UDCA and Parkinson's - Ursodeoxycholic Acid

Our panel of international Parkinson’s experts – the Linked Clinical Trials (iLCT) committee – concluded that following assessments, UDCA (a treatment already used for liver disease) was a top performer as a potential treatment for Parkinson’s.

As a result, Cure Parkinson’s co-funded a UK clinical trial in people with Parkinson’s and the results have been formally presented. Cure Parkinson’s is supporting Professor Bandmann in progressing this iLCT prioritised drug into the next phase of research, as rapidly as possible.

In 2018, Cure Parkinson’s awarded a grant to Professor Oliver Bandmann and colleagues to run a phase 2 clinical trial of UDCA in people with Parkinson’s – called the ‘UP-Study’. The trial involved 30 patients, two-thirds of the volunteers took UDCA while the remainder took a placebo drug – with no active ingredients.

The study was a placebo-controlled, double blind, randomised clinical trial. Before, during and after 48 weeks of daily tablets, various measures were taken from the participants. These included movement and mobility symptoms, along with a high-tech scan to see if UDCA is improving brain chemistry.

UDCA: phase 2 trial results published

Professor Oliver Bandmann discusses the research behind UDCA’s potential as a treatment for Parkinson’s

UDCA – the background

In 2013, researchers found that UDCA was a top performer in a screen for potential anti-Parkinson’s drugs. The researchers were looking for compounds that could address a process known as mitochondrial dysfunction, which is linked to Parkinson’s. They found that the long-established liver drug UDCA appeared to rescue this problem in cells of tissue samples taken from people with Parkinson’s.

Mitochondrial Dysfunction

UDCA – the history behind this promising drug for Parkinson’s

UDCA (ursodeoxycholic acid, or ursodiol) is a natural bile acid that was approved as a drug in the late 1980’s. It is used to dissolve gallstones and treat a form of liver disease called cirrhosis.

A decade after its approval, researchers looking at its action in cirrhosis found that UDCA has a beneficial effect on mitochondria – the small energy processing parts within cells [1]. This has the knock-on effect of preventing liver cells from self-destructing . More recently, scientists in Korea found UDCA exerted similar effects in nerve cells that had been exposed to a toxin [2], to mimic Parkinson’s.

Meanwhile, researchers in Sheffield hit upon UDCA via a different route [3]. Professor Oliver Bandmann and colleagues screened a library of 2,000 compounds specifically for their impact on the faulty mitochondrial processes seen in Parkinson’s. They tested all the compounds in cells of people with Parkinson’s and discovered that UDCA appeared to rescue mitochondria function and normalise the energy levels in the cells.

This finding was then replicated in a further study [4], which also affects mitochondria. A similar effect has also been seen in animal models [5] with induced Parkinson’s – here UDCA also appeared to have anti-inflammatory properties which helped cells to survive.