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.

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

Focus on the research presented in posters in Dublin

Over 100 talks were given at this month’s International Symposium on ALS/MND in Dublin. There were also over 450 posters of research being presented too. Time in the conference programme was allocated on Wednesday and Thursday evening (day 1 and day 2 of the 3 day conference) to visit the posters – you might think that scheduled at the end of the day they would be less well attended – but not a bit of it! It was an extremely loud and buzzy part of the conference.

Below is a brief round-up of some of the posters that caught my eye. Continue reading

Prize winning posters in Dublin

As well as all the networking, debate and new information being shared, the International Symposium on ALS/MND is also a time to celebrate achievements by the giving of awards. The Biomedical and Clinical poster prizes are an opportunity to recognise and celebrate the excellent research and clinical practice being conducted by those early in their career.

Now in its fourth year we hope that the poster prizes will help give the winners career a boost, and give them the encouragement and motivation to continue in MND/ALS research.poster-prize-winners-low-res This year the Panel selected an international group of winners: Dr Albert Lee from Australia and Elsa Tremblay from Canada were jointly awarded the Biomedical poster prize and Ruben van Eijk from The Netherlands won the Clinical poster prize. Each winner received a certificate and a glass engraved paperweight.

The prize winning research ranged from understanding the consequences of a newly discovered gene mutation linked to MND, to why the junction between nerves and muscles is one of the earliest signs of motor neurone damage, to a new statistical analysis to make clinical trials quicker and more efficient. Below I’ve explained more about the research that the winners presented. Continue reading

IPG Prize recognises young research talent

I firmly believe that the quality of research is only as good as the researcher doing it, which is why the MND Association places a lot of emphasis on providing opportunities to attract, train and retain the brightest and best investigators in the UK and Ireland to develop their careers in MND research. These range from our ‘entry level’ PhD Studentships through to our successful Clinical Fellowships (funded jointly with MRC) and our more recent Non-Clinical Fellowship programme, offering opportunities to outstanding young researchers at a variety of career stages.

It’s also one of the reasons why the Paulo Gontijo International Medicine prize, presented at the Symposium Opening Session, is always an early highlight for me. Continue reading

New genetic discoveries tell us more about what causes MND – Part 2

Two sets of MND genetic results were published yesterday. One of these results was about the importance of a new gene called NEK1. The second highlighted the role of gene C21orf2 in MND – we wrote an article about this yesterday. Both sets of results were published in the prestigious journal Nature Genetics.

What are the results and what do they tell us?

Researchers found that variations in the NEK1 gene contribute to why people develop the rare, inherited form of MND. Variations in the NEK1 gene were also found to be one of the many factors that tip the balance towards why people with no family history develop MND.

NEK1 has many jobs within motor neurones including helping keeping their shape and keeping the transport system open. Future research will tell us how we can use this new finding to target drugs to stop MND. Continue reading

Working together towards a world free from MND

During MND Awareness Month we are highlighting some of the research the MND Association funds in our ‘Project a Day’ series. Today, on global ALS/MND awareness day, we wanted to give you a look at the research into motor neurone disease taking place elsewhere.

Thousands of researchers across the globe are working towards a world free from MND. Rather than tell you each of their stories, we have gone to those that fund and facilitate this research, and asked them how their efforts bring us closer to figuring out the causes of MND, and finding treatments for this disease.

“I find huge inspiration in the knowledge that when I finish my work for the day, the MND researchers in Australia are just beginning theirs.” Prof Martin Turner, University of Oxford Continue reading

Collaborating across Europe to find a cure: ENCALS 2016

332 delegates, 135 posters, 41 talks, one goal: to cure ALS

The European Network for the Cure of Amyotrophic Lateral Sclerosis (ENCALS) was set up to find a cure for ALS/MND by working collaboratively across 35 research centres (universities and hospitals) throughout Europe.

The 14th meeting of ENCALS took place in Milan between 19-21 May and was attended by scientists and doctors from across Europe. Researchers from the USA and Canada were also invited to present at this meeting.

Presentations on day one of this year’s meeting looked at some of the techniques to help identify genetic changes (mutations) linked to MND, such as whole genome sequencing. This is a rapidly growing area of research, thanks to Project MinE  – a global effort to find MND causing genes.

Clinical research was the focus on day two, and discussed the latest imaging and biomarker research. This is an important area as it will offer new ways to help track the progression of MND, and help to speed up diagnosis of this disease. Continue reading

Re-evaluating clinical trial guidelines for MND

To mark International Clinical Trials Day (20 May) we reflect on the ALS Clinical Trials Guidelines workshop that took place in March. The MND Association co-sponsored this successful meeting, held at Airlie House in Virginia USA. Approximately 140 delegates from across the world attended, including 11 MND researchers and doctors from the UK.

Why was this meeting held?

The meeting was a key stage in the process to update (and improve) international guidelines for clinical trials in amyotrophic lateral sclerosis (ALS, the most common form of MND).

The first international ALS clinical trials guideline workshop took place in 1998. The guidelines were designed to improve the quality of clinical trials in ALS, and provide evidence based recommendations to those designing and carrying out all stages of clinical trials. Continue reading

Pesticides linked to increased risk of developing MND

The results from a study looking at the possible links between exposure to environmental toxins (found in pesticides) and motor neurone disease (MND) was published yesterday (9 May) in the journal JAMA Neurology.

A group of researchers from the University of Michigan, led by Dr Feng-Chiao Su and Dr Eva Feldman, have found that exposure to pesticides is associated with an increased risk of developing MND.

What did the study involve?

156 people with amyotrophic lateral sclerosis (ALS, a type of MND) and 129 healthy ‘control’ participants from Michigan, USA completed questionnaires on their occupation history, gave blood samples, or did both.

The questionnaire asked about their occupations over four windows of time; at any point during their life, in the last 10 years, in the last 10-30 years, and over 30 years ago. From their answers, the researchers worked out the likelihood of each participant’s exposure to pesticides.

The levels of 122 persistent environmental pollutants (including organochlorine pesticides or OCPs) were tested for in blood samples taken from participants.

Persistent environmental pollutants are those with a long half-life, meaning that they break down slowly. This meant that they could be tested for in the blood, even if exposure happened several years ago. However, the blood sample cannot tell us the source of the pollutants, such as if it was through work, at home or even from eating fruit and vegetables that had been sprayed with pesticides. Continue reading