Stem cells, Symposium planning and Science Communication Conference

As Brian said earlier this week, the news that we are now funding an international stem cell project went national and indeed international through a number of different websites and papers. Getting such cover was fantastic and we’re still receiving enquiries from people wanting to know more information!

At the same time we’ve also been dealing with the abstracts for the symposium. Over the past week we have compiled the scores that an international group of researchers and clinicians who sit on the Programme Committee have submitted to us. After reading over 350 abstracts, I’m sure they’ve got a sense of accomplishment but for a select few, their involvement doesn’t end there…

The Programme Committee give us their time for free. The fact that they read, analyse and judge each abstract in such a short time, whilst conducting their own research studies, being clinicians and generally living their lives is remarkable. We are truly thankful for their help as organising the International Symposium on ALS/MND without their help would be impossible (or at least a lot more stressful!!)

We’ll be taking you behind the scenes next week with what the Programme Committee meeting is like to attend so keep an eye out for a new blog post!

On top of this, three of the science communicators among the team, (Belinda, Kate and Kelly) also attended a Science Communication’s conference in London. In order to make sure that we continue to give you the best research information there is possible, it is important that we attend such conferences to learn more about what’s happening in the world of Science Communication – such as what are the problems, who’s coming up with the solutions and what can we learn from others etc. This event was organised by the British Science Association in collaboration with the Wellcome Trust and overall was an exciting opportunity for us to begin to develop new ideas and gauge what other charities and bodies are up to!

Topics included blogs, tweets, a number of reports on science and society from the Department for Business, Innovation and Skills  and how to engage with parliamentarians. We’re still trying to digest all the information and act upon it.

If you have any ideas, suggestions or comments about the way that we produce research information at the moment – ie our blog, research information sheets and research section of our website then they are always welcomed. New ideas that you would like to see happen would also be welcomed! Please email us at research@mndassociation.org as we’d love to hear from you. Or add a comment on the blog – please note that you do not have to give your email address to comment if you don’t want to but we’d appreciate it if you leave your name.

Mixing with the media

Brian with Profs Chandran and Wilmut

It’s Friday afternoon and I’m just back from a press briefing in London, where we were unveiling an exciting new stem cell research programme. I’ve just realised that we’ve put a press embargo on until Monday, so this won’t be posted until Monday morning – otherwise I’ll be breaking our own embargo…..

The press briefing was held in the Science Media Centre  . The SMC plays a vital role in bringing scientists and the media together, to assist with the accuracy of reporting of scientific issues in the public eye. We’ve developed a very good relationship with them, having held several press conferences at the SMC offices over the years. They were having a frenetic time of it this morning – not only were they setting up our press conference, but they were also dealing with the story of Craig Ventner creating a ‘man-made’ bacterium, which was all over the papers (“Dr God creates artificial life in lab”, was the none-too subtle heading in my morning paper).

‘Dr God’ probably had an impact on the number of journalists in attendance, as many were still out and about following up other reporting angles, but we still managed to attract quite a few of the top science correspondents from TV and radio (BBC, Channel 4) newspapers (Telegraph, Times, Daily Mail) medical press (BMJ) as well as the Press Association (which ensures that the story will go out on the newswire).

By the time I arrived, two of the researchers involved in the project (Prof Siddarthan Chandran and Prof Ian Wilmut from Edinburgh University) were already there. It was straight into the press briefing, where Siddarthan led off by setting out the background to the research programme, for the assembled reporters. Recent advances in stem cell research mean that researchers are now able to model human MND in the lab. Siddarthan explained the promise that research using induced pluripotential stem cells holds for advancing our understanding of MND and for developing methods for the efficient screening potential therapeutic compounds. This is a field that is moving quickly – less than a couple of years ago, the idea that a skin cell could be turned back into a stem cell and then into a motor neuron, would have graced the pages of a Sci-Fi magazine rather than a medical journal.

Prof Ian Wilmut explained in more detail how the stem cells are created in the Edinburgh lab and I followed up by outlining how the other research teams in this initiative, led by Prof Chris Shaw (King’s College London) and Prof Tom Maniatis (Columbia University, New York) would analyse how the cells react under healthy conditions and also under conditions that mimic the damaging cellular environment that occurs in the brain and spine of people with MND. A key message that all three of us put across the importance of international collaboration – if we are going to crack this disease, we have to ensure that the best researchers in the world are working together on the big questions.

