ALS Research News
The latest research articles and news from ALS.net and ALS-TDI.org.
In The News
The ALS Association website's news section.
The Centre for Brain Research Auckland
The pre-eminent place in New Zealand for research into brain functioning, including MND. The Centre is linked to international research into MND. Professor Richard Faull,who leads the Centre, is the Medical Patron of MND NZ.
2018 EXPERT UPDATE
2018 Australasian MND Symposium
In March, members of our team attended the first Australasian MND Symposium. Our notes are summarised in the following PDFs:
Research Update— A broad update about developments in research towards a cure and understanding the causes of MND, including a proposed staging system for MND (similar to that used for cancer) and an important update to our understanding of riluzole
Drug Development— This update discusses the early 2018 drug development pipeline and our current understanding of the disease mechanisms in MND. Of interest to neurologists and those with a research interest in MND.
Clinical Trials— Key global and Australian clinical trials in 2018, the current issues in clinical trial design, and new biomarkers being developed for better clinical trial results. Of interest to neurologists and those with a research interest in MND.
Multidisciplinary Clinics— The life-extending importance of multidisciplinary teams in providing best practice care for people with MND. Of interest to all allied health professionals and people affected by MND.
Palliative Care in MND— This update discusses the importance of quality palliative care throughout the course of MND from all health professionals involved. Of interest to all health professionals and people affected by MND.
Clinical Management— Advice for allied health professionals about communicating about sensitive subjects, new respiratory research, and developments in assistive communication technology.
Biomedical research summary
“It’s a very exciting time to be involved in MND research. There’s a lot of hope and positivity.” – Prof Matthew Kiernan, Bushell Chair of Neurology, Sydney Medical School
“I’m much more optimistic than I was 10 years ago. Researchers are intensely interested in MND.” – Prof Kevin Talbot, Head of Clinical Neurology, Univeristy of Oxford UK
“The rate of progress in recent years is very exciting.” – Lucie Brujin, Chief Scientist, ALS Association
4 to 5 million people now alive today will die from MND.
MND is not one ‘disease’, it’s a spectrum of things converging on one system – the cortico motor-neuronal system. There is enormous variety in how MND affects people.
Because MND/ALS is not one disease, it will not be solved by one treatment.
Riluzole prolongs the final stage of MND. It may also prolong the first stage but this effect is unknown.
18 clinical trials have failed in the last decade. For clinical trials to be more successful in future, they need to select more homogeneous groups of people with similar genetic factors, environmental factors, progression rate and pattern. Better disease modelling and better understanding of disease mechanisms will help.
Diagnosis comes quite late in the progression of MND. People are enrolled in clinical trials well after their system begins dysfunctioning. Earlier diagnosis and studies of pre-symptomatic gene carriers are essential.
Developing MND is now thought to be a multistep process where genetic risk & environmental factors accumulate over time until a threshold of disease is reached. About 6 steps (triggers) are required for MND to develop. We have an incomplete understanding of these triggers and how they cascade.
MND is extremely complex but there is cause for optimism.
We now know of 25 risk genes for MND. At this rate of discovery, in four years we will have found 50. (Each of these risk factors increase your risk of MND by 5-10%.)
These genetic discoveries give us a better idea of the pathways that should be targeted by drugs, including common pathways between genetic and sporadic MND.
It’s much harder to identify environmental risk factors than to find genetic risks. Environmental risk factors that many (not all) studies have agreed on are: smoking, trauma (eg broken bone, surgery), diesel fumes, exercise, being a war veteran.
There has been an exponential increase in our knowledge of basic mechanisms of MND. Knowledge Is increasing at an extraordinary rate, especially in the last 2 or 3 years. Professor Robert Henderson, University of Queensland, simply explained a theory of the mechanisms of MND: “Whatever causes MND results in misfolded proteins, which triggers an immune response, which causes inflammation, which causes damage to functional cells. This cell stress leads to motor neuron death, which leads to motor function loss.”
Why Haven't We Found a Cure Yet?
We recommend watching this presentation by Professor Kevin Talbot, Head of Clinical Neurology at the University of Oxford, UK, which describes the complexity of MND and causes for optimism.
Clinical research summary
“MDT clinics are the biggest advance in the treatment of MND. Patients in MDTs live on average a year longer.” – Prof Jeremy Shefner, Barrow Neurological Institute, USA
Evidence suggests that patient survival and quality of life is improved by:
early intervention with non-invasive ventilation
maintenance of weight and nutritional status
the provision of coordinated care through multidisciplinary clinics (MDTs).
Multidisciplinary clinics (MDTs) are the biggest advance – patients in MDTs live on average a year longer.
There is increasing evidence that palliative care, integrated in a multidisciplinary approach to care, leads to improved symptoms and quality of life in people with MND and their families. These outcomes can only be achieved if palliative care knowledge and expertise is extended beyond the domain of palliative specialist services to include the full scope of health and community-based services.
There is a strong connection between meaningful occupational engagement and a person with MND’s self-perception as capable, engaged and having a sense of control and normalcy in their lives.
The next 5 to 15 years
“I’m really optimistic for the future for the next 5 to 15 years. It will lead to a cure.” – Prof Paul Talman, director, Australian MND Registry
Those with familial (SOD1- and C9ORF72-related) MND are likely to be treated with therapies targeting DNA – eg CRISPR, antisense oligonucleotide therapies (ASOs). Researchers are extremely optimistic about the use of ASOs to treat familial MND within 5 to 10 years. Clinical trials for SOD1 ASOs have begun and C9 ASO trials will begin this year. ASOs could also be useful for lowering ataxin 2 in sporadic ALS.
Sporadic MND will be redefined and separated into sub-types according to biomarkers (eg fast and slow progressors). This will allow those with sporadic MND to eventually be treated with personalised medicines, aka precision medicine. (Precision medicine is based on understanding the disease mechanisms, biomarkers, and individual responses to treatment.)
Researchers are urgently working to identify biomarkers (eg in blood or urine) that reflect the underlying biology of MND, to guide more targeted clinical trials and better measure their effectiveness.
Overall, there are more new therapeutic approaches to MND than ever before, increasing the likelihood that new therapies will become available that provide a meaningful impact on the lives of MND patients.
Several other drug development avenues are being explored and any one could have a big impact. Immunotherapy may have great promise (people with fast progressing MND have higher inflammation).
At the 2016 annual meeting of the International Alliance for ALS/MND Associations, Dr David Taylor of ALS Canada gave a comprehensive update about the current state of MND research.
Scientific Update - Dr David Tayor
2015 EXPERT UPDATE
The videos below were recorded at the MND New South Wales 'Ask the Experts' event in 2015, for people living with MND, their family and friends. They are long, but give a thorough and up-to-date education about what we currently know about MND. If you'd like to learn more about MND, these are worth watching.
