A drug already been in use to treat liver disease may slow down the progression of Parkinson's disease, according to scientists from the University of Sheffield in the United Kingdom. Researchers from the Sheffield Institute of Translational Neuroscience (SITraN), in collaboration with scientists from the University of York, believe that their findings support the fast-tracking of the drug ursodeoxycholic acid (UDCA) for a clinical trial in Parkinson's patients, as published in the journal Neurology and reported in Drug Discovery & Development (http://www.dddmag.com/news/2015/08/promising-drug-parkinsons-disease-study-supports-fast-track-clinical-trials?et_cid=4731052&et_rid=45505806&type=cta).
As the Mayo Clinic explains, Parkinson's disease is a progressive disorder of the nervous system that affects movement. It develops gradually, sometimes starting with a barely noticeable tremor in just one hand. While a tremor may be the most well-known sign of Parkinson's disease, the disorder also commonly causes stiffness or slowing of movement (http://www.mayoclinic.org/diseases-conditions/parkinsons-disease/basics/definition/con-20028488).
According to 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."
Oliver Bandmann, Professor of Movement Disorders Neurology at the University of Sheffield and Honorary Consultant Neurologist at Sheffield Teaching Hospitals NHS Foundation Trust, said that he hopes "the UDCA might be beneficial for other types of Parkinson's disease and might also show benefits in other neurodegenerative diseases." With good results from the in vitro drug screen, the researchers wanted to further investigate and confirm the potential of UDCA in vivo - in a living organism," he said.
According to the study, which was funded by Parkinson's UK, the Wellcome Trust and the Norwegian Parkinson Foundation, while a mutation in the LRRK2 gene is the single most common inherited cause of Parkinson's disease, the exact mechanism that leads to Parkinson's is not clear. Mitochondria defects play a role in many diseases that affect the nervous system, including Parkinson's and Motor Neuron Disease. Because nerve cells have high energy demands, defects in the cell's energy generators will affect their survival.
Collaborators Rebecca Furmston, White Rose PhD student, and Dr Chris Elliott, from the University of York's Department of Biology, showed the effects of UDCA in vivo on the fruit fly (Drosophila melanogaster). In fruit flies, the mitochondrial defects caused by the LRRK2 mutation to dopaminergic neurons can be monitored through the progressive loss of visual function. Flies carrying the mutation maintained their visual response when fed with UDCA, demonstrating the rescue of dopaminergic signaling. The researchers believe that testing the drug, which is already approved for other uses, could save considerable time and money while meeting the urgent needs of patients with Parkinson's disease.