New gene brings fresh hope for Parkinson’s cure
The discovery of a gene controlling the body's waste disposal system may open the door to fighting conditions including Parkinson's and Huntington's diseases.
The breakthrough by Monash and Cambridge University scientists also provides a potential new therapy to boost the body's destruction of rogue proteins that cause a range of neurodegenerative diseases if left to accumulate.
Early trials in animals and on human cells show that by increasing the level of the gene - a microRNA called miR-1 - the clumps of toxic proteins could be cleared.
Associate Professor Roger Pocock and his team at Monash's Biomedicine Discovery Institute will soon began new trials to determine how effectively treatment with a molecule called interferon-beta can clear Huntington's disease in mice.
If successful, he hopes human trials can take place in within the next five years.
Remarkably, the initial breakthrough came while examining the role of a mystery gene present in both primitive worms and modern humans.
"We found, remarkably, that microRNA protects Huntington aggregation in worms," Assoc Prof Pocock said.
"I'm not saying that it is causing it, but having reduced levels of miR-1 could prevent you from dealing with the disease.
"We have framed it with regards to Huntington's and Parkinson's in this paper but, what we have identified with regard to the regulator of autophagy, is generally good for you.
"So any way you can boost autophagy improves your lifespan, improves your general health - not that we want to be providing everyone with more of this gene."
Because transparent Caenorhabditis elegan worms and human are separated by 600 million years of evolution, the presence of the same gene in both species indicated it must have a fundamental role dating back to the earliest requirements for survival.
The research, recently published in the eLife journal, found that having removing the miR-1 gene prevented autophagy - the body's process for clearing malfunctioning proteins.
Cambridge's Professor David Rubinsztein has also found miR-1 is found in low levels in patients with neurodegenerative disease who are no longer able to clear toxic proteins from nerve cells.
But, the collaboration found when human cells were supplied with interferon-beta the production of miR-1 was boosted, and the disease cleared.
"We have looked at exactly the same genes in humans and found that micro RNA relate to the same gene in humans to regulate autophagy, to remove Huntington's aggregation," Assoc Prof Pocock said.
"It was amazing that this same pathway controlled the same events in these two distant organisms."
After gaining a provision patent on their discovery, the scientists will conduct further preclinical tests for Huntington's and Parkinson's disease while in discussions with pharmaceutical companies.