Scientists have reversed Parkinson’s-like symptoms in mice using a brain cell therapy that could one day lead to a cure for humans.
The groundbreaking technique instructs replacement cells to take on the function of dopamine-producing neurons that are killed by the disease.
The cause of the cells’ death is still unknown – but as dopamine levels in the brain drop, patients’ motor systems suffer, potentially causing tremors and making it difficult to walk.
There is currently no known cure for the condition, which is managed through prescription drugs.
The new study, published in Nature Biotechnology, describes how scientists eased the debilitating condition in mice.
The scientists devised a way to replace the damaged-neurons without injecting new ones into the brain, marking a departure from previous research.
Instead, the scientists applied a cocktail of small molecules to a sample of human brain cells, which were reprogrammed to closely match dopamine-producing neurons.
When the cocktail was later tested on mice with Parkinson’s-like symptoms, it appeared to reverse the effects of the disease.
Mice that had previously been struggling to shuffle around, were able to walk in a straight line, suggesting the cells had been reprogrammed.
The early-stage research could pave the way for further studies to see if the therapy is safe and if the dopamine-producing neurons work normally.
Patrick Lewis, a neuroscientist at the University of Reading, said the work was game-changing, but cautioned: “Moving from this study to doing the same in humans will be a huge challenge.”
Professor David Dexter of Parkinson’s UK said: “Further development of this technique is now needed.”
“If successful, it would turn this approach into a viable therapy that could improve the lives of people with Parkinson’s and, ultimately, lead to the cure that millions are waiting for.”
James Beck, chief scientific officer of the Parkinson’s foundation in the US, told STAT the therapy could give life back to someone with the disease, but said: “This is not going to happen in five years or possibly even 10, but I’m excited about the potential of this kind of cell replacement therapy.”