Research Resources

New Zealand MND Research Network
This network connects MND researchers in New Zealand with each other, and shares current research with the public.

ALS News Today
A daily digital news journal that reports the latest science and research news about MND/ALS.

MND Research Blog
Research updates are regularly posted on this blog, run the the UK MND Association.

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.

There are many clinical trials investigating various treatments for MND across the world.

The largest clinical trials database is clinicaltrials.gov which lists all research studies in over 200 countries. You can find studies on MND/ALS here.

The ALS Therapy Development Institute also lists clinical trials and those enrolling worldwide.

Please be aware however that many international clinical trials won’t accept overseas self-funded participants.

The MND Association has some excellent information on clinical trials here.

The International Alliance of ALS/MND Associations’ Scientific Advisory Council

The role of the International Alliance of ALS/MND Associations’ Scientific Advisory Council (SAC) is to be a centralized resource to review and provide global perspective on scientific and biomedical announcements, information and opportunities relating to ALS/MND. For more information on the SAC click here. 

The SAC have provided briefing notes on a number of current clinical trials and treatments. Links to these notes are listed below.

MND New Zealand would like to thank the Board of Directors of the International Alliance of ALS MND Associations and the Scientific Advisory Council for sharing these briefing notes.

June 2020 - updates

Clinical trials:

• Biogen - SOD1 Antisense Oligonucleotide
• Cytokinetics - Reldasemtiv
• Brainstorm - Nurown
• Collaborative Medical Development - CuATSM

Treatments:

• Right to Try legislation
• Unproven (Off-label) treatments

August 2019

Clinical trials:

• Biogen – SOD1 Antisense Oligonucleotide
• Cytokinetics -  Reldasemtiv
• BrainStorm -  NurOwn

April 2019:
MND New Zealand proudly helps fund vital research at the Centre for Brain Research at the University of Auckland and supports the latest genetics study. Dr. Emma Scotter (Centre for Brain Research, University of Auckland), Dr. Richard Roxburgh (Neurogenetics Clinic, Auckland City Hospital), and collaborators are now recruiting for a study of the genetics of Motor Neurone Disease in New Zealand. Dr. Scotter says, “We invite people with MND (familial or sporadic, total 300 people) and people without MND or a family history of MND (total 30 people) to participate.”

The purpose of the study is to understand the genetic causes of MND in New Zealanders. In addition, the study aims to test how certain MND gene mutations affect human cells.

More information is available on our science news blog.

To find out more or participate contact Dr. Emma Scotter: emma.scotter@auckland.ac.nz 

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).

Click here to download this two-page summary (PDF).

2017 ALS Canada Virtual Research Forum

There are some very interesting presentations online from the 2017 ALS Canada Virtual Research Forum. Click here to see the presentations available for on-demand viewing.

2017 International MND/ALS Symposium

Video presentations from the International MND/ALS Symposium, 7 December 2017, are now available to watch online.

Scientific Update, Dr Jonathan Glass

Our other picks for most interesting presentations are: 

• Strategic Planning for a Future without ALS/MND, Tammy Moore
• The Impact of the Edinburgh Cognitive and Behavioral ALS Screen (ECAS) Master Classes, Faith Hodgins 
• Cannabis and its Usage in Symptom Management of ALS/MND, Leslie Ryan (it is interesting to watch this in conjunction with reading the December 2017 report by Kerry Walker for MS NZ and MND NZ)
• The “Model of Doing” for People with ALS/MND, Helen Carey 
• Making Your Home a Smart Home, Sara Feldman (NB Amazon Echo goes on sale in NZ this year)

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

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.

About MND - Prof Matthew Kiernan
PART A
https://youtu.be/yupX2sLXTvc

PART B
https://youtu.be/6-qJOLBaqMo

What’s new in MND Research? Dr Justin Yerbury
PART A
https://youtu.be/3XhTaIDqTLo

PART B
https://youtu.be/06Qdm13RE2I

Hope, Hype and Reality - Prof Megan Munsie
https://youtu.be/6I6lfDCrGIg