You could tell it was a collection of science reporters in the room by the high percentage of intelligent questions – not something you always get when discussing science with the media. Hopefully this press briefing will be the first of many as this and complementary MND research programmes around the world, start to gather pace.

PS – Monday morning. Quite a bit of press coverage, some accurate (well done The Scotsman!) some not quite on the button, but all helping to raise awareness of MND!

GlaxoSmithKline first-time-in-man clinical trial

GlaxoSmithKline (GSK), the pharmaceutical giant, is now recruiting for a clinical trial–often referred to as the NOGO trial. The drug being tested is called ‘GSK1223249’ which isn’t the catchiest of titles which is why it is often refereed to as the NOGO trial!

There are  three centres recruiting from the UK, which are based in Queen Elizabeth Hospital in Birmingham, Royal Free Hospital in London and Addenbrookes Hospital in Cambridge. This drug will be taken by an intravenous infusion at the centres in either one dose, or two doses separated by four weeks. Participants can only be involved with one part of the study and the drug will be tested against a ‘dummy drug’ called a placebo.  This trial will be testing for the safety of these doses (by conducting a number of tests during the trial period) and will not test for the drugs effectiveness.

It is therefore unlikely that people will derive any significant benefit from this trial. However, safety trials are a necessary step in moving the drug forward to a larger trial to test for its effectiveness.

To help GSK recruit for this trial and generally raise awareness of this opportunity, we have created an information sheet which provides more information on what the drug does, what’s involved and the contact details if you think you are eligible to take part.

*note added after posting*

This trial has now completed recruitment. Please see our ‘how to get involved with research’ section on our website for more opportunities to take part.

Raising awareness of MND through Association of British Neurology meeting

A few days ago Professor Karen Morrison rang me. Karen is the director of the Birmingham care centre. She is also one of the principal investigators on our DNA bank project. After giving me an update how she was helping to resolve an issue with the DNA bank, Karen mentioned in passing that she had been at the Association of British Neurologist’s (ABN) meeting the week before.

The aim of the ABN is to improve the health and well-being of people with neurological disorders by advancing the knowledge and practice of neurology in the British Isles. I heard that their annual meeting was taking place last week, so I asked her how it went. I was glad I did! It seems that there was a star* of MND neurologists giving presentations at the four day meeting which took place in Bournemouth.

There was a both a workshop and a teaching session on motor neurone disease. These covered talks on diagnosing MND, managing the symptoms of MND, causes of MND and the cellular mechanisms involved. The workshop was so crowded that they ran out of seats! So awareness of MND of those attending will have definitely been raised!

Professor Michael Swash, a neurologist specialising in MND who has previously chaired our Research Advisory Panel as well as being a former Chair of the Board of Trustees of the MND Association, was awarded the ABN medal this year. The medal is awarded annually to recognise outstanding contributions by British neurologists to the science or practice of neurology, or for contributions to the Association. This news was the icing on the cake!

I hope that this awareness raising will go some way to encourage neurologists to act on their new knowledge, both in terms of caring for people with MND and in keeping up to date with MND research.

*I don’t know that there is a collective noun for neurologists that specialise in MND, but ‘a star of neurologists’ seems quite appropriate – what do you think? Please leave a comment and let us know!

Talk on research at the Association visitor workshop

When the alarm went off at 6.25am on Saturday morning my body shrieked at me to stay in bed! However, as I had accepted an invitation to give a presentation on research to the Association visitor (AV) workshop that was taking place in Harrogate later that morning, it was a case of mind over matter! I was on the road by 7.15am.

 Association visitors   are volunteers that stay in contact with people with MND offering them a friendly, listening ear and ensure they receive the support and services they require. I usually meet one or two AVs when I attend branch meetings, so the workshop was a great opportunity to spend time with them exclusively.

