Repurposing Drugs

Have you heard the latest? Oxford University have developed a unique way to identify existing drugs that could potentially be repurposed for treating Parkinson’s by putting together the cutting-edge stem cell technologies and computational biology.  This research was published in the journal Human Molecular Genetics, and uses a stem cell technique to change a small piece of skin from those with Parkinson’s into dopamine-producing brain cells which is identical to those that are lost in Parkinson’s.  The patterns of gene activity in these Parkinson’s brain cells were studied and compared to those observed in the same brain cell types grown from people of a similar age without the condition.

Investigator at the Parkinson’s UK funded Oxford Parkinson’s Disease Centre, Dr. Caleb Webber and co-lead author of the study, found the following.  When gene activity between Parkinson’s brain cells and healthy brain cells grown in the lab were compared, they found key differences. These chances are like a Parkinson’s fingerprint and can tell why the cells die in Parkinson’s and how they might save them. They can now study the behavior of brain cells grown in a dish and learn important new things about the real human condition.

A global database was used which holds information about the effects of thousands of different drugs, many already in clinical use, to look for ones that could normalize the gene activity of the Parkinson’s brain cells.  A drug named clioquinol that was developed in the 1930’s and still used today in creams to treat skin infections was identified. Taken orally over extended time, it can have serious side effects. As originally developed, clioquinol is unlikely to be a future treatment for Parkinson’s, but new drugs based on clioquinol may deliver the benefits without the risks.

Co-lead author on the study and head of the Oxford Parkinson’s Disease Center, Professor Richard Wade-Martins said they are excited by the power and precision of this new approach for identifying drugs that could be helpful for Parkinson’s. They were able to tap into huge global databases of information about drugs and this provides a short cut to laboriously test each drug individually in the lab.

Being able to immediately identify clioquinol with this new approach is exciting. Dr. Arthur Roach, Director of Research at Parkinson’s UK, funders of the research, believes 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 next step would be to convert interesting leads like this one into effective treatments that can go forward to be tested in clinical trials, as they are planning to do through the Parkinson’s UK Virtual Biotech. They are planning to invest money over the next three years to make new and better treatments a reality for people with this condition.

Dr Fredda Branyon