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.'
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