 The talk I gave was broadly based on the recently approved research strategy for 2010 – 2015. The strategy covers seven strategic themes: finding the causes of MND, creating better laboratory models, identifying disease markers, developing the research workforce, improving the clinical /care research base, facilitating information exchange and strengthening partnerships and influencing. For each of these I explained why they are important to move us towards a world free of MND and giving some examples of current activities and future plans. The strategy isn’t on our website yet, but will be soon!

 I talked for about an hour and took some questions afterwards. Some of the questions were about the talk and some were on more general things, for example, there was a question about the lithium clinical trial . There was also a question about raising awareness of MND research amongst neurologists and GPs.

 Making MND research simple and easy to understand is one of my passions, so I was pleased to get some good feedback from the talk. I caught up with more general news and answered a few more questions over lunch. By 1.30pm I was off across the beautiful Yorkshire countryside to visit friends nearby.

Bringing more young talent into the world of MND research through our PhD studentship scheme

In order to move forward in research it is vital that we help young, talented scientists into the world of MND research, as more top-notch researchers equals more top-notch research!

For the past twelve years we have been funding graduate students to learn more about the disease by conducting their own three year research projects under the careful supervision of an expert in MND. Not only do these studentships offer to bring fresh blood into the world of MND research with new and exciting ideas, but real scientific questions about MND research are answered in the process. Once a student has completed their studentship and has passed successfully they are then awarded their doctorate and hopefully choose to further their career in MND research.

However, we can’t (and should not) cherry pick graduates for studentships. The way in which we fund studentships involves researchers – in this case the ‘supervisors’ applying to us for funding. We will only provide funding to projects that can stand up to the full scientific scrutiny of our Biomedical Research Advisory Panel (shortened to BRAP) – so it’s all about the quality of the research instead of the quantity of applications we fund.

Each year we have two grants rounds and the latest grants round is dedicated to studentships. In total, we received four summary applications – which are normally a two to three page overview of the proposed work. Two are asking for funding for projects looking into the causes of the disease and the other two are looking at developing future MND therapies. These will now need to be reviewed by our BRAP for their scientific integrity and relevance to classical MND – those that match the required criteria will then be offered to submit a full application where the whole planned project with all their facts and figures for how much money they wish to spend on each part of the project will need to be justified.

We’ll keep you updated as the year goes on to tell you how many studentships will go through to full application, and what projects have been accepted for funding.

More information on the research that we fund and how we fund research can be found on our website.

X-cell stem cell centre has been investigated by ALS Untangled

With the internet providing an expanse of ‘quackery’ jumbled up with facts, it’s becoming increasingly difficult for anybody to know what source of information can be trusted. This issue is especially apparent to us when we add unproven treatments into the mix. An unproven treatment is, quite literally, a treatment that has no reliable proof for its benefit as a treatment.

As a bit of background, the only way that a treatment can be moved from being ‘unproven’ to ‘proven’ is by conducting a series of controlled clinical trials that can confirm that it is more effective at treating something than a ‘dummy drug’ – called a placebo. This may seem a bit bureaucratic and time consuming but it is a necessary step in finding a truly beneficial treatment for any disease or ailment. Until a treatment has proven itself to be effective, it remains unproven.

So, to shine some light on the clouded situation of where the facts lie within unproven treatments advertised over the internet, a group of international researchers, collectively known as ALSUntangled (ALSU) was set up. ALSU’s most recent investigation was into a stem cell treatment that is provided by a German clinic called ‘X-Cell Stem Cell Centre’.

The X-cell centre is a clinic based in Germany that injects a person’s own stem cells – extracted from their bone marrow, back into them and claims that it can treat MND (as well as a large number of other conditions). To-date, no such stem cell treatment has undergone any clinical trials that have demonstrated their safety and effectiveness. The use of stem cells as a treatment is therefore regarded as an unproven.

ALSU therefore set out to find out if there was any truth behind the X-Cell Centre by investigating:

•  The procedure that they adopt is scientifically sound
• The progression and opinions of three people who went to the X-Cell Centre.

From this, ALSU concluded that the data provided on the X-cell website is flawed and suggest either its removal, or for them to add a disclaimer to alert readers to its flaws. From the small number of people they followed, none showed signs of improvement. ALSU therefore concluded that until they demonstrate the safety and effectiveness through a rigorous clinical trial that they would not condone X-Cell centre’s protocol for people living with MND.

