Yesterday, we were delighted to unveil a new research collaboration that we believe will greatly improve the quality and pace of MND research – not only in understanding the cellular processes that cause motor neurons to degenerate, but also in helping with drug discovery and development.
The Amyotrophic Lateral Sclerosis Reproducible Antibody Platform (ALS RAP) isn’t anything to do with the musical genre. It isn’t really a research project either. Instead, it’s focusing on the importance of providing the scientific community with ‘gold standard’ tools for their research.
Long before the latest wave of cellular and molecular biology advances started to give us new information on what was going on at the cellular level in MND, some doctors had observed that if the disease started in one particular part of the body, it would be neighbouring parts that became affected next. This suggested that the disease usually starts in a single part of the brain or spinal cord before spreading further, like ripples in a pond.
How this happens is not well understood. It is likely that there are a number of processes going on, but they can broadly be divided into two theories. One of these is that damaged proteins can leak out of sick neurons and ‘infect’ their neighbours – a subject we have discussed at previous international Symposia.Read More »
I usually travel to London two to three times a month for meetings and lab visits. If I’ve got any length of spare time, I head for what I call my ‘London office’ – aka the British Library. It’s close to Euston station, it’s free (!) it has a nice café for informal meetings and it has copies of all the latest textbooks and major research journals.
The way in which a cell turns its genetic instructions into the protein ‘building blocks’ it needs to function and survive is sometimes compared to a library.Read More »
Warmest congratulation to Dr Marka van Blitterswijk of the Mayo Clinic in Jacksonville, Florida, winner of this year’s Paulo Gontijo Prize in Medicine.
The Award is presented to an outstanding young investigator working on ALS/MND, with judging based on the significance of a recent scientific paper published by each applicant, plus an evaluation of the relevance and impact of their career to date. I have had the pleasure of serving on the Judging Committee, Chaired by Prof Mamede de Carvahlo, since 2011 and each year the competition gets tougher and tougher. It is so heartening to see the increasing number of excellent young scientists dedicating their careers to the fight.Read More »
Whilst the vast majority of MND research happens in the lab, there is also an increasing amount of research activity looking into how best to manage the various symptoms of the disease. There are a lot of unanswered questions as to ‘What Works and What Doesn’t?’ and without a decent level of evidence, it is increasingly difficult in these cash-strapped days to get new or even existing types of therapy adopted into mainstream statutory care.
One such ‘Cinderella’ subject is psychological support for people with MND. It’s hardly surprising that studies show almost half of people diagnosed with MND experience depression and almost a third experience anxiety, yet there is very little guidance on how to best address these symptoms. As a result, formal psychological support is not routinely offered and where it is, the particular approach taken is based on best judgement rather than robust evidence.Read More »
At last year’s Airlie House workshop to develop new ALS/MND Clinical Trial Guidelines the focus was, of course, on MND, but there was also important input and learning from outside the field.
One of the most fascinating presentations was from an oncologist who was explaining how detailed genetic analysis of tumours was leading to an understanding of why some experimental cancer drugs appeared to only work in a small subgroup of patients. The take home message from the cancer field was that there should be more effort made in future MND trials to identify and analyse smaller subgroups of patients, in case a potentially positive effect might be missed.
A new research paper, published in the journal Neurology, raises some intriguing findings from the trials of the drug lithium that were carried out several years ago. Lithium generated a lot of excitement when researchers in Italy reported a positive effect of the drug in the SOD1 mouse model of MND. Almost as an afterthought, their research paper mentioned that they had tested the drug in a small short-term trial in patients and it appeared to have some effect.Read More »
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.
During Awareness month in June we reported on the work of Dr James Bashford at King’s College London, exploring new ways of measuring muscle fasciculations in people with MND. The results from the one year pilot study have shown a lot of promise, which has led to Dr Bashford recently being awarded a Clinical Research Training Fellowship.
A common symptom of MND is the ‘rippling’ of muscle under the skin, these are known as muscle fasciculations.Read More »
The idea that drugs licensed for one disease may have some use in another completely different disease is not new, but it has gained much more attention in recent years. Researchers are developing a new understanding of disease processes, leading to new ‘drug repurposing’ opportunities, with the additional potential to reduce the time and cost of drug development.
Significant advances in genetics and molecular biology in recent years have greatly increased our understanding of the pivotal, carefully balanced cellular processes that usually keep motor neurons healthy but, when disrupted, can cause a cascade of degeneration leading ultimately to their death.Read More »
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.
Thank you very much for your help.
For more information, please see a copy of their press release below:Read More »