Sheffield Institute for
Translational Neuroscience


Liver drug trialled in Parkinson’s patients as novel treatment approach

A drug which has been used to treat liver disease for over 30 years is being trialled in patients with Parkinson’s Disease to determine its safety and tolerability in Parkinson’s, but also to assess its potential to slow down disease progression for the first time.

The ground-breaking study, led by researchers at the University of Sheffield in partnership with Sheffield Teaching Hospitals NHS Foundation Trust, will assess the safety and tolerability of the liver drug ursodeoxycholic acid (UDCA) in Parkinson’s patients – a drug which is hoped could potentially be repositioned to help slow down the progression of the disease.

Parkinson’s Disease currently affects around 145,000 people in the UK. The progressive neurological condition severely affects a patient’s quality of life and symptoms include problems with mobility such as walking, coordination or tremor, but can also result in memory loss, low mood or abnormal bowel function.

The symptoms of Parkinson’s are mainly due to the loss of dopamine containing nerve cells in the area of the brain which controls movement. An important reason why these cells die in the brain of patients with Parkinsons is due to a malfunction of the cell’s batteries – known as mitochondria.

The trial, led by Oliver Bandmann, Professor of Movement Disorders Neurology at the Sheffield Insitute for Translational Neuroscience (SITraN) and also an Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, was made possible due to the NIHR Sheffield Biomedical Research Centre (BRC) for Neurological Disorders.

Professor Bandmann and his team previously identified UDCA as the most promising drug to rescue mitochondrial function in Parkinson’s in a drug screen where 2,000 drugs were assessed for their rescue effect on mitochondrial function directly in the tissue of patients with Parkinson’s.

“After nearly a decade of research we are extremely pleased to launch the first clinical trial of UDCA in Parkinson’s patients to see if the drug is safe and tolerated,” said Professor Bandmann.

“This is a pilot trial, which if successful, will lead to a bigger study to firmly establish the effectiveness of the treatment to slow down progression of Parkinson’s. Currently, Parkinson’s is relentlessly progressive but patients tend to respond very well to symptomatic medication in the early stages of the disease.

“A drug which will slow down the progression of the disease – even after the first few years of diagnosis – would help people to have an improved quality of life for longer.”

The trial, known as the UP Study (UDCA in Parkinson’s) will be conducted at two centres in the UK, Sheffield and London, in collaboration with Professor Tom Foltynie at University College London Hospitals. Working in collaboration with researchers from the University of Sheffield’s Institute for Insilico Medicine (Insigneo), the NIHR Sheffield Clinical Research Facility at Sheffield Teaching Hospitals and the NIHR Sheffield Biomedical Research Centre, the effectiveness of the drug will be assessed with two novel approaches:

  •  By using 31P MRI-Spectroscopy (31P-MRS), researchers will be able to quantify the function of the mitochondria to examine whether the drug successfully normalises the function of the brain tissue affected in Parkinson’s. Patients experience 31P-MRS like a normal MRI.
  • Specially designed sensors will also be used to measure the effect of UDCA on a patient’s motor impairment. The bio-sensors will be worn by patients at the beginning and end of the trial, giving more effective results than using a clinical scale which may not be objective. Repeat sensor-based objective measurements of motor impairment throughout the trial may also tell researchers whether UDCA might have the potential to slow down progression of Parkinson’s.

The clinical trial is supported by the JP Moulton Charitable Foundation, The Cure Parkinson’s Trust and the pharmaceutical company PRO.MED.CS.

Helen Matthews, Deputy Chief Executive Officer of The Cure Parkinson’s Trust said: “Through its International Linked Clinical Trials Programme, The Cure Parkinson’s Trust has been working towards bringing UDCA into clinical trials - and this trial is the first step in understanding the drug’s potential to slow Parkinson’s progression. We are delighted to be supporting Professor Bandmann’s important work.”