Revolutionary Alzheimer's drug can stop symptoms - how it could help millions

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A revolutionary drug is bringing hope to Alzheimer
A revolutionary drug is bringing hope to Alzheimer's patients (Stock photo) (Image: Getty Images/iStockphoto)

A revolutionary drug could offer some hope to Alzheimer's sufferers as it has been proven to alleviate symptoms and prevent further damage in people's brains.

The new treatment - currently known as ALN-APP - dials down the gene that produces the proteins that cause Alzheimer's. The results of a phase one trial of the drug, which were published at a medical conference in Amsterdam last month, suggest that a single dose of the drug can reduce levels of amyloid precursor protein by up to 90 per cent.

The test, which involved 20 patients, four of whom were in the UK, also showed that levels of the protein were still around 65 per cent lower after six months. The protein is responsible for clumping into the toxic sticky plaques in the brain, causing memory loss associated with Alzheimer's.

Scientists now believe that if given early enough, the new drug could stop patients from developing symptoms of Alzheimer's by stopping the process at its source. The trial came after two other drugs - lecanemab and donanemab - were found to show down the root cause of Alzheimer's, reducing cognitive decline by between a quarter and a third.

However, the difference between those drugs and the new gene-silencing approach is that the latter stops the proteins from being produced in the first place, explained Dr Catherine Mummery, consultant neurologist at University College London Hospitals, who is leading the UK arm of the trial. She said: "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."

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After proving that a single dose reduces protein protection, the team will now expand the trial by giving repeated doses to see if levels can be brought down even further. The drug is given via injection in the lower back into the cerebrospinal fluid surrounding the spinal cord which enables it to travel to the brain.

Anne Warburton, one of the UK patients who took part in the trial, said she thinks the drug "helps tremendously", reports The Sunday Times. Shortly before her 60th birthday, the woman would ask the same questions several times as her brain was not able to "hang on to information very long", her husband Peter explained. She added: "It drove people nuts!" But the former librarian, from Bedfordshire, said: "You have to make the most of your life. I have a ridiculous sense of humour and that is a godsend."

Last month, health officials in the US granted full approval to a landmark Alzheimer’s drug which has been shown to slow the cognitive decline caused by this form of dementia. The Food and Drug Administration (FDA) approval cleared the way for Medicare and other insurance plans to begin covering treatment using Leqembi for patients with mild dementia and other symptoms caused by early Alzheimer's disease.

Japanese drugmaker Eisai received conditional approval from the FDA in January based on early results suggesting Leqembi worked by clearing a sticky brain plaque linked to the disease. The FDA confirmed those results by reviewing data from a larger, 1,800-patient study in which the drug slowed memory and thinking decline by about five months in those who got the treatment, compared to those who got a dummy drug. "This confirmatory study verified that it is a safe and effective treatment for patients with Alzheimer's disease," said FDA's neurology drug director, Dr. Teresa Buracchio, in a statement.

Chiara Fiorillo

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