ALSU have published these results in a free to read article in the Journal ‘ALS’, which is written in an accessible way. ALSU also have a twitter page where people can suggest unproven treatments that they should investigate. More information on stem cells as a treatment for MND can be found on the stem cell pages of our website.

ALSU are not alone in their endeavour, as within the research development team a number of us (Brian, Belinda, Kate and I) are able to make sense of the claims of unproven treatments for MND. We provide people with the facts so that people affected by MND can make up their own minds about whether it’s an option they would like to consider.

If you are considering an unproven treatment and would like to know the facts about the information they provide, please contact us at research@mndassociation.org.

*update – The X-Cell centre has now been closed down by the German Government due to a loophole in law being tightened.

The importance of FUS

It is really quiet in the office today, with a few colleagues out and about for various reasons. As soon as the thought entered my head about having a productive day with no distractions, an email landed in my In Box. Had I seen the research report mentioned in this press release? A quick scan of the release and my thoughts were ‘no’ (I haven’t seen it), ‘how exciting’ and ‘well there goes my quiet afternoon’ in quick succession!

 The bottom line of the research is that some MND researchers in Chicago, USA led by Dr Han-Xiang Deng and Professor Teepu Siddique have been able to make a connection between a biochemical pathway recently implicated in the rare, inherited form of MND (known as familial MND) and sporadic MND. They have found clumps of the ‘FUS’ protein in motor neurones of people with familial MND AND in motor neurones of people with sporadic MND too.

 One of the keys to understanding what causes motor neurones to die in MND is to understand which proteins are deposited in affected motor neurones. Deposits, or clumps, of proteins are common to many neurodegenerative diseases, the main difference between the diseases is which proteins are found. A protein called TDP-43 was the first protein discovered to be consistently deposited in the motor neurones of people who had MND. The results from this Chicago research group showing that FUS protein accumulates in most cases of people with MND is the second discovery of its kind.

The efforts of many people around the world will now be focussed on confirming these exciting results which take us closer to understanding the causes of MND.

All of these studies have been conducted using the post-mortem brain and spinal cord tissue of those that have donate these tissues for research after their deaths. A big thank you to anyone who has helped this happen for close family and friends. More information on this generous opportunity to help MND research can be found on our website.

Our symposium abstract journey begins…

The deadline for submitting abstracts for the International Symposium on ALS/MND has now officially passed and this year, 354 abstracts were submitted to us for consideration from a total of 25 countries.

However, the deadline is by no means the end of the journey of the abstracts – it is only the beginning! 

This morning we all (at least mentally) took in a deep breath and rolled our sleeves up as we prepared to have ‘all hands on deck’.

Time then spiralled away from us in a whirlwind of printing the abstracts, sorting them into their different themes (genetics, multidisciplinary care etc) and then photocopying each theme onto different coloured paper –which is a mammoth sized task believe me.

While sorting, the urge to have a sneaky read of the abstracts with the most intriguing titles becomes almost too strong. However, in doing so we could  easily lose a few days as each abstract is roughly 450 words! So for now, we’ll feel content in knowing that we’ve received a strong set of abstracts that we hope will be presented at this year’s symposium – the reading will just have to wait until later on this year!

 The dust is now settling from our manic morning and our reign of the photocopier has come to an end. The result of all this are the piles of coloured paper which now lay waiting to be sent to each member of the Programme Committee – of which Dr Brian Dickie sits on as well as eminent researchers and clinicians from across the world.

They have the difficult task of deciding whether each individual abstract should be rejected or accepted as well as what form of presentation it should be given – ie poster, work in progress or an oral presentation.

With 154 people requesting a talk this year and about 80 spaces available, deciding which abstracts should be given the platform for 15 minutes and which should be offered a poster (or rejected) is a difficult decision that is not taken lightly.

We organise the symposium every single year and seeing it develop from a trickle of abstracts to a wave of hundreds and then to be moulded to form the world’s largest medical conference on MND is one of our proudest moments. We’ll keep our fingers crossed that this year will be no exception!