Edaravone (Radicava) approved to treat MND in USA – what does this mean for people with MND in the UK

On Friday 5 May in America, the FDA, the organisation that approves drugs, announced that they’d granted a licence for the drug known as a Edaravone (to be marketed as Radicava ) for the treatment of MND. It’s extremely exciting news and we’re currently working out what this means for people with MND in the UK. Below is more information on what we know so far:

What is this drug and what does it do?
In clinical trials, Edaravone has been shown to slow the progression of MND potentially helping people preserve function longer. Some of the clinical trial results have shown that Edaravone only works on a subset of people at the early stages of the disease – we are seeking to confirm this.

Edaravone is an antioxidant drug that works by mopping up ‘free radicals’ in the body. Our cells have quite effective ways of dealing with free radicals, but these ‘cellular defences’ become less and less efficient with age.

As we age, our energy production processes lose efficiency, causing a ‘double-whammy’ of not only more free radicals being produced, but also less effective ways of dealing with them. When neurones are damaged, as happens with neurodegenerative diseases, then everything gets exacerbated even further, leading to a vicious cycle of events.

It’s a bit like sparks escaping from a campfire – if there are too many sparks and you don’t keep an eye on things, you could end up with the forest ablaze. There’s more information on earlier post on our research blog.

How would people take Edaravone?
Edaravone is administered intra-venously (IV). People with MND would receive the drug every day for two weeks, then take a break for two weeks. A company called Treeway are currently developing an oral preparation of the drug.

What is the process for licencing this in Europe?
We are in contact with Mitsibushi-Tanabe in the USA and have asked them to connect us with their European office in order to understand their plans for licensing in Europe.

The company will have to apply to the European Medicines Evaluation Agency. The drug has already been registered with EMEA as an orphan disease candidate, which means that any licensing application will be fast-tracked. EMEA approval, however, does not ensure UK approval and the drug would need to be approved by the Medicines and Healthcare Regulatory Agency. New medicines are usually also reviewed by the National Institute for health and Care Excellence, which makes recommendations on the cost-effectiveness to the NHS. There is a process for joint MHRA-NICE review which the company will doubtless pursue.

The licencing process does take time, so the company could also apply through the Government’s Early Access to Medicines scheme, which aims to make a drug available where marketing authorisation is not yet approved and there is a clear unmet medical need.

Where can I find out more?
More information on Edaravone is available on the ALS Association website. More information on clinical trials in general is available on our website our website and in our research information sheet. As we learn more about the developments of the drug we will keep everyone updated.

ANXA11 – another gene closer to understanding ALS

A new research paper has been published today in the Science Translational Medicine journal, describing a new gene implicated in developing MND. What is this gene and why is it important for our fight against MND?

Although they are not the sole cause of MND, genes play a big role in someone’s probability of developing the disease. A number of such genes that make a person susceptible to developing MND have already been identified, with most of them causing the rarer, inherited form of the disease.

A new addition to a list of genes that are related to development of ALS, the most common form of MND, has been discovered by researchers from King’s College London. Dr Bradley Smith and colleagues screened genetic data of an unusually high number of people of European origin: 751 with inherited – familial – ALS (fALS) and 180 with non-inherited – sporadic – ALS (sALS). Detailed analysis of this data found that specific mutations in the ANXA11 gene are associated with around 1% of all fALS and 1.7% of all sALS cases. Continue reading

What goes wrong with electrical signalling in MND?

Last year, we introduced a PhD Studentship that we are funding at the University of St Andrews. Under the supervision of Dr Gareth Miles and Prof Siddharthan Chandran, the student working on this project, Amit Chouhan, is investigating why electrical signalling goes wrong in MND.

As the project enters its second year, Amit and the team have made some important discoveries… Continue reading

Epi Epi Epi, Oi Oi Oi

Mention the word Epidemiology and instantly my mind conjures up the Centre for Disease Control (CDC) in America being swarmed by zombies or men in bright orange astronaut-type suits in The Crazies.  While it’s true that it includes studying highly infectious diseases and how they spread (zombies and end of world scenarios aside!), it can be applied to any disease.

Having spent much of my time in the last year working on the data that was collected from our recent epidemiology study, I was keen to shout about the fact that the data is now ready for researchers to use. The analysis of this data will add great value to samples that we already have in our DNA Bank.

