Energising the bench to bedside approach to conquer neurodegenerative disease

Play Video





1. Summary of your research (150 words max)

We live in a time of innovation and discovery, yet a frontier that remains is our ability to treat diseases of the brain. Motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), is a relentless neurological disorder that robs a person of the neurons that control voluntary movement. The loss of these neurons results in progressive paralysis and death, and we are yet to develop a cure.
Our capacity to treat disorders such as MND is limited by our knowledge of the processes that lead to that disease. By adopting a Bedside to Bench to Bedside approach to research, I have increased our capacity to generate a greater understanding of the impact that altered energy metabolism has on the progression of MND. By engaging with MND patients, clinical experts, and basic researchers, I have adopted an integrated research approach to achieve a world free of MND.

2. Describe your approach and broader findings (500 words max)

Motor neurone disease (MND) is a devastating neurological disorder that strips people of their capacity to interact with the world. MND locks people in their bodies until they die from respiratory failure or associated complications, usually within 27 months of diagnosis. Clearly, there is an urgent need to develop strategies to treat this disease.
The Bedside to Bench to Bedside approach
To accelerate our capacity to find a cure or treatment for MND, we must develop innovative and integrative approaches that evolve from previous successes. Research strategies that are based on bench to bedside translation have provided significant breakthroughs to treat disease. In MND, however, this has not been the case. Indeed, the only compound that provides limited benefits in MND was first developed at the bedside, tested at the bench, and then brought back to the bedside as a treatment.
In 2014 I initiated a Bedside to Bench to Bedside initiative, forming a multidisciplinary research program that integrates neurologists, clinical researchers, stem cell researchers, neuroscientists, physiologists, and biomedical researchers across multiple research centres. The vision was to develop comprehensive and innovative screening platforms that would facilitate the identification of novel mechanisms that could be targeted to improve the quality of life and to extend the survival of people living with MND. Integral to this vision was the implementation of a patient-directed research agenda. The Bedside to Bench to Bedside approach allows us to focus on the needs of each individual patient, and it will enhance our capacity to develop tailored treatments for MND.
Emerging discoveries
Our studies in patients with MND have allowed us to investigate whether an inability to maintain metabolic homeostasis may impact a patient’s quality of life, while accelerating the rate of disease progression. Our results confirm that the metabolic needs of some MND patients are greatly increased, and that changes in whole-body energy metabolism may worsen their disease symptoms. In translating this knowledge from the bedside to the bench, we have identified defects in the processes that regulate energy metabolism in the brain and muscle of preclinical models of MND. I am now focussed on investigating the pathways that control energy metabolism in neurons and muscle that have been derived from MND patient induced pluripotent stem cells. As part of these studies I will test compounds that target metabolic flux in these neurons and muscle, with the specific goal of improving energy metabolism to sustain their function and survival. Ultimately, the translation of positive research findings in human-derived cells from the bench back to the bedside will expedite clinical trials for MND.

3. What is the wider contribution or impact to your scientific field(s)? (300 words max)

An increase in the incidence of age-associated neurodegenerative diseases is placing an increasing burden on global health expenditure. Given the complexity of the brain, current treatments for neurodegenerative diseases are largely limited by the multifactorial nature of these diseases and the difficulties associated with identifying viable treatment targets. By adopting the Bedside to Bench to Bedside approach, my goal is to deliver the discoveries of today that will lead to the personalised treatments of tomorrow.
More broadly, my research into the impact of altered energy metabolism on the onset and progression of MND has implications for all neurodegenerative disorders. There are an increasing number of studies that highlight the co-existence of neurodegenerative disorders (including Parkinson’s disease, Huntington’s disease, Alzheimer’s disease, and dementia) and altered metabolic homeostasis. Thus, it is plausible that defects in the processes that regulate metabolic flux may underpin or exacerbate neurological symptoms. By defining how changes in metabolic homeostasis contribute to the aetiology of neurodegenerative disease, we will identify the relationships between energy balance and neurodegeneration. This knowledge will allow us to determine whether accommodating for dysregulated metabolic homeostasis provides a promising strategy for the management of all major neurodegenerative diseases. Ultimately, this will support a healthier ageing population, which will in turn mitigate the pressures associated with increased health spending on age-associated brain diseases.

4. Potential ideas you would like to explore to take this research further? (300 words max)

Clinical trials aim to validate treatments that have the potential to improve disease outcomes. Due to the heterogeneous nature of MND, the success of clinical trials in this area has been slowed, and it is increasingly evident that an umbrella approach to treating this disease may not be an option. If we are to embrace a future of personalised medicine in MND, we must first develop a means to allow the successful completion of personalised clinical trials.
My future research will explore approaches that will assist us in identifying patients who would benefit most from specific clinical treatments, and to conduct clinical trials aimed at addressing individual needs. To achieve this goal I must establish our current patient-screening platform at multiple assessment centres across the globe. I am currently engaging with research teams in the Netherlands (where we are conducting a sister study), and hope to expand our studies to include clinical centres across Australia, Europe and America. Once our preclinical studies have identified suitable drug candidates, it is anticipated that we will have the means to identify individuals who will benefit from these treatments, and thus the capacity to direct trials to meet the needs of individuals with similar disease traits.

5. Please share a link for researchers to access your article, data-set or thesis

More information about this work can be found at https://www.uq.edu.au/sbms/staff/shyuan-ngo

Data on the patient screening platform is currently being prepared for publication and assessment for IP protection.

Voters Map


Wayne Patterson
over 4 years ago

Superstar researcher with style

uqpeterc uqpeterc
over 4 years ago

A dedicated scientist needing funding support.

Nick Merry
over 4 years ago

A wonderful and passionate research scientist doing everything she can to rid the world of MND

Boris Prosper
over 4 years ago

Futur supervisor?

Karin Borges
over 4 years ago

Passionate researcher in lab and clinic.

Ernst Wolvetang
over 4 years ago

passinate about MND and finding a cure

Shyuan Ngo
over 4 years ago

As the Peer Prize for Women in Science winds down and comes to a close, I want to take a moment to reflect on my experience and to express my gratitude to all of the amazing and supportive researchers who have backed me in this campaign.
Through this campaign, I have been given a unique opportunity to reach out to old and new friends who I have met along my career path in Science. Not only have you taken an interest in what I am doing, you have also taken the time to stand in my corner (and battle with the registration and verification process!!) to support my research goals and to support Women in Science.
Everyday, working with people living with MND/ALS, I always hope for a brighter future. As cliche as it may sound, a World Free of MND is truly what I am working towards. The MND/ALS community is a very close-knit and tight one...full of fighters who have a "Never Give Up" attitude. They follow my campaigns and to allow them to see firsthand, the support that you offer me is really something that I can not thank you enough for.
I was hoping to win this prize so that I could buy some equipment for our lab to support ongoing research, but there will always be another opportunity. I will find a way.
Research will carry on and there will always be something to look forward to in the way of positive outcomes. For all of you who supported me, and who will always support me through the ups and downs and all the campaigns in Science, I will see you along the way! Thank you again and always.

Mirana Ramialison
over 4 years ago

Such a privilege to have learned about your research and the amazing work you are doing to fight MND.


Shyuan was the winner of the 2016 Queensland Women in STEM prize. She is the Scott Sullivan Motor Neurone Disease Research Fellow at the Royal Brisbane & Women's Hospital and the University of Qu...