Ursodiol: Parkinson’s Treatment Study Supports Fast-tracking

Ursodiol, a drug used to treat liver disease, could turn out to be an effective treatment for slowing down the progression of Parkinson’s disease, new research suggests. The research, led by academics from the Sheffield Institute of Translational Neuroscience (SITraN), along with scientists from the University of York, supports the fast-tracking of the drug ursodeoxycholic acid (UDCA), known in the U.S. as ursodiol, for a clinical trial in human Parkinson’s patients.

UDCA is already approved for use in human liver disease. Explained Dr Heather Mortiboys, Parkinson’s UK Senior Research Fellow from the University of Sheffield:

“We demonstrated the beneficial effects of UDCA in the tissue of LRRK2 carriers with Parkinson’s disease as well as currently asymptomatic LRRK2 carriers. In both cases, UDCA improved mitochondrial function as demonstrated by the increase in oxygen consumption and cellular energy levels.”

The research is also the first to show beneficial effects of UDCA on dopaminergic neurons, the nerve cells affected in Parkinson’s disease, in a fly model of Parkinson’s disease which carries the same genetic change as some patients with the condition.

Oliver Bandmann, Professor of Movement Disorders Neurology at the University of Sheffield and Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, added:

“Whilst we have been looking at Parkinson’s patients who carry the LRRK2 mutation, mitochondrial defects are also present in other inherited and sporadic forms of Parkinson’s, where we do not know the causes yet. Our hope is therefore, that UDCA might be beneficial for other types of Parkinson’s disease and might also show benefits in other neurodegenerative diseases.”

Dr Arthur Roach, Director of Research and Development at Parkinson’s UK, which part-funded the study, said:

“There is a tremendous need for new treatments that can slow or stop Parkinson’s. Because of this urgency, the testing of drugs like UCDA, which are already approved for other uses, is extremely valuable. It can save years, and hundreds of millions of pounds.

It’s particularly encouraging in this study that even at relatively low concentrations the liver drug still had an effect on Parkinson’s cells grown in the lab.

This type of cutting-edge research is the best hope of finding better treatments for people with Parkinson’s in years, not decades.”

Mortiboys, H., Furmston, R., Bronstad, G., Aasly, J., Elliott, C., Bandmann, O. (2015)
UDCA exerts beneficial effect on mitochondrial dysfunction in LRRK2G2019S carriers and in vivo.
Neurology 85:1-7 doi: 10.1212/WNL.0000000000001905

Photo: Annie Cavanagh. Wellcome Images