Worm study provides hope for deadly disease of the brain

July 16, 2014

An untreatable and deadly neurodegenerative disease has been modelled and treated in worms by University of Liverpool researchers, suggesting a cure could be found for humans.

The scientists studied adult onset neuronal lipofuscinosis (ANCL) which usually strikes around 1 in 100,000 people in Europe and North America in their 30s and results in death by the mid-40s. There is currently no known treatment for this disease, though it has recently been identified as being caused by mutations in the gene called DNAJC5.

For the first time scientists, from the University's Institute of Translational Medicine, have replicated the disease in and successfully treated it with a drug called resveratrol.

The worms have a gene called dnj-14 which is their version of the human DNAJC5. Since the worms lifespan is normally only a few weeks, the symptoms of the disease show within a few days and this opens up the possibility of testing thousands of new for treatment within a short period of time – hastening the development of a cure.

One compound, resveratrol, has already been shown by the research team to be effective in treating the and in a way not previously seen – without the need to act through an enzyme produced in the body called SIR-2.1.

Physiologist, Professor Alan Morgan led the study. He said: "ANCL is fortunately rare but it is currently untreatable. This research allows us to quickly test compounds which could be used for treatment. Of the first batch of compounds we used in testing our model, one has already shown encouraging effects."

Professor Morgan and his colleagues Professor Bob Burgoyne and PhD student, Sudhanva Kashyap now plan to test more compounds with the model and to explore how nematode can be used to study more .

Professor Morgan concluded: "As we face an increasingly ageing population, having treatments for these conditions becomes ever more critical. Studying how these diseases work in a simple organism which is easy and cheap to breed, can speed up the process of developing effective drugs."

More information: Human Molecular Genetics hmg.oxfordjournals.org/content/early/2014/07/02/hmg.ddu316.long

Related Stories

Scientists use worms to unearth cancer drug targets

August 9, 2012

Through novel experiments involving small nematode worms, scientists from Wyoming have discovered several genes that may be potential targets for drug development in the ongoing war against cancer. Specifically, researchers ...

Mutation stops worms from getting drunk

July 15, 2014

Neuroscientists at The University of Texas at Austin have generated mutant worms that do not get intoxicated by alcohol, a result that could lead to new drugs to treat the symptoms of people going through alcohol withdrawal.

Recommended for you

Flow means 'go' for proper lymph system development

July 27, 2015

The lymphatic system provides a slow flow of fluid from our organs and tissues into the bloodstream. It returns fluid and proteins that leak from blood vessels, provides passage for immune and inflammatory cells from the ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.