Scientists believe they are a step closer towards developing a nasal spray to treat motor neurone disease (MND) and some types of dementia. Experts at the University of Sheffield suggest using a small peptide – a molecule containing several amino acids – could block mutant repeated strands of information from being used by the body to make toxic components.
It could prevent the death of nerve cells and protect them from degeneration.
The scientists believe a 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.
Dr Brian Dickie, of the MND Association, which backed the study, said: “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.”
For the study, published in Science Translational Medicine, researchers worked in the lab with nerve cells reprogrammed from MND patients, plus some fruit flies and mice.
Guillaume Hautbergue, who led the study, 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.
“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.”
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