approving any treatment.  However, there are still some improvements that can be done to make trials easier to access and provide more accurate estimates of drugs’ effectiveness much faster.

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Cognition and FTD: Highlights from Glasgow

Written by Rachel Boothman and Kaye Stevens

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.

Exploration into cognition and frontotemporal dementia with MND/ALS continues to attract attention. At the Symposium in Glasgow 2018, we heard of several studies adding to the growing knowledge bank in this field.

A history of other mental health conditions or psychiatric disorders within the family, or for the individual, indicates a correlation with MND/FTD. There seems to be a link between this increased history and a risk of apathy. Early screening is recommended where these histories exist. This can help prepare families and enable early discussions with the person diagnosed with MND, so they can make decisions that may be important to them in the future (C41) C McHutchison.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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MND research around the world

worldmap annotated and marked

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!

alliance_logo_landscape_rightThe 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.Read More »

Exercise after diagnosis – a closer look at the evidence

Physical activity and MND – part 2

This is part two of our series of blogs looking at links between physical activity and MND. This article will look at whether or not exercise should be continued after diagnosis.

Exercise is widely recommended to the general population due to its benefits to health and wellbeing. It improves the cardiovascular, respiratory, musculoskeletal, and endocrine functions and leads to psychological wellbeing. Many people with MND specifically ask whether they can safely continue to exercise regularly without fear of accelerating their disease. At present, there is no firm evidence that exercise exerts a harmful effect, although avoidance of very strenuous activity would seem to be sensible. Low-grade, managed, exercise programmes may even be of benefit.Read More »

Tackling weight loss in MND – from ProGas to PostGas

Swallowing problems are an incredibly common cause of malnutrition and weight loss in MND patients. To add to this, weight loss in MND is associated with shorter survival. This means managing swallowing problems effectively is crucial to ensuring people living with MND can have the best possible quality of life.

Managing swallowing problems using gastrostomy

Swallowing problems in MND are often managed by placing a feeding tube directly into a patient’s stomach – this is known as a gastrostomy. The feeding tube can either be placed into the stomach via the mouth, or directly from outside the body.

An MND Association-funded study that concluded in 2015 provided much needed evidence on the best method and timing for gastrostomy. This study, known as ProGas, was led by Professor Chris McDermott at the Sheffield Institute for Translational Neuroscience (SITraN).Read More »

Commitment to COMMENDable research

Whilst the vast majority of MND research happens in the lab, there is also an increasing amount of research activity looking into how best to manage the various symptoms of the disease.  There are a lot of unanswered questions as to ‘What Works and What Doesn’t?’ and without a decent level of evidence, it is increasingly difficult in these cash-strapped days to get new or even existing types of therapy adopted into mainstream statutory care.

One such ‘Cinderella’ subject is psychological support for people with MND. It’s hardly surprising that studies show almost half of people diagnosed with MND experience depression and almost a third experience anxiety, yet there is very little guidance on how to best address these symptoms. As a result, formal psychological support is not routinely offered and where it is, the particular approach taken is based on best judgement rather than robust evidence.Read More »

Networking to progress in the world of science: Mini-Symposium on MND

Conferences and symposia are a crucial part of the research world – not only for the amount of knowledge that is communicated to large audiences but also for the exchange of ideas on a more inter-personal level. Novel ideas are created there as well establishment of collaborations that might lead to new research projects and clinical trials – all in all, putting a bunch of researchers in a venue with a projector, coffee and biscuits can only lead to good things!

One of the recent events that I had the pleasure to attend was a small-scale conference – the Mini-Symposium on generic disease mechanisms in MND and other neurodegenerative disorders. Held at the Brighton and Sussex Medical School in late June, this event was a precursor to the inauguration of a new MND Care and Research Centre for Sussex, directed by Prof Nigel Leigh.Read More »

Life of an MND researcher: part 1

Each year, the MND Association dedicates the month of June to raising MND awareness. This year, we focus on the eyes – in most people with MND the only part of their body they can still move and the only way left for them to communicate. Alongside the Association-wide campaign, the Research Development team selected six most-enquired about topics, which we will address through six dedicated blogs.

We all know that rigorous research is the key to finding a cure for MND. Scientists are working hard every day to find the causes of MND, developing new treatments that would help tackle the disease and also looking for new ways to improve the quality of life of people currently living with the disease. But what does it take to have research at heart of everything you do? What is the typical day in the life of a researcher and what does carrying out a research study actually involves?

We asked eight researchers to give us an idea of what their research is all about and what their typical day looks like. Read about four of them in the following blog and keep an eye out for ‘Part 2: PhD edition‘ in the next few days…Read More »