Disappointing results from UK based lithium clinical trial

Yesterday, we announced on our website the disappointing news that the UK-based lithium clinical trial showed that lithium carbonate is ineffective at treating MND.

Commenting on the lithium clinical trial, Dr Brian Dickie, our Director of Research Development said:

“As many people will know, when lithium was first proposed as having benefit in MND, a couple of small, short-term trials were performed to establish whether the drug had a large and rapid effect on physical changes in disease progression. This trial, by contrast, was developed to ask whether the drug had a more subtle benefit over a longer time course, as is the case with riluzole, using survival times as the primary measure. The only way to answer this question was by performing larger, lengthier and more comprehensive studies.

“While the result is deeply disappointing, we now have a clear answer.

“Lithium can be described as a messy drug. It can act in multiple ways in the body, producing potentially beneficial effects as well as possible unwanted side effects. An overall beneficial effect, even modest, would have refocused scientific interest in the drug to try and separate ‘the good from the bad’ with the longer-term goal of developing more effective compounds. This is a strategy that is presently being pursued with regard to riluzole, in a project co-funded by the ALS Association, the University of Reading and ourselves.

“This trial was the first of its type in the UK, devised and run by clinicians without the need for drug company funding. A number of MND clinics that previously had little or no experience in clinical drug trials for MND have developed vital expertise and confidence in delivering trials to the highest standards. This can only help make the UK a more attractive place in the future for drug companies looking to push potential treatments from lab to clinic.”    

Two hundred and fourteen people with MND took part in this trial, each giving up their time to help find us the answers. We’d like to thank those that have taken part in this trial.

One person who took part in the UK lithium clinical trial was Colin Knight. We spoke to him a few years ago about his views on taking part. Please be aware that in the film clip, Colin speaks frankly about his diagnosis.

 

Read our official press release.

Dexpramipexole clinical trial enrols first participant

We’re aware that thing’s may seem quiet in terms of ‘big’ MND research moments… almost too quiet…

Well, today, the pharmaceutical companies Biogen Idec and Knopp Biosciences announced that the first person has been recruited into their clinical trial to test the effectiveness and safety of a drug called dexpramipexole.

-       Please note: UK trial recruitment has not yet opened.

This is exciting news as it marks an important milestone of a drug reaching a new level in its development. It’s the equivalent of the drug going to University after going to primary school, secondary school and college and passing all its exams with flying colours. It is only by successfully completing these previous phases that the drug can be tested in a large Phase III clinical trial.

With a whopping 804 people with MND set to be recruited into this trial from 11 countries around the world, in three continents, it’s certainly a big trial. Although each centre is set to recruit approximately 10 people, it’s a fantastic opportunity for people with MND from around the world to be involved in this collaborative research effort to find the answer to whether this drug is effective for MND.

With strict inclusion criteria, we are aware that a lot of people will be disappointed that they cannot participate – more information on this can be found in our news in research article on our website.

The official Biogen Idec press release can be found here.

Brain and spinal cord donations provide a timeless legacy to MND research

Tissue donation has played a vital role in many important MND research findings. Without the generosity of individuals who decide to donate their brains and spinal cords to MND research, many of the recent advances wouldn’t have happened or at the very least, the relevance of the findings wouldn’t have been known!

In recent years, tissue donations from patients with the randomly occurring ‘sporadic’ form of MND that account for approximately 90% of cases of MND, and tissue donations from patients with the inherited form, have played an essential role in recent advances.

Timeless legacy
The most recent example of the impact that brain and spinal cord donation has played in MND research is the finding that a gene called VCP causes an inherited form of MND. But what do genetic studies have to do with tissue donation? In order to demonstrate that a mistake in a gene can cause MND, it is important to show the ‘effect’ that the gene mistake had in the body.

In the case of the VCP finding, a brain sample was donated by a patient with MND in the 1970s who had a form of inherited MND. The patient’s descendants then went on to be involved in the study where mistakes in the VCP gene were identified. Even though the brain sample was over thirty years old and had already been used once, the researchers were able to re-use it. By re-staining the sample, the research group were able to show that a protein called TDP-43 accumulates in motor neurones when the VCP gene is faulty.

This is an important finding as it provides further evidence that TDP-43 plays a pivotal role in the development of MND. Without that brain donation back in the 1970s, this finding wouldn’t have happened and we wouldn’t know about the strong link between VCP and TDP-43.

