Scott Sullivan MND Research Fellowship – Update April 2017 – MND and Me Foundation

Scott Sullivan MND Research Fellowship – Update April 2017

Recipient:  Dr Shyuan Ngo

As the Scott Sullivan MND Research Fellow, Dr Shyuan Ngo has developed an independent translational research program. By combining research in the clinic and the lab, her research aims to understand how changes in the way the body and cells make and use energy can affect how motor neurone disease (MND) progresses.

Where are we up to? What have we found?

In my first two years as the Scott Sullivan MND Research Fellow, I have driven collaboration between the RBWH and UQ, and national and international MND clinicians and scientists. These collaborations are playing an important role in driving the development of new research ideas, and in speeding up the progress of our research. For example, at UQ, collaborations with Prof Naomi Wray are helping us to better understand the genetics behind MND, while collaborations with Prof Ernst Wolvetang are allowing us to generate human stem cell derived neurons for studying MND.

My clinical research aims to study the relationship between energy balance, dietary intake, gut function, physical activity and disease progression. We have found that ~50% of our MND patients have higher energy needs when compared to age- and sex-matched controls, and that this increase in energy need is associated with faster disease progression.

We have also found that increased energy need in MND may be due to changes in how efficiently skeletal muscle can use energy. We are now studying MND patient muscle biopsies and MND patient stem cell derived neurons to understand if changes in how these cells use and make energy occur in MND.

Importantly, by studying muscle and neurons from MND patients, we are moving towards precision medicine for MND. To support this, we aim to repurpose compounds that are used for other human conditions, so that we can overcome the lengthy drug development process.

My basic research forms the foundation upon which we hope to identify pathways that can be targetted for drug repurposing in MND. Our data in mouse models of MND show that there is a change in the way that sugar and fat is used as energy throughout disease. By repurposing a drug that has previously been used for cancer, we have been able to improve the ability for mice to use sugar as energy, and have been able to delay the onset of muscle weakness. We are now testing other compounds to determine whether or not they can delay muscle weakness and extend survival in our MND mice.

We look forward to keeping you informed as we progress on this journey.