New route to repurposed drugs for Parkinson's

Researchers in Oxford have developed a unique way to identify existing drugs that could potentially be repurposed for treating Parkinson's.

The new approach has already highlighted the potential of clioquinol, used in creams to treat skin infections. Although clioquinol is unlikely to be a future treatment due its side effects when taken orally, it could lead to new drugs that deliver the benefits without the risks.

This promising research, funded by Parkinson's UK, is published in the journal Human Molecular Genetics.

What the team did

The team first used a cutting-edge stem cell technique to turn skin cells from people with Parkinson's into dopamine-producing brain cells – identical to those that are lost in the condition.

Next they studied the patterns of gene activity in these Parkinson's brain cells and compared them to those observed in the same brain cell types grown from people of a similar age without the condition.

Dr Caleb Webber, Investigator at the Parkinson's UK funded Oxford Parkinson’s Disease Centre and co-lead author of the study, explains :

"When we compared gene activity between Parkinson's brain cells and healthy brain cells grown in the laboratory we found some key differences.

"These specific changes in gene activity, which are like a Parkinson's fingerprint, help tell us why the cells die in Parkinson's, and how we might save them."

Finally they used a global database which holds information about the effects of thousands of different drugs to look for ones that could make the gene activity in the Parkinson's brain cells more similar to that of healthy brain cells - and found clioquinol.

A powerful way to find new treatments

Professor Richard Wade-Martins, co-lead author on the study and Head of the Oxford Parkinson's Disease Centre, said:

"We're excited by the power and precision of this new approach for identifying drugs that could be helpful for Parkinson's.

"Because we're able to tap into huge global databases of information about drugs, this is a short cut to laboriously testing each drug individually in the lab.

"It's particularly exciting that this new approach immediately identified clioquinol – a drug whose properties are already being investigated for neurodegenerative conditions like Parkinson's and Alzheimer's.

"This gives us huge confidence that our approach works and we're now excited to start unearthing more promising potential treatments for Parkinson's."

The next step

Dr Arthur Roach, Director of Research at Parkinson's UK, added:

"There is a desperate need for new and better treatments for Parkinson's, and this research opens up a unique and speedy avenue for identifying drugs and compounds that have serious potential.

"The crucial next step is to convert interesting leads like this one into effective treatments that can go forward to be tested in clinical trials.

"This is what we aim to do through the Parkinson's UK Virtual Biotech, and we're planning to invest £11 million over the next 3 years to make new and better treatments a reality for people with the condition."