THE ARISING · Longevity & Healthspan
Old Liver Drug UDCA Joins 2,000-Person Parkinson's Trial Targeting Mitochondria
UDCA, a long-used liver medicine, is joining the EJS ACT-PD platform trial as a potential treatment to slow Parkinson's disease. The trial aims to recruit 2,000 people; efficacy remains unproven.
The drug is ursodeoxycholic acid, or UDCA. According to the University of Sheffield, it has been used to treat certain liver conditions for over 30 years. Researchers there found it has considerable promise for improving how mitochondria work in Parkinson's disease.
Mitochondria are often called the cell's power plants. They supply the energy cells need to function. According to the report, losing mitochondrial function in dopamine-producing nerve cells, in the brain area that controls movement, is believed to play an important role in how Parkinson's progresses. The idea is that protecting those cells from malfunction could slow the disease.
UDCA was found through a screening effort. The university reports that Sheffield-led researchers tested an entire drug library in tissue samples from people with Parkinson's, which it describes as a first. UDCA emerged as a candidate.
An early-stage clinical trial, called the UP study, then tested it in people with Parkinson's. According to the report, the drug was safe and well tolerated. Oliver Bandmann, a professor of movement disorders neurology at Sheffield who led the drug's development, said the study also provided encouraging evidence of effects on brain energy metabolism.
Now UDCA joins the Edmond J Safra Accelerating Clinical Trials in Parkinson's Disease trial, known as EJS ACT-PD. It becomes the third active treatment arm, alongside telmisartan and terazosin. The trial is led nationally by University College London and Newcastle University. It aims to recruit 2,000 participants across 40 NHS hospital sites. UCL reported 26 open sites and 189 participants randomized as of November 4.
Participants who join from now on will be randomly assigned to one of five groups: the three active drugs and two placebo groups, depending on which treatments they are eligible for. Neither participants nor the study team know who receives which treatment while the trial runs.
UDCA was picked after experts and people with Parkinson's reviewed hundreds of potential therapies, looking for those with the strongest evidence for slowing progression. Bandmann will co-lead the UDCA arm with Tom Foltynie of UCL and Camille Carroll of Newcastle.
Carroll said the flexible design was built to add promising drugs as evidence emerges, so more treatments can be tested faster within the same trial. The report notes it is rare for an academically led drug discovery program to move from laboratory discovery through a phase 2 trial to a definitive phase 3 trial.
Whether UDCA slows Parkinson's remains to be shown, and large-scale testing is what will answer it. A positive result would give a long-established drug a new use. A negative one would help narrow the search for treatments that act on cell energy.
Watch for the first results from the EJS ACT-PD trial as the three drug arms are compared against placebo.