Association Receives ‘Good Practice’ Certification

Back in March I mentioned that the Association’s peer review practices were being audited by the Association of Medical Research Charities. I’m pleased to say that we’ve been given a clean bill of health and are now the proud owners of a new AMRC Certificate of Good Practice.

With increasing numbers of scientists seeking funding and an explosion of new ideas and research avenues to pursue, the process of independent and objective review by other researchers helps us to ‘separate the wheat from the chaff’, ensuring that the research we do fund is of the highest scientific quality and greatest relevance to MND. It also provides our supporters with assurance that we are using their generous donations appropriately and not being led by fashion or vested interest.

Of course, the best review processes in the world won’t guarantee that any specific project will be successful. By its very nature, research is exploring the unknown – to quote Albert Einstien: “If we knew what we were doing, it wouldn’t be called research!”  But it does give us confidence that we are heading in the right direction, towards a World free of MND.

Warning of spam email offering an ‘electro medicine’

We are aware that a number of MND organisations, forums and people living with MND have received spam messages regarding a gentleman who claims that he can stop the deterioration for MND through an ‘electro medicine’ treatment. He urges readers of the message to reply to his email so that he can provide his proof.  We are concerned about this as the mailer, a Mr Oholiav, is selling an unproven treatment for MND that could either be harmful, or non-existent – either a ‘snake-oil’ salesman or a complete hoax.

Trust in evidence not hearsay

We are aware of countless internet sites and scams that offer unproven treatment regimes that ‘prove’ the effectiveness of treatments through anecdotal evidence: through quotes from people who have taken the treatment; through articles in the media and other publications that have not been peer reviewed; case study reports; and documents written by ‘Drs’ that look credible at a glance. However, these treatments have not been through clinical trials, and so there is little proof whether a treatment is safe or beneficial.

A treatment is proven to be effective through carefully controlled clinical trials that test both safety and effectiveness. Trials have to be very carefully designed and carried out to ensure that the treatment is tested in the most unbiased circumstances possible, while giving the treatment the best chance of showing that it is safe and effective. If a treatment successfully shows this through trials, then treatments can be licensed for use. Clinical trial results can then be published in reputable scientific journals – the cornerstone of knowledge sharing in terms of research.

Without researchers publishing their results, and their studies being reviewed by their peers for integrity, we simply wouldn’t know what to believe. Allowing your work and your claims to be subjected to peer review is the internationally recognised way of evaluating research work and medical treatments. And by researchers sharing their knowledge and experience in a controlled setting – through publishing peer reviewed scientific journal articles- we can trust in evidence, not hearsay.

We recently created a new page of our website which discusses what a good ‘gold standard’ clinical trial is if you’re interested in finding out about this.

Sharing evidence: A chalk and cheese affair

As I’m writing this, we’re in the process of proof reading study overviews (abstracts) ready for the International Symposium on ALS/MND to be held in Sydney, Australia from 30 November to 2 December this year. It’s a real opportunity for researchers, clinicians and health and social care professionals to come together to share their recent study results – their evidence.

Flitting between reading the abstracts (which I must add have been peer reviewed), Mr Oholiav’s unproven treatment email, and our news that we’re now part of UK PubMed Central (read Belinda’s Blog entry published this morning for more information on this great news) is a real chalk and cheese affair. It’s a definite case of comparing how research should, and should not, be shared.

There are hundreds of researchers, clinicians, and health and social care professionals around the world dedicated to ending MND. These are the people who methodically set up their research projects; who place great care and attention in publishing their research papers in reputable journals; who attend the International Symposium on ALS/MND to learn about current avenues of MND research; and who share their knowledge with other researchers from around the globe through the appropriate channels. These are the people who have the right tools to unlock the secrets of MND.

We believe that exceptional claims require exceptional evidence. We believe too, that allowing treatments to be made available which have not been through the established clinical trial processes can encourage unscrupulous individuals to market ineffective treatments for financial gain and provide false hope to patients and their families.

We have already received feedback from a number of people affected by MND who have been emailed by Mr Oholiav who have informed us of their anger and upset over his correspondence. They have told us that they have deleted the email as they know it’s content is full of mis-information.

But we know that some people will want to explore unproven treatments as they see it, understandably, as a beacon of hope. This is why we urge anyone contemplating an unproven treatment to contact the research team here at the Association so that we can give them the latest information on unproven treatments to help them make an informed decision.

If you’ve received an email from Mr Oholiav, please let us know your thoughts by commenting below.

Lifting an invisibility cloak -making MND research results more readily available

It goes without saying (but I’ll say it anyway!) that it is important for MND researchers to know what each other are up to – so they can build on results, develop fruitful collaborations and avoid duplication of effort. The MND Association joining the UK PubMed Central (UK PMC) Funders Group is a new way of encouraging researchers to share more results and allow them to move further and faster towards a world free of MND.

