MIROCALS: Episode MND

A clinical trial of Jedi proportions…

The MND Association is backing a new clinical trial in MND, known as MIROCALS. This will be a joint clinical trial between France and the UK that will aim to dampen the overactive immune system by increasing the amount of interleukin-2.

It is important to stress that planning for this MND clinical trial has only just started and the next step is to lay the essential groundwork and perform some short-term pilot studies. The main trial is likely to begin recruiting participants in autumn 2016.

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An insight into TDP-43 – An ENCALS 2015 meeting report

Dr Jakub Scaber, University of Oxford, UK
Dr Jakub Scaber, University of Oxford, UK

Medical Research Council (MRC)/ MND Association Lady Edith Wolfson Clinical Fellow, Dr Jakub Scaber, attended the European Network for a Cure of ALS (ENCALS) meeting from 21-23 May 2015.

Reporting back from the event in Dublin, Dr Scaber summarised the TDP-43 session, including his presentation on recent developments in his own Association-funded research:

The fifth session of the ENCALS meeting focussed on a protein called TDP-43: This is the protein that accumulates in the brains of people living with MND and has been tightly linked to the development of the disease. Abnormal forms of this protein can be found in 98% of cases and this session had some very interesting basic science discoveries around this topic. Read More »

Oxidation Resistance 1 – tackling oxidative stress

MND Association-funded researchers, Prof Dame Kay Davies and Dr Peter Oliver, both based at the University of Oxford, have identified the oxidation resistance 1 (OXR1) gene as a neuroprotective factor in MND.

Published in the journal Brain on 9 March 2015, Prof Davies has shown through their recent research in mice that OXR1 may serve a new target for future drug development.

We’re funding Matthew Williamson’s PhD studentship to follow up this exciting research. Find out more about this and the other research projects funded by the Association in our newly updated Research we fund information sheet.Read More »

ProGas study results on gastrostomy in MND published

Under the leadership of Dr Christopher McDermott, based at the Sheffield Institute for Translational Neuroscience (SITraN), research published today on 29 May 2015 in the Lancet Neurology highlights that better weight management in MND is key to survival.

Following on from initial results presented at the 25th International Symposium on ALS/MND in December 2014, the Prospective Gastrostomy (ProGas) study in MND aimed to investigate the optimal timing for gastrostomy in MND due to the lack of evidence available.

Dr Chris McDermott (Sheffield Institute for Translational Neuroscience, University of Sheffield)
Dr Chris McDermott (Sheffield Institute for Translational Neuroscience, University of Sheffield)

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Breaking the Human Genome Code

Dr Johnathan Cooper-KnockProfessor Winston Hide gave his inaugural lecture on 17 March, during Brain Awareness week, entitled ‘breaking the human genome code – opening Pandora’s box’, which you can watch in full at the end of this blog post.

Professor Hide recently joined the University of Sheffield, and MND Association/ Medical Research Council (MRC) Lady Edith Wolfson Clinical Research Fellow, Dr Johnathan Cooper-Knock has written a blog below about Professor Hide’s research and how they are working together towards a world free from MND: Read More »

Researchers identify the TBK1 gene as a risk factor in MND

recyclePublished on 19 February 2015 in the Journal Science, an international team of scientists have found mutations in the gene TBK1 as a contributory risk factor in MND.

Identifying TBK1

The majority of cases of MND are caused by a combination of subtle genetic, environmental and lifestyle factors. These subtle genetic factors in the majority of cases of MND (sometimes known as susceptibility genes) may increase someone’s risk of developing MND, but they do not solely cause the disease (they must be present in combination with a number of other factors in order to tip the balance for someone to develop MND). Find out more here.

Under the leadership of Dr Goldstein, based at Columbia University, the researchers have identified a new MND susceptibility gene – TBK1.  The researchers used whole genome sequencing to sequence the entire DNA of over 2,874 MND samples in America – you can find out more about this technique here. By screening a large number of samples, the researchers identified mutations in the TBK1 gene as a common subtle genetic factor involved in some cases of MND in America.Read More »

Making antisense of RNA editing

helena chaytow 2Association-funded PhD student, Helena Chaytow (Royal Holloway, University of London), is using DNA to develop a targeted treatment for MND. Now entering her final year, we report on what she’s achieved so far and her future plans.

The background

Helena’s research is looking at the chemical messenger ‘glutamate’. Glutamate is released by motor neurones in order to stimulate a nerve impulse from one motor neurone to the other, which is how the instruction to move our muscles travels from our brain to our limbs.

In order to pass the message on, glutamate needs to bind to the second nerve cell, and it does this by acting like a ‘lock and key’. Read More »

New fellowship awarded to further our understanding of RNA in MND

Dr Pietro Fratta (University College London) received his initial Training Fellowship through the MND Association/ Medical Research Council (MRC) Lady Edith Wolfson Programme in 2010. Starting on 1 February 2015, Dr Fratta was awarded a Clinician Scientist Fellowship to continue his research into MND.

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Totalling £1.16 million, of which the Association has committed to contribute £280,000, this new fellowship will allow Dr Fratta to find out what RNA molecules are present in both the cell body of the motor neuron, and the nerve fibres. Read More »