Biomarkers in motor neuron disease - a longitudinal translational neuroimaging and CSF study

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Motor Neuron Disease (MND) is a devastating neurodegenerative condition affecting around 5000 people in the UK. Loss of motor neurons produces muscle wasting variably affecting the limbs, swallowing, and ultimately breathing. The average survival from symptom onset is 3-4 years. There is no specific test for MND and diagnosis relies mainly on clinical examination. Many patients wait more than a year from symptom onset to receive a firm diagnosis. Over 75 drugs have failed in clinical trials but it can take many months to establish lack of efficacy with certainty. This research will study a group of MND patients every six months using magnetic resonance imaging (MRI) of the brain in combination with molecular analysis of the surrounding cerebrospinal fluid (CSF). The Oxford Centre for Functional MRI Brain (FMRIB) has developed techniques highly sensitive to damage within nerve pathways in the brain, and there is local expertise to study the CSF for molecules associated with these changes. In combination it is expected this will provide markers of disease activity to improve the diagnostic process and the way clinical drug trials are monitored, so ineffective drugs can be more quickly discounted. New insights into disease mechanisms may provide novel drug targets.