Mutations in the LRRK2 gene in humans are known to be the most common genetic contributor to Parkinson’s disease, although the mechanisms have been poorly understood.
But in tests on in vitro rat neurons, University of Sheffield researchers found that these mutations interfere with how vital proteins and minerals are transported across nerve cells, causing impaired motor functions.
The study, published in Nature, also looked at fruit flies with faulty copies of the LRKK2 gene. It found that by targeting the transport system in these neurons with specific drugs, these defects could be reversed, thereby alleviating Parkinson’s disease-like symptoms in the flies.
'Rescue effect' for patients
The drugs, known as deacetylase inhibitors, work by restoring normal transport along the faulty neurons and therefore could have a ‘rescue effect’ in affected cells.
Study author Dr Alex Whitworth said: ‘By targeting the transport system with drugs, we could not only prevent movement problems, but also fully restore movement abilities in fruit flies who already showed impaired movement marked by a significant decrease in both climbing and flight ability.’
The authors concluded that the ‘very promising results’ show that drug treatments ‘may have therapeutic potential’ for treating humans with Parkinson’s, although much more research will be needed to see if the effects could be replicated outside of animal tests.
Dr Beckie Port, from Parkinson’s UK, which helped to fund the study, said: ‘This research gives hope that, for people with a particular mutation in their genes, it may one day be possible to intervene and stop the progression of Parkinson’s.’