On the eighth day of Christmas MND research gave to me: Eight members of the Research Development team

“On the eighth day of Christmas MND research gives to you… EIGHT members of the Research Development team”

We would like to wish all our blog readers Happy New Year! Looking forward, like many others, we have made our New Year’s resolutions of what we’d like to achieve in 2015:

L-R: Sadie, Belinda, Pauline, Lucy, Samantha, Natasha, Laura Front: Brian
L-R: Sadie, Belinda, Pauline, Lucy, Samantha, Natasha, Laura
Front: Brian

Brian Dickie (Director of Research Development): “I’m looking forward to developing closer relationships with other funding agencies, to look at research opportunities that we may not be able to do on our own. We expect researchers around the world to work together, so their ‘joined up thinking’ should be supported by our ‘joined up funding’.” Read More »

MND meerkats raising awareness around the globe

Using social media to establish their presence, the MND meerkats have been raising awareness of MND research. From the labs of Sheffield to the shores of Perth, they’ve covered huge distances in their awareness raising mission. Here Gino, Mo and Oz write about their recent activities:

Gino D’Amino

gino1Gino D’Amino was named by staff at the MND Association prior to the 24th International Symposium on ALS/MND, which was being held in Milan, Italy. He was also lucky enough to get his outfit designed too!

In Milan, Gino was a huge success. After catching up with Mo LeCule during a coffee break he then went on to help sell 100 meerkats – raising 500 Euros for the Association!

After the symposium, Gino visited a number of our funded-researchers during the European Network for a Cure of ALS (ENCALS) meeting in May 2014. As well as being present at these international research events he has also helped members of our Biomedical Research Advisory Panel (BRAP) decide on which new research projects the Association should fund.Read More »

Same disease.. two very different mice!

The exact course, duration and rate of progression of MND often varies greatly from person to person; even when there is a known family history of the disease caused by a specific MND-causing gene (eg SOD1).

This same variability also occurs in mice. Researchers, funded by the MND Association, took two mice with the same SOD1 gene mutation from two different families (strains). By using these two mice the researchers identified a number of key changes in motor neurones that differ between fast and slow progressing forms of the disease.

Two mice… One gene

The SOD1 mouse
The SOD1 mouse model has been one of the most important MND research tools for scientists

Developing new disease models enables us to both understand the causes of MND and test potential new therapies.

Mice are commonly used in MND research and for the past 10 years or more, the SOD1 mouse model has been one of the most important research tools for scientists working in the field, particularly with testing potential new therapies.

Research published in September 2013 was carried out in a joint collaboration between Dr Caterina Bendotti (Mario Negri Institute for Pharmacological Research, Milan Italy) and Prof Pam Shaw (University of Sheffield, UK).

Read More »

Demystifying the symposium

The International Symposium on ALS/MND is the largest MND-specific


conference in the world, which is expected to attract over 900 delegates this year. The planning of the Symposium is a year long event and is pretty much like organising a film premiere, with a number of different teams involved. The ‘symposium team’ ranges from the Conference team to volunteers at the host Association, and from the programme committee to the Research team.

Louise Hough (Communications and Information Assistant at the MND Association) asked us a number of questions about the symposium. We thought you’d be interested in the answers too which we’ve posted below.

Read More »

Screening for Cognitive and Behavioural Change in MND

sharon abrahams and princess royalMND Association-funded researcher Dr Sharon Abrahams (University of Edinburgh) has recently published an article on the Edinburgh Cognitive ALS Screen (ECAS) in the prestigious journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration.

It is now recognised that, in up to 50% of people living with MND not only the motor system (walking, talking breathing etc) but also other areas of the brain, particularly those involved in thinking, language and behaviour are affected.

Frontotemporal Dementia

Cognitive and behavioural changes are increasingly common in MND. It is also well known that a small proportion of people living with MND display features of frontotemporal dementia.

Read More »

Researchers in Australia identify how blue-green algae may cause some cases of MND

A toxin known as β-N-methylamino-l-alanine (BMAA), which is found in blue-green algae, has been shown to cause proteins inside cells to clump together and cause cell death.

This finding suggests that BMAA may be a cause of neurodegenerative diseases like Alzheimer’s and MND and could lead to the development of new treatments.

What is BMAA?

BMAA is a non-protein amino acid. This means, that unlike the 20 amino acids that our bodies use to make proteins, it does not make a human protein.

BMAA is found in a type of bacteria called Cyanobacteria (more commonly known as blue-green algae), which are usually found in waterways as well as damp soil and on the roots of cycad plants.

