Scientists in drug ‘breakthrough’ on Parkinson’s disease

The breakthrough announced today by experts from the world-leading Sheffield Institute for Translational Neuroscience follows a major drugs trial in the laboratory using skin cells from people with Parkinson’s disease.

Researchers tested more than 2,000 compounds to find out which could make faulty mitochondria work normally again. Mitochondria act as the power generators in all cells in the body but when they malfunction they trigger the death of brain cells which leads to Parkinson’s.

One of the promising medications identified though the research is a synthetic drug called ursodeoxycholic acid. The therapy has been used for several decades to treat forms of liver disease, which means researchers will be able to immediately start to test its safety in people with Parkinson’s.

This will look for the optimum dose to ensure enough of the drug reaches the part of the brain where Parkinson’s develops. Based on this information, larger trials can be carried out to assess its potential to treat the disease, which affects one in 500 people in the UK.

The extensive drug screen, which took over five years to complete, was funded by research charity Parkinson’s UK.

Oliver Bandmann, reader in neurology at the Sheffield institute, said: “Parkinson’s is so much more than just a movement disorder. It can also lead to depression and anxiety, and a host of distressing day to day problems like bladder and bowel dysfunction.

“The best treatments currently available only improve some of the symptoms, rather than tackle the reason why Parkinson’s develops in the first place, so there is a desperate need for new drug treatments which could actually slow down the disease progression.”

Kieran Breen, director of research at Parkinson’s UK, said: “This is a really exciting time for Parkinson’s research.

“For the first time, we are starting to identify drugs that will treat the Parkinson’s – possibly slow down or halt its progression – rather than just the symptoms. This will bring us closer to our ultimate goal of a cure for Parkinson’s.”

The results of the ground-breaking study are published in the journal BRAIN today.