The first few patients have now been recruited to our study to test the safety and tolerability of ursodeoxycholic acid (UDCA) in Parkinson’s disease led by Professor of Movement disorders, Prof Oliver Bandmann. The ‘UP Study’ (UDCA in Parkinson’s) is a flagship example of pulling through the preclinical work carried out at the Sheffield Institute for Translational Neuroscience (SITraN) into clinical trials through the Sheffield Biomedical Research Centre. SITraN offers a state-of-the-art drug discovery suite where we are able to screen libraries of drug and drug-like compounds in our validated models of neurodegenerative disease for their disease modifying effect. Professor Bandmann working with Dr Heather Mortiboys carried out the first ever drug screen in Parkinson’s disease patient cells with over 2000 compounds and found that UDCA which is already used in liver disease and other disorders, and so known to be safe, rescued mitochondrial dysfunction. The drug screening facilities in SITraN have attracted interest and investment from industry partners and are now set to expand opening up even more pipelines of therapeutic translation.
With external funding and support from the core Sheffield BRC team, including a new Clinical Trial Manager and Data Coordinator last year, we have been able to translate the preclinical work on UDCA into the patient co-designed UP study that is being delivered by the NIHR Sheffield Clinical Research Facility at Sheffield Teaching Hospitals. Patients on the trial will be monitored using several novel techniques including using wearable sensors to assess daily activities rather than just rely on constrained movement assessments during a clinic visit, and an advanced medical imaging technique to measure energy levels in the brain.