It’s been over a month since the announcement by the FDA of their decision to licence edaravone / Radicava for people with MND in the USA. The speed of the FDA’s decision took the drug company MT Pharma and the MND research community by suprise. It is encouraging that edaravone has been licenced to treat MND after two decades of failed drug trials. Since the FDA announcement the effects of the drug and what it means for people with MND has been extensively discussed and some of the trial data has been published.
This blog is an update on what studies have been done on edaravone and the likelihood of people with MND noticing a beneficial effect if they were to receive it. Continue reading →
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 →
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 unexpected 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:
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. 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 →
A few months ago we wrote an article about the ALS Clinical Trials Workshop which took place in Virginia, USA. Since then the Guidelines Working Groups have been busy turning the large number of issues debated into a first draft of a new set of guidelines. This is open for comment from 1- 31 August.
Study design and biological and phenotypic heterogeneity
Therapeutic / Symptomatic interventions in clinical trials
Patient recruitment and retention
Different trial phases and beyond – (there are two sections on this)
Within each of these sections, there are many recommendations. The Clinical Trials Guidelines Investigators want to ensure that all interested people and stakeholders have an opportunity to provide input – whether you are a researcher, clinician or person with MND.
Previous research in humans and zebrafish has shown that before symptoms arise in MND, early changes occur in the interneurones. This type of nerve cell provide a link between the upper and lower motor neurones in the brain and spinal cord.
The job of one type of interneurone (called inhibitory interneurones) is to apply the brakes on motor neurones. They work just like brakes on a bike stop the wheels from moving.
The interneurones control when chemical signals/messages (or action potentials) can be passed along the nerve cell. In MND these brakes are less effective (so to use the bike analogy, the brakes might be rusty or not connected properly).
Interneurones are being studied in more detail in a project led by Dr Jonathan McDearmid (University of Leicester), in collaboration with Dr Tennore Ramesh and Prof Dame Pamela Shaw (Sheffield Institute for Translational Neuroscience) (our reference: 835-791). Continue reading →
During the early stages of MND it is proposed that motor neurones are more susceptible to an imbalance of oxygen within the cells, known as oxidative stress. Prof Dame Kay Davies, at the University of Oxford, has previously shown that increasing the levels of the gene Oxr1 can protect motor neurones from death caused by oxidative stress and delay MND in mice. You can read about this work here. Continue reading →
If you looked at the motor neurones of people with MND down the microscope you would see clumps of a protein called TDP-43. Researchers around the world are working to find why these clumps form and how they are linked to MND.
Dr Jemeen Sreedharan has been looking at the effects of TDP-43 in fruit flies. Initially he investigated how TDP-43 caused its effects, later moving on to find ways to reduce or prevent the damage. He spent the first two years of his MRC and MND Association-funded Fellowship (our reference: 943-795) working at the University of Massachusetts, Boston USA returning last autumn to perform the next stages of his research at the Babraham Institute near Cambridge, UK. Continue reading →
Developing disease models is important for furthering our understanding of MND and allows researchers to screen potential new drugs for a beneficial effect. Moving a promising ‘nearly drug’ from the lab to being tested in people is known as ‘translational research’.
Dr Richard Mead
Dr Richard Mead was awarded the Kenneth Snowman/MND Association Lectureship in Translational Neuroscience in May 2014. The Lectureship is part funded by the MND Association (our reference 983-797).
We have recently received a progress report from Dr Mead. Its clear that his background and experience in this area – including several years working in the pharmaceutical industry – has helped him to rapidly develop a portfolio of projects and collaborations with academic and industry partners. Continue reading →
Deposits of the protein TDP-43 are found within the motor neurones in the majority of cases of MND, and are considered a pathological hallmark of the disease. While we do not fully understand how these deposits are formed, previous research has shown that activation of a process called the Unfolded Protein Response (UPR) can cause TDP-43 protein to deposit in the motor neurones. Continue reading →