Blue-green algae can occasionally cause algal blooms. This is when there is a rapid growth of organisms due to high levels of nutrients in the water. The resulting bloom can sometimes become so large that it can be toxic to wildlife.

Read More »

Unproven treatments: Have you really got nothing to lose by trying it?

The only ‘proven’ treatment to slow the progression of MND is Riluzole. There are, however, many organisations offering expensive ‘unproven’ and ‘alternative’ treatments for MND.

We have worked with the organisation Sense about Science to help produce their I’ve got nothing to lose by trying it’ information booklet so that people living with MND can make up their own minds about unproven treatments.

What is an unproven treatment?

Unproven treatments are treatments that have not undergone rigorous testing for their safety and effectiveness by means of a clinical trial. Also, they are often not supported by any reliable evidence.

People or organisations offering unproven or ‘alternative’ treatments may suggest that they will work better than riluzole. They often claim they can cure MND or significantly slow disease progression. These remarkable claims are not supported by any scientific evidence.

ALS Untangled

ALS Untangled
ALS Untangled

The MND Association co-funds an international group of scientists and clinicians, collectively known as ALS Untangled  to investigate unproven or alternative treatments for MND. This group look for the evidence behind these unproven treatments for people living with MND concluding with their own recommendations.

These reviews are freely available and are published Open Access. You can download the most recent review on propofol here.

“Thank you for your support of ALSuntangled! Dr Bedlack et al. are doing wonderful work on behalf of people living with MND everywhere!” – Doug Hetzel, USA

A neurologist’s point of view

Association-funded researcher Prof Ammar Al-Chalabi explains his views on unproven treatments:

Prof Ammar Al-Chalabi
Prof Ammar Al-Chalabi

Many people with MND seek out unproven treatments because of the very difficult situation they and their families are in. My advice is that this is generally OK if the treatment is not harmful, is not expensive and is not obvious quackery. The difference of opinion between medical staff and people with MND mainly comes in the first part – whether the treatment is harmful. As doctors we are taught, “first do no harm”, whereas someone with MND will often say they are dying anyway so it makes no difference if the treatment is harmful because there might be a benefit. This is a powerful argument that can only be countered by the point that someone may have a slow form of MND and be shortening their life significantly or may greatly reduce their quality of life.

“I see people who are desperate, spending tens of thousands of pounds on treatments that are definitely pointless, like stem cell therapy consisting of giving an unknown substance intravenously at an unlicensed clinic. I see others who shun conventional medicine because they believe in alternative therapies, even though conventional treatments have a known side effect profile and alternative treatments do not. As medical staff we have an ethical obligation to provide clear advice in these situations, but I have not had a life threatening illness before and cannot say how I would react, so there is always a seed of doubt in my mind about whether I am doing the right thing.” 

A person living with MND’s point of view

Charlie Fletcher is living with MND and explains her point of view on unproven treatments after reading the new Sense about Science booklet:

Charlie Fletcher

“Having been diagnosed with MND and confronted by the stark reality that there is no cure and incredibly limited medications available, was a hard pill to swallow. To then be bombarded by the media with advert after advert of extraordinary treatments and radical cures was overwhelming. Those with long-term illnesses tend to have a heightened sense of impending doom and a desperate need for hope so it is not surprising that many are swayed by these claims. I will admit that at times the little voice in the back of my mind suggests I might be overlooking the answer I’m after, but as a natural sceptic I am not likely to be tempted unless there is substantial evidence to support these theories.  

“Whilst I can appreciate the draw of untested drugs and alternative therapies, I am not about to start licking trees on the off chance. However, medical research is an integral part of the search for possible treatments for diseases like MND and I am fully supportive of the incredible work being done. In my opinion, participating in clinical trials of drugs and methodologies is beneficial in the development of our knowledge of the disease and eventually a cure, and therefore, is something for which I would willingly offer my assistance.

“It is often difficult to separate the head from the heart, particularly for family and friends who are naturally driven by their need to make things better. The passing on of unlikely remedies and anecdotes for seemingly successful treatments, in my personal experience, is more of a hindrance than a help. Following all this advice can be exhausting, time-consuming and expensive, but more importantly it can create false hope, which can have a devastating effect especially when something doesn’t work.

“To my knowledge there is only one drug available and it is not even close to a cure; as far as I’m concerned, if the only drug that has passed its clinical trials and can be prescribed barely makes any difference, I am not likely to be convinced by the promises made by the latest, untested ‘miracle drug’.

