Dementia and MND sufferers given hope by UK boffins' innovative nasal spray idea

01 March 2023 , 19:00

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Professor Guillaume Hautbergue was the author

Scientists could develop a nasal spray to treat the most common genetic form of motor neurone disease (MND) and some types of dementia.

“World leading” British researchers believe they could be a step closer to halting the devastating death of nerve cells that leads to disability and death.

Early-stage studies by Sheffield University suggest using a small peptide could block mutant repeated strands of information from being used by the body to make toxic components that contribute to frontotemporal dementia.

The peptide is a molecule containing several amino acids and lab studies suggest it could prevent the death of the nerve cells.

Author Prof Guillaume Hautbergue said: “When we tested our innovative approach by adding the peptide to the food eaten by fruit flies, not only did the peptides block the damaging mutations which cause MND and FTD from being transported to the cell’s nucleus, we actually saw an improvement in their neurofunction.

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“This means the peptide is effectively blocking the progression of the neurodegenerative condition and also helping to restore the function to the affected nerve cells.

“This concept of using peptides to block destructive mutations unlocks such an exciting and innovative treatment pathway which until now has not been explored by scientists.

“MND and FTD are devastating diseases which currently have no cure.”

“You could imagine using this peptide as a nasal spray,” he added.

Professor Hautbergue suggested it could be a nasal spray

For the study, published in the journal Science Translational Medicine, researchers worked in the lab with nerve cells reprogrammed from MND patients, as well as testing the peptide on fruit flies and mice.

The peptide could self-propel itself into the nucleus of a cell to stop mutant repeated RNA molecules being transported into the cell’s fluid, known as the cytoplasm.

In illnesses such as MND, these mutant repeated RNA molecules are used in the cytoplasm to produce toxic repeat proteins which lead to the death of nerve cells.

Dr Brian Dickie, director of research at the MND Association, which backed the study, said: “These findings from a world leading research team in Sheffield demonstrate the importance of funding fundamental ‘discovery’ science.

“This work has provided important evidence in support of a completely new strategy to treat the most common inherited cause of both MND and FTD, with the ultimate goal of developing effective therapies for these devastating diseases.”

Researchers said a simple nasal spray could be developed for people with the commonest subtypes of MND and frontotemporal degeneration (FTD), which is the most common form of dementia among people under 60.

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Symptoms of frontotemporal dementia include personality and behaviour changes, such as acting inappropriately or impulsively, appearing selfish or unsympathetic, neglecting personal hygiene, overeating, or loss of motivation.

People may also suffer language problems, such as speaking slowly, getting words in the wrong order or using words incorrectly.

MND affects the brain and nerves, causing weakness that gets worse over time.

Early symptoms can include weakness in an ankle or leg, slurred speech, difficulty swallowing, a weak grip and muscle cramps and twitches.

Martin Bagot