The Daily Observer London Desk: Reporter- John Furner
A breakthrough new treatment for Alzheimer’s disease could ‘turn off’ the gene that helps cause the disease, scientists hope.
A trial currently taking place on 20 people, including four Brits, uses a new technology called ‘RNA interference’ which stops cells producing toxic proteins.
It has so far found that a single dose of the ‘gene silencing’ treatment – currently known as ALN-APP – can cut the levels of a dangerous amyloid precursor protein by 90 per cent, with the levels still 65 per cent lower after six months.
The research follows recent scientific breakthroughs to target the disease, where drugs lecanemab and donanemab managed to clear the protein once it was already present.
The ALN-APP treatment, however, goes a step further to stop it being produced in the first place by toning down the proteins that cause the disease.
A breakthrough new treatment for Alzheimer’s disease could ‘turn off’ the gene that helps cause the disease (file image)
Dr Catherine Mummery, the neurologist leading the trial in the UK, told The Times: ‘Instead of mopping up the proteins, this is about going upstream and stopping them being produced in the first place.
‘If you’re just mopping up the proteins that are already there, you’ve got to constantly clear the damage while the tap is still on.
‘But if you turn off the tap, you’ve got a much better chance of preventing further damage.’
Around 850,000 Britons and 5.8million Americans have Alzheimer’s disease.
The disease is the leading cause of dementia, a condition where suffers have an impaired ability to remember, think, or make decisions that interferes with doing everyday activities.
In April a study from the Office for National Statistics (ONS) revealed that dementia and Alzheimer’s were the leading cause of death in the UK in 2022, collectively accounting for 65,967 deaths.
Results from a phase one trial were published at a conference in Amsterdam last month, showing that the treatment is safe and displays minimal side effects.
In the trial, six people have been given a placebo drug, while the remaining 14 were given the real thing.
Neurologist Catherine Mummery (pictured) is leading the UK trial and hopes the treatment will stop dangerous proteins being produced in the first place
Amyloid proteins can stick together and form clumps, which later become plaques in the brain. It is thought to be toxic to brain cells.
Scientists hope that the treatment could prevent patients developing symptoms.
However further tests are still needed before it can be approved for NHS use.
While lecanemab and donanemab need to be given a couple of times a month, the new treatment, which is received through an injection directly into the spinal cord via the lower back, will only be needed once or twice a year.
Donanemab has been found to halt a reduction in the ability to perform daily activities by up to 40 per cent, according to initial findings published on May 3.
Less than a year ago lecanemab was found to slash cognitive decline among those with the memory-robbing condition by 27 per cent. It was approved for use in the US on July 7, while a UK introduction of the drug is ‘on the horizon’.
Donanemab works by clearing plaque clusters from the brain known as amyloid, which are closely associated with Alzheimer’s disease.
