150,000 Australians are currently living with Parkinson’s disease, with a further 38 diagnosed everyday. It’s the fastest growing neurological disorder in the world, and there is still no cure. In our ‘Solving the Parkinson’s Puzzle’ series, we are speaking to leading Australian researchers to find out how their work is helping to slow, stop and ultimately cure Parkinson’s disease.

In this conversation with Professor Carolyn Sue AM, we hear about her Nix Gene Therapy research which aims to restore energy levels to the brain cells affected in patients with Parkinson’s disease. Professor Sue and her research team have identified an alternative way of recycling and recharging the batteries in the brain cells, so that the brain cell itself can function for longer and better.

Carolyn, can you tell us about your role and research focus?

I’m a neurologist and clinician scientist, and I work at Neuroscience Research Australia (NeuRA) and Prince of Wales Hospital in Sydney. In the research laboratory, and in the clinic, what we’re trying to do is restore energy levels to the brain cells that are affected in patients with Parkinson’s disease.

How is your work helping to solve the Parkinson’s puzzle?

So most patients who have Parkinsons’ disease are able to have their symptoms controlled with medications and various different treatments. But what we’re trying to do is slow the process of the disease progression down.

How would you explain your research to a five-year old?

Energy is vital for the brain cells to function normally. When you use energy from the brain cells, then it’s all used up, it’s consumed, and it runs out just like a battery would run out on a toy, or a mobile phone, or a computer. And so what we’re trying to do is recharge that battery. There are normal mechanisms in the brain cells that that can do this, and we’ve found an alternative way that we can recharge those batteries. So what we do is we try and give the cell instructions as to how to recycle their batteries in a more efficient and effective way so that you can have endless  supplies of energy, so the brain cells can continue to work. And that’s what Nix Gene Therapy is all about. We are actually trying to get this cell to utilise this alternative way of recycling and recharging the batteries in the cell, so that the brain cell itself can function for longer and better.

What is the next step of your research project?

So the next step in our research is to build the right set of instructions to help the cell be able to recycle its energy more efficiently. What we’re trying to do is build a treatment which will be able to be supplied to the cells, which will instruct the cells how to recycle the batteries in the cell (they’re called mitochondria). The new batteries will be able to recharge the nerve cells that are affected in Parkinson’s disease. So we need to build that set of instructions and provide them to the cells so they can work efficiently and safely. We’re doing that in both the dish and also in models of Parkinson’s disease, so that we can develop the most effective and safest form of treatment, to bring that to human clinical trials.

How will your research help someone living with Parkinson’s in 10 years time?

We hope that in 10 years’ time, patients will be able to have a treatment that will stop their disease progression. What that means, is that patients will be able to have treatments that restore their motor function, and then live a life not having to worry about the disease getting worse. And in a stage of their disease when the treatments will always work.

How has the funding from Shake It Up helped to make your research a reality?

The funding from Shake It Up has been transformative. We’re able to progress our experiments quickly and rapidly so that we can bring this treatment to in-human clinical trials as fast as possible. So it’s been a critical injection of activity, and allowing the experiments to progress quickly so that we can help patients with Parkinson’s disease as quickly as possible.