Dr Mortiboys drug re-purposing project for Parkinsons UK

People with Parkinson’s do not have enough of a chemical called dopamine because nerve cells in their brain have died. Without it, people can find that their movements are slower, and a lack of coordination can cause major problems. Parkinson’s causes people’s hands and bodies to shake or freeze, which makes everyday activities such as eating, getting dressed, crossing a road or using a computer, difficult or dangerous. Once someone has Parkinson’s, life is never the same again.

Parkinson’s is also degenerative, so in the absence of a cure, those diagnosed have to face the bleak prospect of living with a debilitating condition that is just going to get worse. Current treatments only help mask the symptoms, and the main drug used to treat symptoms (Levodopa) becomes less effective over time and can have severe side effects, including involuntary muscle movements, hallucinations, delusions and confusion. Parkinson’s UK offers support from billboard to boardroom, helpline to health settings, we’re working together for everyone living with Parkinson’s.

Finding new uses for drugs that are already used for other conditions has great promise for quickly bringing new treatments to people with Parkinson’s. These drugs are already widely used, so they can speedily enter clinical trials. This approach has already been successfully used to identify diabetes drugs with potential for treating Parkinson’s.

The Gisela Graham Foundation has kindly given a grant to the work of Dr Heather Mortiboys who is currently looking for drugs that can improve the function of two key players inside cells that are known to stop working properly in Parkinson’s. Previous research has shown that the mitochondria, the energy-producing batteries inside cells, do not function properly and this contributes to cell death.

Dr Heather Mortiboys working on the drug re-purposing project

Normally, when mitochondria stop working they are removed by the waste disposal system of the cell which is called the lysosome. But blockages in this system also seem to occur in Parkinson’s.

The project has already made a list of 4,000 drugs which are already used to treat other conditions and ranked them by their ability to get into the brain and their known side effects. She has taken the top 224 ranked drugs and tested them on skin cells from people with an early-onset form of inherited Parkinson’s, people with a late-onset inherited form of Parkinson’s, and people with the condition who do not carry any genes known to increase risk.

The project has recently been extended so that Dr Mortiboys can investigate further the drugs she’s identified in greater detail and understand more about how they might work in Parkinson’s. To do this she and her team have made brain cells by reprogramming the patient skin cells.

The Mitochondria Bacteria

This means they can test the three groups of drugs in brain cells that are very like those that are lost in Parkinson’s to study their beneficial effects on the mitochondria and lysosomes and find out whether they have real promise as treatments for people with the condition.

If Dr Mortiboys is successful and finds strong evidence that any of these drugs have real promise, she plans to take them forward as rapidly as possible to be tested in clinical trials. Ultimately, she hopes her work will lead to better treatments that can slow or stop the development of Parkinson’s.

It can take up to 20 years to develop a new treatment. But as Dr Mortiboys is studying drugs that are already used for other conditions, a new treatment could be a reality in years rather than decades.