Collaborating to find treatment for MND

21 June – MND Awareness Day

Each year, the MND Association dedicates the month of June to raising MND awareness. This year, we focus on the eyes – in most people with MND the only part of their body they can still move and the only way left for them to communicate. Alongside the Association-wide campaign, the Research Development team selected six most-enquired about topics, which we will address through six dedicated blogs. 

It is at the heart of the Association to fight MND by funding and promoting research into understanding the disease so that we can defeat it. However, we would not be able to fight this battle on our own and the support of various people is crucial to defeat this MND monster.

Everyone working in the field of MND research has one aim – to find what causes this disease and find a treatment to cure it. We have already written about the long elaborate process behind developing and licensing new drugs but we have not yet talked about the people who are essential for this process to run successfully. Continue reading

The journey of a drug – what it takes to be approved

Each year, the MND Association dedicates the month of June to raising MND awareness. This year, we focus on the eyes – in most people with MND the only part of their body they can still move and the only way left for them to communicate. Alongside the Association-wide campaign, the Research Development team selected six most-enquired about topics, which we will address through six dedicated blogs.

So far, there is no cure for MND. In the past 22 years, we have only seen approval of two drugs that were either shown to prolong the life of MND patients by several months (riluzole in 1995 in the US) or to slow down symptom progression (edaravone in 2015 in Japan). It is only reasonable that you might wonder ‘what is taking so long?’ or ‘why are there not more drugs available?’.

It is very competitive in the world of medicinal drugs. From thousands of chemical compounds that are gradually eliminated as they go through different stages of drug development, only one makes it near the finish line. This line represents approval for marketing authorisation and there is no guarantee that this ‘top compound’ will actually make it to the end. So let’s have a closer look at the individual stages that a potential drug has to go through in order to be crowned the champion. Continue reading

Understanding more about how the cell’s batteries are affected in MND

PhD student Emma Smith has recently started the second year of her MND Association-funded research project at the Sheffield Institute for Translational Neuroscience (SiTRAN) in Sheffield (our project reference: 870-792). With her supervisors Dr Kurt De Vos and Dr Andrew Grierson she is investigating the role of mitochondria in C9orf72-related MND.

basic cell illustration - E Coulthard

Mitochondria (the brown and orange kidney bean shaped structures shown above) provide the cell with energy. Illustration by Emma Coulthard

Mitochondria are the cell’s batteries, providing them with energy. Earlier research has linked damage to mitochondria as a contributor to why motor neurones die in MND. Based on preliminary evidence, the team are aiming to find how the C9orf72 protein causes damage to the mitochondria, where it happens and what might be done to prevent it. Continue reading

Do retroviruses contribute to the common, sporadic form of MND?

New research from scientists at the American National Institute of Neurological Disorders and Stroke in Bethesda suggest that they might. In a research paper published in Science Translational Medicine yesterday, Li, Nath and colleagues proposed that sporadic MND may be linked to an endogenous retrovirus called ‘HERV-K’. So they conducted a series of experiments to investigate their ideas further.

What are endogenous retroviruses?
These are viruses that are our body’s equivalent of fossil – a left over from our evolution many thousands of years ago. Everyone has them but they are normally in an inactive state.

They are a bit like a family heirloom, lets say a vase. You might walk past the vase every day without really noticing it until one day the cat knocks it off and it smashes onto the floor in front of you.

What did the researchers do?
First, they compared brain tissue of 10 people who died from the sporadic form of MND to brain tissue of 10 people who died from Alzheimer’s Disease. They found proteins made by the virus in MND brain tissue but not in Alzheimer’s Disease brain tissue. Next, studying one of these proteins (called ‘env’) in more detail, they found it was toxic to motor neurones.

Li and colleagues then took a step back and asked ‘what triggered HERV-K to become active in the first place?’. (In other words, going back to my analogy, what caused the vase to fall on to the floor?). They found that the trigger was activation by a protein called ‘TDP-43’ – and this protein is already linked MND.

So what does this really mean?
In a comment article giving a wider perspective on the research study, Professors Bob Brown and MND Association grantee Ammar Al-Chalabi concluded:

The exciting observations of Li, Nath and colleagues will provoke further follow up studies that will illuminate the interplay between the biology of endogenous retroviruses and seemingly impenetrable neurodegenerative disorders like ALS”.

So while this study in itself might not give us the answer, its an exciting step forward in understanding the most common form of MND, that other researchers around the world will build on.

Li et al Human endogenous retrovirus-K contributes to motor neurone disease Sci Transl Med 7 307ra153 (2015)

Brown and Al-Chalabi Endogeneous retroviruses in ALS: a reawakening? Sci Transl Med 7 307fs40 (2015)

‘Dormant viruses may cause MND when awoken’ article in The Guardian