Discovering the importance of TDP-43 through tissue donation
Tissue donated by people with sporadic MND is also playing a vital role in better understanding the role that TDP-43 has in MND. Without people donating brain and spinal cord samples, we wouldn’t know that TDP-43 clumps together in about 90% of cases of MND.

We simply wouldn’t know how important TDP-43 is to MND.

Spot the difference
Having tissue from patients with MND is important, but so is having healthy samples to compare them with. Being able to ‘spot the difference’ between MND and healthy controls is as important as having the patient samples in the first place. Without these samples, it would be like trying to ‘spot the difference’ in one picture.

Make a difference
Tissue donation contributes to groundbreaking MND research leaving a lasting legacy to push our understanding of MND to a new level. It only takes one person to make a massive difference to the future of MND research – just as in the case of the discovery of the VCP gene.

Unfortunately, it isn’t possible to have a look at what happens inside motor neurones of a living patient – the closest we can get at the moment is through imaging studies, which as advanced as they are, are not able, and are not designed, to show what’s happening inside motor neurones. So, the only way researchers can learn about what happens in the brains and spinal cords of patients with MND is to study them.

If you’re interested in donating your brain and spinal cord to MND research then you can read more about it in our tissue donation information sheet.

Please remember that if you are interested then it’s important to set the wheels in motion by talking to an MND tissue bank to ensure that the appropriate paperwork is completed (details of banks are available in linked information sheet). This ensures that arrangements can be made as quickly as possible. It’s also important to tell your friends, family, doctors and neurologists that you would like to donate your brain and spinal cord to MND research so that everybody is aware of your wishes.

Tissue donation is just one way that people affected by MND can have an impact on MND research. If tissue donation isn’t for you, then you can find out more ways to get involved with research by visiting our ‘take part in research’ section of our website.

First results from BioMOx study have been published!

We are pleased to announce that the first results from the Oxford Study for Biomarkers in MND/ALS (known as BioMOx) study have been published in the prestigious journal Neurology.

From this study, a common signature of nerve damage has been identified in the brains of people living with MND using an advanced MRI technique.

The findings demonstrate the importance of MRI in the development of a new biomarker for MND as well as being a significant stepping stone forwards toward two of our research goals – to identify disease markers, and to develop the research workforce.

MND Association’s Press Release
MND Association’s News in Research Article

Identifying disease markers
One of our research aims (set out in our research strategy 2010-2015) that we are working towards is that through our funding, we will have contributed to the identification of disease markers. By funding the BioMOx project (which is ongoing), we are already moving towards this aim.

Developing the research workforce
The BioMOx project is led by Dr Martin Turner from the University of Oxford who was awarded with the Medical Research Council (MRC)/ MND Association Lady Edith Wolfson Clinical Research Fellowship in 2008. This project is ongoing and means that we are not only funding cutting edge research, but we are also aiding Dr Martin Turner to develop his career as an MND clinician and a researcher.

As research is only as good as the researcher, it is important for us to continue to develop the UK basic research capacity by encouraging young clinicians into MND research. We currently fund four fellowships – our most recent of which was announced last month to Dr Pietro Fratta from the University of Sheffield.

Journal article reference: Filippini et al. Corpus callosum involvement is a consistent feature of amyotrophic lateral sclerosis Neurology November 2, 2010 75:1645-1652 

Campaigning for science funding

This week has been quite exciting in the research development team. The symposium abstract book has been printed, the biomedical research advisory panel are meeting today to discuss the applications submitted to us for research funding in May and the campaign to save government spending on science that we are supporting has been stimulating a lot of discussion and debate…

On Saturday 9 October, our very own Dr Belinda Cupid, head of research spoke to BBC Radio Kent about the proposed plans to cut the science budget and what this would mean to us. You can listen to the interview via the BBC iPlayer for Pat Marsh’s show on 9 October 2010 (it starts at 2 hours six mins 46 seconds and finishes at 2 hours ten mins).

Later that day, our president, Prof Colin Blakemore spoke at the Science is Vital rally held in London. Colin has been heavily involved with the campaign and was interviewed by the BBC alongside a Science is Vital representative. You can see the BBC coverage here: http://www.bbc.co.uk/news/uk-11508105

Cuts to medical and health research may further marginalise ‘orphan’ diseases like MND which traditionally receive less government funding.

So far, at least 16 people living with MND have signed the petition – which has been mentioned in a New Scientist blog article. So, thank you so much to all those who have already signed as your voices really are being heard!