“I find the information presented in this booklet an accurate and in-depth view of the realities of long-term, degenerative conditions and for this reason felt moved to add my contribution.” 

The International Symposium on ALS/MND

Every year in December the MND Association organizes the largest medical and scientific conference specific to MND in the world.

The International Symposium on ALS/MND attracts over 800 researchers, clinicians and healthcare professionals, representing the energy and dynamism of the global MND research community. The symposium in Chicago last year had a record attendance of over 900 delegates with 419 abstracts presented. This year’s event promises to be just as successful with a record number of 510 abstracts!

What is the symposium?


The aim of the symposium is to enable leading researchers around the world to foster strong collaborations and encourages the sharing of new knowledge of the disease as rapidly as possible. To find out more see our website.

This year’s symposium will be held in Milan, Italy from 6 – 8 December and we will be reporting live from the event via this blog. We will be blogging about the exciting clinical and scientific MND research developments discussed at the event; from biomarkers to clinical trials and genetics.

We will also be tweeting from the symposium, as well as posting updates leading up to the event, using the hastag #alssymp.


Abstracts or ‘scientific summaries’ are an overview of a particular piece of research. These summaries briefly explain a piece of research to give the reader a ‘taste’ of what is to come.

All 510 abstracts have been proof-read by myself, Lucy and Belinda in preparation for them to be published Open Access on 4 November 2013. Once published these abstracts can be read by the research community, whom can then register to attend the symposium if they want to find out more!

These abstracts give us an exciting ‘hint’ of what is going to be discussed at this year’s symposium but we won’t know the juicy stuff until the day so-to-speak.


As well as organising and processing all of these abstracts, we also have to compile a programme (that is now available to view online here). This programme shows the schedule of talks, themes and topics to encourage researchers to register to attend the event.

An experience!

Lucy Smith, Research Information Administrator
Lucy Smith, Research Information Administrator

This is Lucy’s first experience of the preparation of the symposium and she summarised her thoughts about the event below:

“I’ve found that organising the symposium is very much like organising a wedding! There’s a lot to do, but you can only do certain things at a certain time – which can be a bit manic at times! For example: Once the withdrawal deadline had passed we had to edit, proof read, check and number all of the abstracts in three weeks!

“I was surprised, being from a science background, that I found the clinical work more interesting than some of the scientific abstracts. In particular what I found interesting was how the work is really changing the day to day lives of people living with MND and making a difference to actual people.”

Milan has already broken the abstract record, we’ll have to wait until December to see if anymore records can be broken!

How to use this blog

The MND research blog aims to provide you with up-to-date information on the research development team’s activities as well as exciting news on MND research. You can view this information anytime by clicking on the ‘How to use this blog’ tab in the top right hand corner.

What is a blog?

The word ‘blog’ stands for ’web log’ and is effectively an online diary where someone can record their thoughts or comments on a particular story/event. The MND research blog is written by the research development team to provide you with updates and comments on recent MND research.

Neural progenitor cells, courtesy of Chandran lab, University of Edinburgh

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I know I read it somewhere..

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Stem cells and growth factors: from bench to bedside

After a brilliant first day at ENCALS, which included a talk by Dr Johannes Brettschneider, the second day began with a talk by Thierry Latran Speaker Prof Clive Svendsen (Director of the Cedars-Sinai Regenerative Medicine Institute) arriving directly from attending at the Anne Rowling Regenerative Neurology Symposium.

Prof Svendsen gave a riveting talk to over 200 delegates, explaining his research on treating ALS (the commonest form of MND) with stem cells and growth factors, and the journey taken from bench to bedside.

The talk began with Prof Svendsen explaining his earlier research into Spinal Muscular Atrophy (SMA) – a genetic disease which causes severe paralysis in children. He explained how he and his collaborators took skin cells that had been banked for over 10 years from a patient with SMA and ‘reprogrammed’ them back into stem cells which were then pushed forward again into motor neurones. Stem cells are ‘immature’ cells, which have not yet ‘matured’ into a specific cell type (eg nerve cell or heart cell). Prof Svendsen’s research was similar to that by Prof Chandran (who did a post-doc with Prof Svendsen) who took skin cells from an MND patient.

Mo Le Cule on our stand at ENCALS
Mo Le Cule on our stand at ENCALS

A little bit of everything is good for you

Like red wine and chocolate (which are both allegedly good for us in moderation) Prof Svendsen highlighted that “a little bit of everything is good for you ” particulary with regards to radiation.

