Motor Neuron Disease

The SITraN scientific research programmes are dedicated to developing effective disease-modifying therapies for neurodegenerative diseases of the human motor system.

The challenge

MND is a group of diseases with multiple causes, rather than a single disease with a single target. This is likely to have major implications for potential MND therapies. We suspect that, due to the varying underlying causes of the disease, there are subgroups of patients who will respond only to certain types of therapies. Therefore, we might not find the one therapy that suits all patients, but will rather have to provide a cocktail of drugs or tailor treatments according to the molecular subtype of MND.

In order to develop effective MND treatments we need to:

1. Understand the causes and disease mechanisms underlying all forms of MND and identify the right targets and cellular pathways on which potential drugs can work.

2. Generate and optimise experimental models that mirror human disease to evaluate new treatments.

3. Develop new treatments based on this knowledge and screen therapies in a hierarchical cascade, tackling these targets in several cell and animal models to prove effectiveness and optimise delivery into the central nervous system.

4. Develop tools for earlier pre-symptomatic diagnosis of familial MND in order to give neuroprotective therapies the best chance to work effectively.