If you haven’t, and are interested in getting involved then please visit http://scienceisvital.org.uk for more information.

Today, the MND Association was given a high profile in two articles in The Times, warning the government against cuts to the science budget.

The first is a letter written by the Association of Medical Research Charities (AMRC), warning the government of the effects that potential funding cuts could have. As the Association is a member of the AMRC, our chief executive Kirstine Knox was a signatory on this letter. We have received permission to share it with you:

Sir,

Our work benefits millions of patients across the UK. Last year alone the 124 members of the Association of Medical Research Charities funded more than £1 billion of medical and health research. As a proportion of public expenditure that is more than any other country. This contribution is driven by the combined efforts of volunteers, supporters, donors, clinicians, scientists and patients themselves. If ever there was an example of the “Big Society” in action this is it.

Ahead of the Comprehensive Spending Review (CSR) we have asked the coalition Government to sustain science funding and ensure an environment that allows charities to fund research on behalf of patients. Failure to do so will lead to the UK losing its position as an international leader in science. Additional unacceptable strictures on research, such as the proposed cap on non-EU migrants, can only fuel concerns that our future scientists will be expected to work with one hand tied behind their backs.

Ministers are mistaken if they believe that charities are a substitute for Government expenditure. One of the great strengths of UK science is the synergy that exists between public, charitable and industry sources of funding. Only last week we saw evidence of what this collaboration can mean with the bowel-screening announcement heralded at the Conservative Party conference. It is such progress and the opportunity to improve health and wellbeing that has enabled us to build public support for research, support that leverages funding from other sources for the common good.

We recognise the very difficult decisions facing George Osborne. But in these final days before the CSR announcement he may wish to reflect on the comment by the American health activist Mary Lasker: “If you think research is expensive, try disease.

Yours faithfully,

AMRC member charities

The second Times article is an overview written by Mark Henderson which refers to the letter and specifically mentions the MND Association alongside such funding bodies as The Welcome Trust. Having our name printed alongside major funding bodies is brilliant coverage for both motor neurone disease and the Association!

We’ll keep you posted on the progress of the campaign via our blog and our twitter account at www.twitter.com/mndresearch.

We are supporting the ‘Science is Vital’ campaign

We are backing a campaign calling for the Government to recognise the importance of science; and to lay out a supportive strategy for UK science and engineering which maintains a level of investment in line with economic growth.

The ‘Science is Vital’ coalition is made up of concerned scientists, engineers and supporters of science, who are campaigning to prevent the destructive levels of cuts to science funding in the UK. The coalition warns that Government spending cuts could result in ‘an unprecedented scientific crisis’.

It is predicted that Government funding for medical research will be slashed in the Treasury-led Spending Review – part of the Government’s austerity drive. The medical research community needs to act now to prevent counter-productive, short- term cuts.

The Association fears that spending cuts will result in a decrease in MND research activity, seriously harming the UK research community’s ability to make major breakthroughs in the understanding of MND.

Research into rarer conditions such as MND already remains poorly funded compared to more common long-term conditions; and the Association is campaigning for increased investment into MND research.

You can support the Science is Vital campaign by signing the petition at www.scienceisvital.org.uk.

Busy times in the research team!

The last few days have been quite a hectic for the research development team for many reasons.

Annual conference workshop
This year, our Annual Conference and AGM were held in Nottingham on 11 September. For this event, Kate and I had been set the challenge of organising a workshop on taking part in research. In our workshop, we explained how the contribution of people living with MND and healthy individuals is so important to moving MND research forward. We also gave an idea of the wide range of ways in which people can take part and how much difference there can be between one project and another in terms of the time a participant needs to commit, how much they need to travel, and of course what they actually have to do.

After showing a video clip of Colin Knight, who is living with MND talking about his reasons for taking part in research (we’re currently waiting for approval to put the video on here – so look out for it!), we then asked our audience to read out a few quotes that we’d received from MND researchers. Here’s one of them:

“Without the generous support of patients with MND who donate DNA and tissue to our research department, much of the work that I do would literally be impossible. I am constantly amazed that at a very difficult time in their lives, people have such commitment to provide time and energy to participate in clinical trials and studies, and this is very inspiring to those working in this field” Dr Alice Brockington, clinical researcher.

Taking part in research can be inspirational for both people living with MND and researchers alike. Seeing the determination of both groups to push research forwards really demonstrates that we’re all on the same path and are all looking to help in some way to find the answers to MND and hopefully, one day find a cure.

