Breakthrough ‘Gene Silencing’ Treatment for Alzheimer’s Disease Shows Promise in Trial

by time news

New Treatment Offers Hope in the Fight Against Alzheimer’s Disease

A groundbreaking new trial is currently underway, testing a treatment that could potentially revolutionize the way we combat Alzheimer’s disease. The trial, which includes 20 participants, four of whom are British, is utilizing a cutting-edge technology called RNA interference to target the gene responsible for causing the disease.

The treatment, known as ALN-APP, aims to “turn off” the gene that produces the dangerous amyloid precursor protein, a major contributor to Alzheimer’s disease. The trial has shown promising results, with a single dose of the treatment reducing the levels of the protein by 90%. Even after six months, the levels remained 65% lower. This approach goes beyond previous treatments that focused on clearing the protein once it was already present.

Dr. Catherine Mummery, the neurologist leading the trial in the UK, explained the significance of the new approach, stating, “Instead of mopping up the proteins, this is about going upstream and stopping them from being produced in the first place. If you turn off the tap, you’ve got a much better chance of preventing further damage.”

Alzheimer’s disease affects millions of people worldwide, with around 850,000 Britons and 5.8 million Americans currently living with the condition. It is the leading cause of dementia, impacting an individual’s ability to remember, think, and make decisions necessary for daily activities. In 2022, dementia and Alzheimer’s were the leading cause of death in the UK, responsible for 65,967 deaths.

Results from the phase one trial, which demonstrated the treatment’s safety and minimal side effects, were recently presented at a conference in Amsterdam. While further testing is required before the treatment can be approved for NHS use, it offers hope for preventing the development of Alzheimer’s symptoms by targeting the production of toxic proteins.

The ALN-APP treatment stands out not only for its effectiveness but also for its dosing schedule. Unlike other treatments that need to be administered multiple times a month, the ALN-APP treatment requires only one or two injections directly into the spinal cord via the lower back per year.

Although the treatment is still in its early stages, scientists believe it has the potential to transform the landscape of Alzheimer’s treatment. The ongoing trial will provide valuable insights into the long-term effects and efficacy of the treatment, bringing us one step closer to finding a cure for this devastating disease.

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