A drug can reverse the effects of two Parkinson's disease-causing mutations in fruit flies, a study reports.
Parkinson's disease does not always have an obvious cause, but in some cases it runs in families and can be traced to a mutation in a specific gene, such as LRRK2.
Researchers have now shown that two mutations in LRRK2 interfere with the system by which proteins and cellular structures are transported within neuron cells. They were able to counteract this effect on the transport system using drugs called deacetylase inhibitors, both in rat neurons, and in fruit flies expressing mutant LRRK2. These flies exhibit impaired flight and climbing abilities akin to the movement impairment seen in patients with Parkinson's disease.
'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, ' said Dr Alex Whitworth, one of the lead authors at the University of Sheffield.
The study, which was published in the journal Nature Communications, builds on prior reports that mutant LRRK2 binds to microtubules, the cell's transport tracks.
The study was partly funded by Parkinson's UK. Dr Beckie Port, research communications officer, 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.'
However, both she and the researchers stressed that any therapeutic implications of the study remain some way off.
'These are very promising results which point to a potential Parkinson's therapy. However, further studies are needed to confirm that this rescue effect also applies in humans,' said lead author Dr Kurt De Vos of the Sheffield Institute for Translational Neuroscience.
Dr Port said: 'The study has only been carried out in fruit flies, so much more research is needed before we know if these findings could lead to new treatment approaches for people with Parkinson's.'
Sources and References
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Sheffield drug study gives hope to disease sufferers
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New lead for potential Parkinson's treatment: Effects of high-risk Parkinson's mutation are reversible
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Increasing microtubule acetylation rescues axonal transport and locomotor deficits caused by ÔÇïLRRK2 Roc-COR domain mutations
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Studies suggest possible future treatments to reverse Parkinson's
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