We’re optimistic that everybody went home with a clearer idea of how they might be able to participate in MND research!

Preparing for the Biomedical Research Advisory Panel (BRAP) meeting
We’ve also been busy preparing the paperwork ready for the next BRAP meeting, who will meet in exactly a month’s time to discuss five applications that have been submitted to us for funding through this grant round. If any of these are successful, we’ll let you know once the start details and costs etc have been approved by our Board of Trustees.

Welcome Dr Sadie Vile!
Last week, we also welcomed our newest addition to the research development team – Dr Sadie Vile, our research grants manager. As she becomes more settled into the role, we’ll ask her to introduce herself on the blog!

Dr Brian Dickie to be drawn by Patrick – the incurable optimist!
Last week we also heard that Brian had won the online vote on Patrick’s Facebook page to be painted next in his quest to paint 100 optimists. Needless to say, Brian was extremely delighted at this news and we all can’t wait to see the painting develop!

One small step for MND research… one giant leap for the MND Association’s DNA Bank

We’ve been extremely excited about this research finding for a while now and have closely followed the progress of Prof Ammar Al-Chalabi’s publication as it was accepted into the prestigious journal Lancet Neurology. At times this was a rollercoaster journey – one that we were carefully watching from the sidelines with baited breath.

The reason why we’re more excited than normal is that this is also the first time that results from DNA samples from our very own DNA bank have been published. The DNA bank has been collecting blood samples from people living with MND, family members and controls in the UK for over seven years and it now collectively holds nearly 3,000 samples.

Over 600 of the samples donated by people living with sporadic MND were used in this ‘whole genome wide association study’ (which uses state of the art technology to scan the genetic code for spelling variations as compared to healthy controls) to find a region of DNA (that contains three genes) within chromosome 9 that is associated with sporadic MND, a condition called ‘fronto-temporal dementia’ (FTD) that affects behaviour, emotional response and language skills, as well as a rare inherited form of MND called MND-FTD.

This means that people who have already donated a blood sample to our DNA bank have played a vital role in this research finding. As all of the samples are anonymised, it is not possible to find out which samples have been used in this research project.

So, this is not only another ‘hop’ forward onto the next stepping stone on our journey to understand more about the underlying causes of MND, but it’s also an exciting step forward in the development of the DNA bank. We hope this is the first of many!

At present, only the researchers who have helped us to set up the DNA bank can apply to use the samples in their studies but at some point in the near future we will open the bank to other researchers so that they can apply to use them in their studies.

Read our news in research article on this finding
Read our press release on this finding
Read the scientific paper:  Shatunov A et al. The Lancet Neurology 2010; DOI: 10.1016/S1474-4422(10)70197-6

Thank you to those who took part in ‘Voicing you Views’ project

Amanda Dean, a PhD student who we’ve been helping to find recruits for her project to ‘voice the views’ of people living with MND, has sent us an email to thank all those who have been involved, as well as those who expressed an interest in taking part. Here’s what Amanda had to say:

Dear All

What seems a very short time ago I appealed to you all, searching for a few who might be willing to take part in my research. As I sat back and nervously waited for my in box to light up I wondered if I would be successful in gaining a handful who might be interested in meeting with me, maybe a few more that may take part via e-mail. But in fact what I got was an overwhelming response that at times kept me up into the small hours trying to respond to you all. My introduction to research within the MND community could not have been more welcoming or encouraging.

In an ideal world I would have liked to have met every one of you, spoken with you, and let you know personally how much I appreciated your time, your insight and the level of detail you included in your responses which has provided this research with so much depth so far. I feel  privileged to have met, either in person or online, some truly inspirational people, bubbling with positivity, stories of achievement, bravery. I have shared moments of humour, moments of happiness, and some moments of sadness too…

I wanted to take this opportunity to thank everyone that got in contact; this includes those that were not able to take part on this occasion but expressed an interest and offered their help. It is important to me that each and every person who gave their time freely for this research, and this includes those who wrote or typed on behalf of others, got a heartfelt thank you, and that knew that their input was greatly appreciated. I hope some of you might feel like you can stay in touch, you would be most welcome!

I hope that in the future I may meet some of you at a conference, but until such a time, take good care of yourselves. With all my best wishes,

Amanda

As Amanda has now finished recruiting people living with MND to voice their views, it is no longer possible for people to take part in this project. If you are interested in taking part in another study, please look at our ‘how to get involved with research‘ pages of our website.

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.