What is Epidemiology?

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Exploring the interaction between TDP-43 and RNA

In light of the upcoming Biomedical Research Advisory Panel meeting happening on Friday 7 April that will discuss which new research projects the MND Association will fund, we are pleased to report on the progress of one of our already-funded researchers. In their three year project, funded by the MND Association, Prof Annalisa Pastore (King’s College London) and Prof Gian Tartaglia (University Pompeu Fabra, Barcelona) are investigating the process by which TDP-43 binds to RNA. Below is a summary of the progress they made during their first year.

Background to the project

Alumni Board Meeting 2008

Annalisa Pastore, King’s College London

One of the causes of amyotrophic lateral sclerosis (ALS), the most common type of motor neurone disease (MND), is related to faulty functioning of the TDP-43 protein, a component that is naturally present in all of our cells. In healthy cells, TDP-43 resides in the centre of a cell (the nucleus) where it attaches to RNA and supports correct gene expression – that is, it helps to extract information carried by a gene to form proteins, the main building blocks of our bodies.

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What is the deal with magnetic fields?

A recently published paper exploring the connection between occupational risk factors and MND has sparked lots of interest, especially by the media. The study in question, led by Dr Roel Vermeulen from Utrecht University, The Netherlands, reviewed and studied five occupational exposures that had previously been suggested to be associated with developing MND (specifically, amyotrophic lateral sclerosis; ALS). These factors included exposure to electromagnetic fields, electrical shocks, solvents, metals and pesticides. While a few studies investigating these factors were already conducted in the past, their results are not consistent.

Despite the vast coverage of this topic in tabloids, we wanted to describe the research paper itself – to explain what exactly the researchers did, what they found and what it all means. Continue reading

Funding for Gut-sy MND research announced

Yesterday the Reta Lila Weston Trust announced that they will be funding Dr Nikhil Sharma and colleagues at the Leonard Wolfson Experimental Neurology Centre (LWENC) to investigate whether the bacteria that live in our guts could alter the progression of MND. The grant is for £1.2 million over a period of four years. The LWENC is run jointly by the National Hospital for Neurology and Neurosurgery (NHNN) and University College London (UCL).

Incredibly, researchers have found a link between the bacteria that live in our guts and important cells called microglia. We know that microglia help regulate the function of the motor neurones. This study aims to find out whether the balance of gut bacteria in MND could be linked to changes in microglia. Continue reading

New research projects agreed to help improve palliative and end of life care

Today we announced the results of an exciting new funding partnership with Marie Curie. Together we will be co-funding three research grants that help to answer some questions that people with MND identified as a priority for end of life care research. This is the first time that the MND Association and Marie Curie have worked together with a joint funding call. Each organisation has committed an equal amount of money to the funding of these projects, a total cost of £450,000 over the duration of the projects.

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New urine-based biomarker opens a gate to improved tracking of MND

Researchers from the Flinders University, Australia and University of Miami have discovered a new protein that can act as a biomarker to track disease progression in people with MND. A paper written under the leadership of Dr Shepheard and Dr Rogers was published today in the research journal ‘Neurology’.


What is p75 and what do we know so far

mndassociationgeneral3The biomarker is a protein called p75, which initially
supports the growth of neurones during embryonic development and its levels markedly decrease after birth. Throughout our lives, p75 only reappears in higher levels when the body detects injury of the nervous system, and shows its presence in urine.

The researchers have previously shown that, after birth, mice with a mutation in the SOD1 gene, known to cause MND, had high levels of p75 after about 40 days from the onset of MND. This also coincided with increased levels of p75 in motor neurones found in tissue of people with MND after death.

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Families for the Treatment of Hereditary MND (FaTHoM)

Scientists from the University of Oxford have set up ‘Families for the Treatment of Hereditary MND’ (FaTHoM), an initiative to bring together the community of families affected by inherited forms of MND. Their first meeting will take place in Oxford on Tuesday 18th April.

Most people living with MND cannot identify a relative who has also had the condition. However, around 5% of people with MND will have a family history of the disease, which is known as inherited or familial MND. This happens when a single faulty gene is passed down from parents to their children across number of generations.

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