Working toward a motor neuron disease clinical trial

A ‘model’ of MND has been created which mirrors the disease in humans. This incredible advancement is helping us learn more about the progression of MND (at a cellular level) and is used to test and monitor new drug therapies in a safe environment.

Even more encouraging is what we’ve learnt about a protein called TDP-43. We now know that in more than 90% of MND cases, changes to this protein play a vital role in motor neurone degeneration. In pre-clinical (non-human) trials, we have found that this protein can be controlled by a specially engineered peptide sequence (i.e. medication) which has the potential to stop MND in its tracks.

But here is the most exciting development… we are observing improvements in movement, behaviour and memory upon administering this medication.

This is innovative, ground-breaking research in action. We need your help to accelerate this research, which will in time enable clinical trials in people living with MND.