Motor neurone disease
Motor neurone disease (MND), sometimes referred to as amyotrophic lateral sclerosis (ALS) , is the name given to a group of progressive neurological conditions where specific nerve cells, or neurons, don’t work normally.
This leads to increasing physical disability, as muscles are unable to receive messages from the neurons to move or activate. Without these signals, muscle weakness, wasting and paralysis develop.
MND usually develops quickly once symptoms begin, with an average life expectancy of 2-3 years from diagnosis. It can affect anyone, at any age, but usually develops in people over the age of 40.
How The Florey is making a difference
There has been steady progress in identifying genetic factors that cause or predispose people to MND. This has resulted in a large number of genetically engineered laboratory models to test new treatment approaches for MND.
The Florey has an outstanding program of research in MND using patient-derived stem cell based and animal models. Our research using cutting edge techniques spans key areas in MND, including protein folding, energy metabolism, support cells, immune cells, gene and stem cell therapy approaches.
In Associate Professor Brad Turner’s laboratory, we have a strong focus on drug discovery, high throughput drug screening and translational medicine with the ultimate goal to develop effective treatments and a cure for MND.
Our research has also been instrumental in progressing a copper-binding drug, CuATSM, into a Phase 1 clinical trial in conjunction with Neuroscience Trials Australia.
- Accelerating disease in patient iPS cells using the living brain model of MND & PD
- Assessing the autophagy pathway in humanised astrocytes of motor neuron disease
- Defining unique molecular markers of upper motor neurons in MND
- Developing BBB-permeable ASO to target ataxin-2 and mitigate TDP-43 proteinopathies for amyotrophic lateral sclerosis therapy
- Developing SMN gene therapy for SMA and MND
There are a number of active and upcoming clinical trials for MND. For more information about MND and clinical trials can be found at MND Australia and FightMND.
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