A drug used to treat liver cirrhosis may be an effective treatment for a form of dementia and motor neuron disease, the scientists found.
The research, conducted by the University of York in collaboration with the University of Sheffield, used brain cells from fruit flies and rats to model the neurodegeneration process that occurs in patients with frontotemporal dementia (FTD).
Researchers identified new proteins involved in protecting neurons and found that ursodeoxycholic acid – an already approved drug with very low toxicity – increases these proteins and protects neurons from death.
The study authors will now undertake further research to find out exactly how the drug works to protect neurons and whether more targeted drugs could be developed to treat FTD and a number of other neurodegenerative conditions.
FTD affects the frontal and temporal lobes of the brain. Unlike other forms of dementia that primarily affect people over the age of 65, FTD tends to start at a younger age with most cases being diagnosed in people between the ages of 45 and 65.
Senior author of the study, Dr Sean Sweeney, of the Department of Biology at the University of York, said, “We are on the verge of being able to ‘reuse’ a drug used for a liver disorder, which has very little toxicity. in humans “.
“The mechanism of action of this drug is currently unknown and the work we will now do to increase our understanding of how it works could help us lengthen and improve the lives of patients with FTD and potentially other neurodegenerative conditions as well.”
Up to 50% of FTD cases have a genetic history of the disease in the family, and previous research has identified nine genes that may play a role in its development.
Lead authors of the study, Dr. Ryan West and Dr. Chris Ugbode, used one of these genes to develop their unique genetic models of the disease in fruit flies and rat neurons. In these models, they found that ursodeoxycholic acid keeps neurons in better health, but the drug is not a potential cure for the disease.
Dr West of the University of Sheffield said: “In our laboratory models the drug was effective for the treatment of frontotemporal dementia and motor neuron disease, but it does not correct the underlying deficits, suggesting that the drug is neuroprotective but not a care”.
Fiona Carragher, Director of Research and Influenza at the Alzheimer’s Society, added: “At present, there is no way to slow or cure frontotemporal dementia, one of the most common forms of dementia in people under 65. so we’re thrilled to see an existing drug stop brain cells from dying. Even though this is in the early stages, it’s a valuable first step on the road to finding a way to improve the lives of people with FTD and help them live longer. We need help to continue funding studies like this, so we ask the government to honor their commitment to funding for double dementia research – while finding new drugs from scratch costs billions and takes decades, we need to make sure that every penny. count on seizing the opportunities to reuse drugs already approved for other conditions. “
The neuroprotective activity of ursodeoxycholic acid in CHMP2B Intron5 models of frontotemporal dementia is published in Neurobiology of the disease.
The research was conducted in collaboration with the University of Sheffield and the Sheffield Institute of Translational Neuroscience and was funded by Alzheimer’s Society, Alzheimer’s research UK, Motor Neurone Disease Association and Wellcome Trust.
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