Tirasemtiv not found effective for treatment of MND

In their official press release published on 21 November 2017, Cytokinetics Inc. announced that they will not be continuing work on tirasemtiv after disappointing results in the latest Phase 3 clinical trial. The trial, known under the acronym ‘VITALITY-ALS’, tested whether the drug has a beneficial effect on the breathing function and muscle strength of people with MND. This is very unfortunate news for everyone affected by the disease, however, Cytokinetics are already testing another compound with the hope that this will be more effective and better tolerated than tirasemtiv.

Tirasemtiv is a drug that aims to improve quality of life of people living with MND by increasing strength of their skeletal muscles (controlling body motion and posture) and therefore postponing muscle fatigue. It compensates for the missing nerve signal from a motor neurone to a muscle that instructs it to contract. Tirasemtiv activates a protein called troponin by increasing its sensitivity to calcium, which is crucial for muscle contraction. Continue reading

Symposium abstracts available online

28th Abstract bookThe 28th International Symposium on ALS/MND in Boston, USA is fast approaching with only three weeks to go. Over 1,000 delegates will come from across the world to listen to over 100 talks and see around 450 posters. To see what will be discussed in these presentations, you can now download the full abstract book from the Taylor and Francis website (volume 18, S2 November 2017).

For an overview of the talks, you can visit the Symposium website or download a print version of the programme.

We will be reporting from the Symposium to update you on what was discussed in the sessions via the Symposium website and using #alssymp via our Twitter account @mndassoc.

Introducing the Research Programmes and Partnerships Manager

As I settle into my role as the Research Programmes and Partnerships Manager with the MND Association, I would like to take the opportunity to introduce myself to you all and share with you what my role will entail.

My background is mainly in genetic laboratories (even having the opportunity to extract and test DNA from ancient Egyptian Mummies), although I have worked in a tissue typing laboratory (to make sure that the recipient of a donated organ and the donor matched to avoid the chance of the organ being rejected) as well as a Phase I clinical trials laboratory. Immediately prior to starting with the MND Association, I worked as a Senior Scientific Project Manager for one of the UK’s largest biorepositories, responsible for setting up studies, and collection, processing, storage and shipping of samples. Continue reading

Lithium revisited: Is there a baby in the bathwater?

At last year’s Airlie House workshop to develop new ALS/MND Clinical Trial Guidelines the focus was, of course, on MND, but there was also important input and learning from outside the field.

One of the most fascinating presentations was from an oncologist who was explaining how detailed genetic analysis of tumours was leading to an understanding of why some experimental cancer drugs appeared to only work in a small subgroup of patients. The take home message from the cancer field was that there should be more effort made in future MND trials to identify and analyse smaller subgroups of patients, in case a potentially positive effect might be missed.

A new research paper, published in the journal Neurology, raises some intriguing findings from the trials of the drug lithium that were carried out several years ago. Lithium generated a lot of excitement when researchers in Italy reported a positive effect of the drug in the SOD1 mouse model of MND. Almost as an afterthought, their research paper mentioned that they had tested the drug in a small short-term trial in patients and it appeared to have some effect. Continue reading

Lighthouse Project shines a beacon on HERVs and their role in ALS

There is recent evidence to suggest that Human Endogenous Retroviruses (HERVs) may be involved in amyotrophic lateral sclerosis (ALS). HERV-K has been directly linked to motor neurone damage and has been found in the brain tissue of patients with ALS.

The MND Association recently awarded a small grant to fund part of the ‘Lighthouse Project’ which is investigating the safety and any beneficial effects of an antiretroviral drug on ALS symptoms. Continue reading

Networking to progress in the world of science: Mini-Symposium on MND

Conferences and symposia are a crucial part of the research world – not only for the amount of knowledge that is communicated to large audiences but also for the exchange of ideas on a more inter-personal level. Novel ideas are created there as well establishment of collaborations that might lead to new research projects and clinical trials – all in all, putting a bunch of researchers in a venue with a projector, coffee and biscuits can only lead to good things!

One of the recent events that I had the pleasure to attend was a small-scale conference – the Mini-Symposium on generic disease mechanisms in MND and other neurodegenerative disorders. Held at the Brighton and Sussex Medical School in late June, this event was a precursor to the inauguration of a new MND Care and Research Centre for Sussex, directed by Prof Nigel Leigh. Continue reading

Investigating the role of the cell’s waste disposal systems in TDP-linked MND

In April 2016, Dr Jackie Mitchell gave a talk at the Regional Conference in Gatwick to explain the aims of her three year MND Association funded research project. We have now received her second year report. In this blog we explain a little bit more about what she’s been doing. She has already made some good progress.

A little bit of background
One known genetic cause of MND is a defect in the TARDBP gene, which makes the protein TDP-43, that can be found in the nucleus of a healthy cell. The nucleus is the part of the cell that contains all our DNA. Healthy cells also have two major ‘waste disposal systems’ which break down and remove unwanted proteins from cells. More information on the role of TDP-43 in MND can be found on our blog. Continue reading

Motor Neuron murder mystery: who killed Mr Motor Neuron?

This blog is a fabricated story inspired by the current knowledge of MND.

Today, we wake to news that Mr Motor Neuron (one of the brightest stars in Hollywood) has been killed. A very specialised actor, he was well known for his lightning fast reactions and action-packed roles, often playing characters that had very important messages to deliver.

The alarm was first raised at 2am, when Neuron was found dead in the kitchen of his house by the cleaner, Miss Phagocyte. Dr Riluzole was called and attempted CPR, but nothing could be done to revive him. Early speculation is that he may have been poisoned with a highly toxic protein substance. Neuron’s bodyguards (hired from the prestigious company MicroGlia) have also gone missing, leading many to believe that they too have been murdered. Continue reading

More information for families affected by inherited MND available online

In April this year MND clinician-researchers Professors Martin Turner and Kevin Talbot at the University of Oxford organised an information day about the rare, inherited form of MND called ‘Families for the Treatment of Hereditary MND’ (FATHoM). The day was filmed and podcasts of the talks have recently become available. This article gives an overview of each talk and a link to the video. Continue reading

11th Lady Edith Wolfson Clinical Fellowship awarded

We are delighted to announce that Dr Arpan Mehta has been appointed as our latest Lady Edith Wolfson Fellow, jointly funded by the MND Association and Medical Research Council.  This clinical research training fellowship will help to launch his career as an aspiring academic neurologist, providing comprehensive training in cellular, molecular and bioinformatics technologies in a world-class environment. Continue reading