Radiation is a word that people associate with cancer and being dangerous but Prof Svendsen explained that low doses of radiation actually increases DNA repair.  Work by Dr. Seigo Hatada at the Cedars-Sinai Regenerative Medicine Institute has shown that when induced pluripotent stem (IPS) cells are given a low dose of radiation in the lab this enhances the ability to put new genes into the stem cells (homologous recombination) an important technique needed to either label the cells or correct bad mutations.   This is a very important new finding that may help the stem cell field in the future.

Ageing astrocytes

Astrocytes are support cells that are known to play an important role in keeping motor neurones healthy. SOD1 astrocytes (positive for the SOD1 MND-causing gene) were previously found to be toxic to motor neurones but TDP-43 astrocytes were found not to be toxic. Prof Svendsen showed that aged wild type (normal ‘healthy’) astrocytes were also toxic to motor neurones, suggesting that ageing of these cells may have an important role in MND.

Not only were the aged adult wild type cells toxic, they were almost as toxic  as a SOD1 astrocyte (upto 40% more than foetal wild type astrocytes)!

Astrocytes are the key

Replacing damaged motor neurones with stem cells, or healthy motor neurones, is just not possible today”. This is because motor neurones have incredibly long connections and replacing them in the body is a hard thing for researchers to do.

Prof Svendsen explained that replacing astrocytes offered a much better alternative. This is because astrocytes are easy to transplant and are sick and aged in MND. His approach, as described previously at the Anne Rowling Regenerative Neurology symposium, involves a combination of gene therapy and stem cells. Prof Svendsen converted human stem cells into astrocytes and then genetically modified them to produce large quantities of a nerve protecting factor called glial-derived neurotrophic factor (GDNF).

Genetic modification of these astrocytes was carried out by infecting them with a harmless virus. This virus then inserts a gene into the astrocyte, which enables it to produce and secrete GDNF. These modified astrocytes are then inserted into one side of the spinal cord of a SOD1 rat (expressing signs of MND). Prof Svendsen successfully showed that these astrocytes secreted GDNF and protected the motor neurones in the rat at the side of the transplant.

Prof Svendsen explained that the modified astrocytes do not seem to cross to the other side of the spinal cord and are only a ‘partial protection mode’ which means they don’t affect paralysis. They do, however, protect the healthy motor neurones. It is important to note that these experiments used the SOD1 rat model. Only 20% of inherited MND cases have the SOD1 MND-causing gene so this model is not a complete representation of other inherited and sporadic MND cases.   It is now important to try these exciting new stem cell and growth factor treatments directly in patients – they are the only real representation of the disease.

A phase I clinical trial after twelve years of research

Prof Svendsen concluded his talk by mentioning that with funding from the California Institute for Regenerative Medicine (CIRM) he is seeking U.S Food and Drug Administration (FDA) approval for a phase I/IIa clinical trial, which aims to transplant these genetically modified astrocytes into the lumbar (lower) spinal cord of ALS patients.

This trial plans to begin in 2015 by transplanting the GDNF secreting astrocytes into one side of the spinal cord to see the effects on the patient’s legs. Because, the astrocytes can’t cross the spinal cord, this will mean that the researchers will be able to compare both legs to look for differences in disease progression. The trial is double-blinded (with only the surgeon knowing which side the astrocytes are transplanted) and is across three centers in America. Prof Svendsen mentioned that he is on track for the first patient in 2015 providing the safety studies in animals work out as planned.

Thierry Latran Speaker Prof Clive Svendsen (Director of the Cedars-Sinai Regenerative Medicine Institute)
Thierry Latran Speaker Prof Clive Svendsen (Director of the Cedars-Sinai Regenerative Medicine Institute)

Prof Svendsen stressed that this has been a long road and shows just how long it takes to go from making observations in the lab to a clinical trial (he started this work back in 2003).

What next?

Prof Svendsen’s research has shown a great deal of work; including how he converted stem cells into astrocytes, showed that aged wild type and SOD1 astrocytes are toxic to motor neurones, found that GDNF prevented motor neurone death and the start of his clinical trial in 2015.

Prof Sevndsen commented on what the future might be. “If this therapy is found to be effective in ALS patients during this phase I/IIa trial we plan a much bigger trial!! We would aim to move from protecting the legs to protecting respiration – as we have shown the cells can work there too.”

Finally, Prof Svendsen stated what this research means to people living with MND with two simple words. “New hope”