London: In a first, researchers have replicated a rare but deadly human brain disease in nematode worms and successfully treated it with a drug called resveratrol, suggesting a cure could be found for humans.
There is currently no known treatment for onset of adult neuronal lipofuscinosis (ANCL) which usually strikes some adults in their 30s and results in death by the mid-40s.
"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," said professor Alan Morgan from University of Liverpool in Britain.
ANCL has recently been identified as being caused by mutations in the gene called DNAJC5.
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 compounds 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 disease and in a way not previously seen - without the need to act through an enzyme produced in the body called SIR-2.1.
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, Morgan said.
"As we face an increasingly ageing population, having treatments for these conditions becomes ever more critical."
The study appeared in the journal Human Molecular Genetics.