The database offers an opportunity for anyone to track what we are funding, who we are funding and which research results were generated as a direct result of our funding. You can also find out which other members of the Funders Group are also funding MND research. These opportunities are in addition to the searchable list of ‘research papers’ that is UK PMC’s key function.

One way that researchers share their results with each other is by the publication of ‘research papers’. These are detailed, technical reports of what experiments were done, how they did them, what the results were and what the authors feel the results mean for their area of research. They are written in quite a structured way, that their peers will be very familiar with.

Where once these research papers were circulated exclusively in hard copy journals and catalogued on library microfiches (remember them?!) increasingly research papers are published electronically and stored and catalogued in online databases. Many of these databases only offer a summary of the research report to the general public; and a library subscription or a login are required to get the full research paper.

UK PubMed Central aspires to make as many papers as possible available in full, increasing the ease of access for researchers and allowing anyone else to read them too. We will be working with our grantees to contribute to this ambition. It’s all part of our drive to ensure that the best possible environment exists for MND research to prosper!

More information on the Association joining UK PMC is available in our press release

Come fly with me

The Fly. Courtesy of MND Association funded researcher Dr Frank Hirth, based at King's College London

Although millions of years of evolution separate humans from insects, a tiny fruit fly called Drosophila melanogaster has been one of the most extensively studied organisms for more than a century, leading to many advances in research. But why are flies so useful? And can we really learn anything from them?

Why fly?

It is easy to see that this fly has advantages in the laboratory. They are very small and easy to keep, but still large enough to study in detail with relatively simple microscopes. They breed easily from 10 days old, producing many genetically identical offspring from each mating. This makes it easy to study several generations over a matter of weeks.

Simple yet sophisticated

Although considered a simple species, the fly is actually quite sophisticated, with structures that are equivalent to organs such as the heart, kidneys and gut.  The brain and nervous system are considered particularly complex, making the fly valuable for the study of neurodegenerative diseases.

Genetically the fruit fly is also much simpler than a human – it has approximately half the number of genes that we do. But it’s not the number of genes you have that counts; it’s what you do with them!

Luckily, about three-quarters of the genes implicated in human disease have a related gene in the fly, with a high level of similarity between the two. Many methods and techniques have been developed, so researchers can switch the fly’s genes on and off at various points in its life-cycle, or in different parts of the body, and then observe the consequences.

MND fly research

Between 2004 and 2009, only about four scientific papers per year described studies using these fruit flies for MND research. In conjunction with the recent upsurge in genetic discoveries related to MND, there has been a rapid increase to twelve publications in 2010, and a further seven already in 2011.

The MND Association is a leader in funding and promoting cutting edge research and we are currently funding two PhD studentships making extensive use of the fruit fly. You can find out more about these projects on our website:

Understanding disease mechanisms of MND in the fruit fly

How does faulty TDP-43 affect MND in fruit flies?

Learning to fly toward drug discovery

There is considerable interest in using the fly to test potential drugs for MND, as there has been some success in this approach in other conditions.  Like the zebrafish model many more substances can be tested than would be possible with a mouse model, and the results may tell scientists more than a cell-based screen. However, this is not yet a routine approach to drug discovery – historically fruit flies have not been used in this way by pharmaceutical companies. It remains to be seen whether any promising compounds identified using fly models will actually progress to being drugs for the treatment of human diseases.    

For such an approach to be useful for MND, there needs to be a reliable and relevant fly model. Recently published work has been focussed on exploring the role of proteins known to be involved in MND such as TDP-43 and FUS. When they publish their work, researchers often hint that their models will be useful in the development of new treatments, even if this was not their main aim.

The use of the fly to discover new medicines may still be some way off, but we can be sure that the tiny fruit fly is already contributing to research in a very big way.

Happiness is…

What brings you happiness? What makes life worth living? Is it going on holiday, spending time with loved ones or just being able to get around?

We are asking people living with MND and carers to post a note or upload a picture onto our Facebook page to explain why the chosen activity brings happiness.

As today sees the official launch of ‘what makes you happy’, we started to think about what makes us happy as the research development team. Here’s our top 5:

 5. When a decision is made on what cutting-edge research projects we should fund, following an accumulation of six months worth of planning (see our video on how we fund research to see the scale of this…)

4. Seeing the International Symposium on ALS/MND through from the planning stages to the conference and knowing that we’ve played a major role in its development to make it the best medical conference on MND

4. When other people and funding bodies spend money on MND research, meaning that our funds can be spread further

3. Feeling that we’ve given people with MND useful information to help them do something positive, such as take part in research

2. Knowing that every penny raised by our incredible fundraisers for MND research is spent wisely and toward our mission of a world free of MND by having rigorous procedures in place

1. New findings published and shared with the world, pushing MND research forward to a new and exciting level

Now that you know what makes us happy, we’d like to know what makes you feel happy on our ‘what makes you happy’ facebook page. Your views really will help shape the MND Association’s future plans as we strive to enable everyone with MND to achieve the highest quality of life possible.