Using stem cell technology to understand more about how MND and FTD develop

The MND Association are funding Prof Kevin Talbot, Dr Ruxandra Dafinca (née Mutihac) and colleagues at the University of Oxford, who are investigating the link between the C9orf72 and TDP-43 genes in MND. We wrote about this research earlier in the year. As we’ve recently received their first year progress report we wanted to give you an update on what they’ve achieved. Continue reading

Stem cell trials in the news

The recent announcement about the use of stem cells to treat a form of multiple sclerosis (MS), together with early results from the BrainStorm stem cell amyotrophic lateral sclerosis (ALS) clinical trial in Israel have raised the profile of stem cells as a possible treatment for motor neurone disease.

Stem cells are unspecialised cells in the body which do not yet perform a particular function. They can renew themselves and have the ability to give rise to different types of cell, including nerve cells (motor neurones and the surrounding support cells).

Both the ALS/MND study (ALS is a type of motor neurone disease) and the MS study used stem cells found in bone marrow taken from the patient, and then given back to the same patient later on in the process. The MND study gave a new use to the bone marrow stem cells, whereas in the MS study ‘corrupt/damaged’ stem cells were replaced with a new healthier set.

Below we look at both trials in more detail and describe what they mean for people living with MND. Continue reading

Clinical trials update from Symposium

Clinical trials determine if potential treatments are safe and aim to prove beyond reasonable doubt whether a drug is beneficial. They are therefore the gold standard of treatment research.

More information on the different types of clinical trial can be found on our website and in our information sheet on the topic.

This year the Symposium session on clinical trials looked at three drugs and one therapy. Dr Brian Dickie has posted a separate blog on one of these drug treatments – Edaravone.

A summary of the results from the drugs and treatments discussed is below. More information on each of them in detail is later on in this blog.

Ibudilast: This drug was safe and well tolerated in those who were not using non-invasive ventilation. However, these are results from an early stage trial so more research is needed to establish possible long-term benefit.

Methylcobalamin (Vitamin B12 injections): If this treatment is given early (within 12 months of diagnosis) then it showed an effect at increasing survival in a small sub-group of those taking part in the trial. This effect was not seen when the treatment was given further on from diagnosis.

Stem cell therapy: This small, early Phase 1/2 trial was testing the safety of bone-marrow derived stem cell injections into the spinal cord. The researchers found this treatment had no major side effects. Further studies are needed to evaluate the effectiveness and safety of this treatment over the long-term.Clinical trial flow chart

Continue reading

Measuring the nerve impulse

Devlin et al (2015)

Researchers identify that loss of nerve signalling may be an early sign of MND

Published in Nature Communications on 12 January 2015, Association-funded PhD student Anna-Claire Devlin, based at the University of St Andrews, has identified that loss of nerve signalling may be an early sign of MND.

Under the leadership of Dr Gareth Miles and Prof Siddharthan Chandran (University of Edinburgh), Anna-Claire measured the nerve impulses in stem cell derived human motor neurones and identified that the ability to send a nerve impulse is impaired during the early stages of the disease. Continue reading

On the sixth day of Christmas MND research gave to me: Blog a Day 2014

“On the sixth day of Christmas MND research gives to you… on the SIXTH month of 2014 we wrote a Blog a Day, and you can still read them all online today.”

Stem cell-derived human motor neurones are being used by researchers to understand more about MND (image courtesy of Dr Scaber, University of Oxford)

Stem cell-derived human motor neurones are being used by researchers to understand more about MND (image courtesy of Dr Scaber, University of Oxford)

The sixth month of the year is June, which is also MND Awareness month. During 2014 we brought you a blog a day (sometimes two) during the month of June,

Ranging from research updates to experiences of getting involved in research, we covered the variety of research funded by the Association and the work of the Research Development team.

Dr Jakub Scaber’s blog post on stem cells was the top-rated blog post of the month, with an impressive image of stem cell-derived motor neurones.

Click here to read all the Blog a Day posts

On the fourth day of Christmas MND research gave to me: a new stem cell research project

“On the fourth day of Christmas MND research gives to you… on the FOURTH month of 2014, we announced that we’ll fund an exciting new stem cell project”

Prof Linda Greensmith, University College London

Prof Linda Greensmith, University College London

During our April Biomedical Research Advisory Panel Meeting we agreed to fund seven new MND research projects. These projects included Prof Linda Greensmith’s research on Restoring muscle function with transplanted stem-cell derived motor neurones.

Based at University College London, this study will use stem cell technology to restore muscle function in a mouse model of MND. The researchers will transplant stem-cell derived motor neurones and then guide them to where they’re needed using light.

Prof Greensmith and her team aim to restore function to the muscles that are responsible for breathing and develop an optical stimulator, which can then be implanted into the body to stimulate the transplanted cells for long periods of time. If successful, this technique could form the basis of future treatments that could potentially restore muscle function in MND.

Click here to read more about the research that lead to us funding this project

Posters, posters!

During the 25th International Symposium on ALS/MND there were two dedicated sessions for researchers to view over 300 posters. These posters varied from brain imaging to therapeutic strategies. But what is a poster? In this blog I’ll explain more about the session, as well as highlight some of my personal favourites.

Dr Jakub Scaber next to his poster in Brussels

Dr Jakub Scaber next to his poster in Brussels

A biomedical or clinical poster, is in many ways, like an advertising poster. Researchers use colour and text to present their research in a visual way, to engage and discuss their work.

This year’s poster sessions during the symposium were extremely busy, with large crowds often surrounding just one poster and its presenter! The whole room was a real ‘buzz’ of excitement with poster presenters benefitting from the interest and discussion of their work from researchers around the world.

Our Lady Edith Wolfson Clinical Research Fellow, Dr Jakub Scaber (University of Oxford) said: “I didn’t expect such an interest in my work, I ended up being in discussions for well over half an hour – I didn’t even get chance to remove my coat! I really enjoyed the symposium and got to speak to a few more people than I did last year!” Continue reading

Science in neon flashing lights

The opening presentation of this morning’s session of the International Symposium on ALS/MND set the tone for the rest of the day – almost literally, it was as if there was a neon light outside the lecture theatre saying ‘exciting science being presented here’.

Stem cells to study electrical activity of neurones  Continue reading

A taste of Brussels…

Brussels 2014On 4 November, we welcomed two of our funded researchers to our offices in Northampton. Ruxandra Mutihac and Matt Gabel gave us a ‘taste’ of what’s to come for this year’s 25th International Symposium on ALS/MND, by speaking to us about their research.

The symposium is the World’s largest MND-specific research conference and is now only two weeks away! Continue reading

Our DNA Bank: the times they are a changing..

DNABankLogoThis autumn sees an exciting new development in the MND Association’s DNA Bank. Researchers can now use the samples within it to understand why motor neurones die as well as what the triggers are for MND.

How the DNA Bank began

Beginning in 2003 and running until 2012, approximately 1,500 people with MND, 1,000 healthy ‘controls’ – often the partner or spouse of someone with MND – and a further 500 members of families affected by MND gave a blood sample to help researchers understand more about the genetic causes of MND.

Continue reading