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.

How do we fund research?

As we’ve mentioned in previous posts, our current research grants round is our biggest yet with 19 full applications being reviewed by our research advisory panel. But what does that look like? And what do we have to do to prepare these applications for our research advisory panel?

To answer these questions (and more), and give you a brief insight into the work that our research grants team do to ensure we fund the very best research, we’ve made a short film featuring Dr Sadie Vile, our research grants manager and Natasha Rowe, our research grants administrator as the voiceover.

To find out more about how we fund research please visit our website.

Please do also let us know what you think about our video by leaving a comment below!

Completing our peer review audit for the AMRC

Form filling is tedious.

For those of us of a certain age (I’m not saying any more!) online form filling is even more stressful. So recently, I ‘girded my loins’ and completed the Association of Medical Research Charities (AMRC) 2010-11 Peer Review Audit.

AMRC is a body that represents over 125 medical research charities across the UK, ranging from small organisations through to the big boys such as the Wellcome Trust and Cancer Research UK. It plays an important role in facilitating information exchange between charities and in influencing national science policy by providing disparate organisations with a single voice. It also acts a little like a Trade Association, setting standards for the charity research sector.

One such standard we have to achieve relates to the research we fund. Every five years, the AMRC performs a comprehensive audit to ensure that the decision making process is impartial, independent of vested interest and transparent to both researchers applying for funding as well as donors supporting the research.

It might sound like an exercise in bureaucracy, but it is important part of demonstrating that we are using our funds to support research of the highest scientific merit and greatest relevance to MND. The AMRC ‘kite mark’ is also important in enabling us to raise money for research – for example, some charitable foundations will only accept applications from charities that can produce the AMRC Certificate of Good Practice. Similarly, our collaboration with bodies such as the Medical Research Council stems from our ability to demonstrate good research governance.

Of course, no two charities are the same and there may be some places where we do things a little differently from others, due to the nature of the disease or the specific research avenues we are exploring, but the important thing is that the key principles of impartiality, accountability and transparency are being followed.

In truth, the fifteen page form wasn’t as taxing as I thought, largely because last year we established our Research Governance Framework to make our process more transparent.

Still, I’m not disappointed it’ll be another five years….

Blue-green algae called Spirulina investigated by ALSUntangled

An unproven treatment called ‘Spirulina’, which is a type of blue-green algae, has recently been investigated by ALSUntangled*. They concluded that there is no evidence that Spirulina is effective for treating MND. Their findings also suggest that it could be toxic to people with MND.

In their investigative paper, which is free to download and easy to read (link at the bottom of this article), ALSUntangled discuss the research behind this unproven treatment.

The study that was critiqued examined the effect of feeding mice that model MND with Spirulina against those that were not fed the supplement. From their findings, the research group concluded that “a Spirulina supplemented diet may have future clinical benefit in treating ALS as an alternative or adjunctive therapy”. By reviewing the original research paper, ALSUntangled did not come to these same conclusions and identified significant flaws in the study.

For example, the study in question had no data on whether it slowed progression of motor symptoms. The treatment was also given before the onset of the disease, which is not clinically possible for people with MND.

ALSUntangled also used PatientsLikeMe.com as a resource to find out how many people had taken Spirulina. All six members with ALS (the most common form of MND) who had stated they were taking blue-green algae had decided to stop taking the treatment due to lack of effectiveness or cost –which had been reported as being between $50-$200 a month.

 

Importantly, the possible toxic effects of Spirulina are also discussed in the ALSUntangled paper, stating that it could theoretically accelerate the progression of the disease.

 

 Read the ALSUntangled article published on in the journal ALS:  http://informahealthcare.com/doi/pdf/10.3109/17482968.2011.553796 

 *ALSUntangled is a group of international clinicians/researchers in MND who investigate the claims of unproven treatments.

 

 ALSUntangled also have a twitter page where people can suggest unproven treatments that they should investigate.

ALSUntangled are not alone in their endeavour, as within the research development team a number of us 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.

Painting the way to new motor neurones from stem cells

A study led by MND Association funded researcher Prof Siddharthan Chandran from the University of Edinburgh has developed a new method to create a diverse group of motor neurones from stem cells. The research, published in the journal Nature Communications could be used to create more accurate and clinically relevant laboratory dish models to learn more about the differences in vulnerability and connectivity of motor neurones in MND.

 Why are the subtypes of motor neurones important to MND research?
When we first start to develop as embryos in the womb, chemical messages are used as cues to tell our cells what to turn in to. At the start of this process our cells can be thought of as blank canvases that have the potential to turn into any type of cell. Mixtures of ‘colourful’ chemicals are then used to create a unique ‘hue’ signal in order for the cell to know what to become.

So, depending on the ‘hue’ of chemicals around them, neuronal precursor cells will turn into different subtypes of motor neurone. In their fully formed state, these motor neurones subtly vary in their chemical makeup (due to acting on the different ‘hue’ signals given), their vulnerability to degenerate in MND, as well as the way they connect and communicate with other cells.

The subtle differences in subtypes of motor neurone have not been replicated in a laboratory dish model of MND to date. However, being able to develop such a model would provide MND researchers with a true spectrum of the way that MND affects the different subtypes of motor neurones. They would then also be able to develop new and better treatments that can target specific types of motor neurones that may be more vulnerable to MND.

What did the researchers do to find this?
The collaborative research group from Universities of Edinburgh, Cardiff and Cambridge tested a new method for creating different types of motor neurones in a dish from human embryonic stem cells.

To do this, they first added a chemical that accelerates the process of turning stem cells into neurone precursor cells – it’s the equivalent of being able to add a ‘quick drying’ additive to a painting. By adding this chemical, which has been given the catchy name of SB431542, the process of changing an embryonic stem cell into motor neurone progenitor cells is sped up from approximately 30 days to just 12 days.

They then tested whether a certain chemical called ‘retinoic acid’ is needed for the process of making different types of motor neurone. By measuring the chemical makeup of the functional motor neurones produced without retinoic acid, they were able to determine that they had produced a different type of motor neurone that is different from those created with the use of retinoic acid.

What’s next?
By defining a new process to create new and better models using stem cell technology, a new multi-motor neurone type model could be created for MND to study the similarities and differences between motor neurones in MND.

By learning more about these differences, we could learn more about how and why some motor neurones remain spared in MND.

To find out more about the future of stem cell research, please read Dr Brian Dickie’s account of the recent stem cell conference. Or, download a copy of our stem cell information sheet.

Journal Reference: Patani, R. et al. Retinoid-independent motor neurogenesis from human embryonic stem cells reveals a medial columnar ground state. Nat. Commun. 2:214 doi: 10.1038/ncomms1216 (2011).