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.Read More »
Bar a few bacteria usually found hitching a ride on our dental plaque and digestive system, every living cell in the human body needs oxygen. Some cells need more oxygen that others, dependent on much energy they need to produce to function. Neurones are particularly active cells (the brain uses a fifth of all the oxygen consumed by the human body) and motor neurons are amongst the most energy hungry of all.
Unfortunately, the process of producing cellular energy isn’t 100% efficient: a small but constant amount of waste products called free radicals (yep, those things that the beauty product industry bangs on about) can build up in the cells. If not kept in check, they can start to wreak havoc within the cell.
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.Read More »
The MND Association is backing a new clinical trial in MND, known as MIROCALS. This will be a joint clinical trial between France and the UK that will aim to dampen the overactive immune system by increasing the amount of interleukin-2.
It is important to stress that planning for this MND clinical trial has only just started and the next step is to lay the essential groundwork and perform some short-term pilot studies. The main trial is likely to begin recruiting participants in autumn 2016.
“On the eighth day of Christmas MND research gives to you… EIGHT members of the Research Development team”
We would like to wish all our blog readers Happy New Year! Looking forward, like many others, we have made our New Year’s resolutions of what we’d like to achieve in 2015:
Brian Dickie (Director of Research Development): “I’m looking forward to developing closer relationships with other funding agencies, to look at research opportunities that we may not be able to do on our own. We expect researchers around the world to work together, so their ‘joined up thinking’ should be supported by our ‘joined up funding’.” Read More »
The 25th International Symposium on ALS/MND began today in Brussels, Belgium. More than 900 delegates joined the opening session to hear Dr Alfred Sandrock from Biogen Idec’s opening talk.
The only proven drug for MND is riluzole. This is the only treatment to have passed all stages of clinical trial testing, showing it to be both safe and beneficial in MND. New treatments are desperately needed, but what is needed to advance a treatment that has shown promise in animals to humans? Read More »
A question was submitted to the Association’s AGM last weekend, which could only be answered in brief at the time, due to the number of issues raised, some of which are of a technical nature. Below is a more detailed response from the Association’s Director of Research (in bold italics) to each point raised.
The day finally arrived on 11 April 2014 for our biannual Biomedical Research Advisory Panel (BRAP) Meeting. This important date in our research calendar is when grant funding decisions are discussed before being put forward to our Board of Trustees for approval.
But before we get to the meeting, there is a lot of preparation that is needed. As you are aware from previous blog posts, applications go through various stages of review, including summary review, invites for full applications and external review. Before the meeting itself there is yet another stage of review for the applications, which is known as internal review. This might seem a bit ‘admin-heavy’, but since we are only able to fund a quarter of such a wide variety of proposals, ranging from cell-based studies to clinical research, we need to be confident that we’re funding the ‘best of the best’. With so many new ideas, ‘separating the wheat from the chaff’ can be a difficult and time-consuming process!
On 3 March 2014, researchers based at the University of Edinburgh published a research paper that extends our understanding of the childhood disease – Spinal Muscular Atrophy (SMA).
Although this is not MND so-to-speak, the disease does affect the motor neurones. Plus, the results were so interesting; I couldn’t resist writing a blog post about them.
Floppy baby syndrome
SMA is a childhood disease of the motor neurones and is sometimes known as ‘floppy baby syndrome’. It affects 1 in 6,000 births making it more common than MND, which affects approximately 1 in 100,000 people.
The exact course, duration and rate of progression of MND often varies greatly from person to person; even when there is a known family history of the disease caused by a specific MND-causing gene (eg SOD1).
This same variability also occurs in mice. Researchers, funded by the MND Association, took two mice with the same SOD1 gene mutation from two different families (strains). By using these two mice the researchers identified a number of key changes in motor neurones that differ between fast and slow progressing forms of the disease.
Two mice… One gene
Developing new disease models enables us to both understand the causes of MND and test potential new therapies.
Mice are commonly used in MND research and for the past 10 years or more, the SOD1 mouse model has been one of the most important research tools for scientists working in the field, particularly with testing potential new therapies.
Research published in September 2013 was carried out in a joint collaboration between Dr Caterina Bendotti (Mario Negri Institute for Pharmacological Research, Milan Italy) and Prof Pam Shaw (University of Sheffield, UK).
A toxin known as β-N-methylamino-l-alanine (BMAA), which is found in blue-green algae, has been shown to cause proteins inside cells to clump together and cause cell death.
This finding suggests that BMAA may be a cause of neurodegenerative diseases like Alzheimer’s and MND and could lead to the development of new treatments.
What is BMAA?
BMAA is a non-protein amino acid. This means, that unlike the 20 amino acids that our bodies use to make proteins, it does not make a human protein.
BMAA is found in a type of bacteria called Cyanobacteria (more commonly known as blue-green algae), which are usually found in waterways as well as damp soil and on the roots of cycad plants.
Blue-green algae can occasionally cause algal blooms. This is when there is a rapid growth of organisms due to high levels of nutrients in the water. The resulting bloom can sometimes become so large that it can be toxic to wildlife.