A drug used to treat liver disease is being trialled in people with Parkinson’s.
Professor Oliver Bandmann has been working for the last 10 years to identify potential drugs that might target mitochondrial dysfunction in Parkinson’s – thought to be a major contributory factor. The team in Sheffield led by Professor Bandmann, tested 2,000 drugs in genetically stratified patient tissue – tissue from people living with Parkinson’s (PwP) with the PARKIN gene mutation. They tested for mechanisms leading to cell death and screened drugs that affect mitochondrial dysfunction. They found the drug, ursodeoxycholic acid (UDCA), a safe drug that had been used to treat liver disease for 30 years, crossed the blood brain barrier and then tested this in other genetic models known to have mitochondrial dysfunction.
In 2015 The Cure Parkinson’s Trust (CPT) wrote a dossier on UDCA which was highly prioritised by the International Linked Clinical Trials (LCT) committee that year, giving support to Professor Bandmann’s work. In 2018, CPT was delighted to award a grant to Professor Bandmann and his team.
“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.” – Helen Matthews, Deputy CEO, The Cure Parkinson’s Trust.
This 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. The effectiveness of the drug will be assessed with two novel approaches:
By using MRI-Spectroscopy (31P-MRS) researchers will be able to quantify the function of the mitochondria in the patients’ brain tissue to examine whether the drug normalises function. Also, specially designed bio-sensors will be used by triallists 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 measurement of motor impairment throughout the trial may also tell researchers whether UDCA might have the potential to slow down the progression of Parkinson’s.
“Mitochondria are the power stations of each cell in our body, and it is critical to our general well-being to keep mitochondria healthy. In Parkinson’s, research has suggested that some of the mitochondria are not functioning correctly, which may be leaving cells in a more vulnerable state. UDCA has been shown to correct not only this mitochondrial dysfunction but also provide protection to distressed cells. And it is for these reasons that the Cure Parkinson’s Trust is very excited to be supporting the Sheffield clinical trial evaluating UDCA in Parkinson’s.”
Article written by Dr Simon Stott, Deputy Director of Research, The Cure Parkinson’s Trust