Recipient:  Dr Frederik Steyn

Tipping the Scales on MND: Preclinical testing of a compound with multiple actions to slow disease progression in MND

Existing treatment options for Motor Neurone Disease (MND) modestly extend survival, and there is an urgent need to identify treatments to improve clinical and prognostic outcomes for all patients with MND. In 2019 we were honoured by the award of The Fat Rabbit Grant. This grant is supporting the funding of critical staff needed to test a new compound aimed at treating MND.

Our approach to finding a treatment for MND is based on our experience gained from working alongside people with MND. MND is a complex disease, and progression is highly variable between people living with MND. While research is still looking for the cause for MND, we have identified a number of factors known to impact disease progression. Our unique focus has been to find a compound that will target many of these factors, with the hope that this will slow disease progression and improve quality of life for a wide range of patients living with MND. With support from the Fat Rabbit Grant, and in collaboration with The University of Queensland, we are now working hard to complete the first set of experiments aimed at testing our new approach to treating MND.

Using a compound that targets different factors known to accelerate disease progression (including derangements in metabolism, loss of appetite resulting in weight loss, loss of the endogenous production of neuroprotective and anabolic factors, and neuroinflammation), we have made considerable progress in demonstrating that we can slow the progression of disease in a mouse model of MND. Our compound slowed weight loss and the loss of fat and muscle mass, and delayed the progression of disease in MND mice. While initial experiments are still under way (and are not yet published), it is clear that our approach has potential to benefit those with MND. As a next priority, we will initiate testing of our drug using a different treatment approach to maximise drug exposure. In doing so, we expect to see a further slowing of disease progression, and an extension of life. These more ambitious experiments will be completed in two different mouse models of MND – providing evidence needed to transition our drug towards clinical testing.

These are only the first series of studies to validate the use of our compounds to treat MND. We have also developed a research strategy to test our compound in cell models of MND that more closely resemble that of the patient. We have submitted this research strategy to two independent organisations with the hope that they will fund these critical studies. The fat rabbit grant supported our collection of data needed to submit these funding requests. If funded, we will generate convincing data that will hopefully support the funding and conduct of studies in patients within the next 5 years.