Is this the groundbreaking drug that could finally put an end to Alzheimer’s symptoms? Scientists are optimistic that they have discovered a new treatment that can ‘turn off’ the gene responsible for causing the disease. The treatment, known as ALN-APP, utilizes a technology called ‘RNA interference’ to prevent the production of toxic proteins in cells. In a trial involving 20 participants, including four individuals from the UK, a single dose of the gene silencing treatment reduced the levels of a dangerous amyloid precursor protein by 90%. Even after six months, the levels remained 65% lower. This approach goes beyond previous drugs like lecanemab and donanemab, which only cleared the protein after it had already formed. By inhibiting the protein’s production, the ALN-APP treatment aims to halt disease progression at its source.
Dr. Catherine Mummery, the neurologist leading the UK trial, explained that stopping the production of these proteins is more effective than trying to remove them after the damage is already done. She compared it to turning off a tap instead of continuously mopping up the water. With an estimated 850,000 Britons and 5.8 million Americans affected by Alzheimer’s, the disease remains a pressing health concern. In 2022, dementia and Alzheimer’s were the primary causes of death in the UK, accounting for 65,967 lives lost. However, the ALN-APP treatment shows promise in its phase one trial, demonstrating safety and minimal side effects.
Amyloid proteins that clump together into plaques in the brain are believed to be toxic to brain cells and contribute to Alzheimer’s. By preventing their formation, scientists hope to prevent the onset of symptoms. While further testing is necessary before it can be approved for NHS use, the new treatment offers advantages over existing drugs. Unlike lecanemab and donanemab, which require frequent administration, ALN-APP only needs to be injected directly into the spinal cord via the lower back once or twice a year.
Donanemab, for example, has shown promising results in halting the decline of daily functioning by up to 40%. Lecanemab, on the other hand, has been found to reduce cognitive decline by 27% in individuals with Alzheimer’s. It was recently approved for use in the US and is expected to be introduced in the UK soon. Both drugs target the clearance of amyloid plaques from the brain.
Alzheimer’s disease is a degenerative brain condition characterized by the accumulation of abnormal proteins that cause nerve cells to die, resulting in memory loss, cognitive decline, and eventually the loss of everyday functioning. It affects over 5 million Americans and more than 1 million Britons. Early symptoms include memory loss, disorientation, behavioral changes, mood swings, and difficulties with tasks like managing finances or making phone calls. As the disease progresses, individuals may experience severe memory loss, become anxious and frustrated, lose the ability to walk, and require 24-hour care.
The discovery of the ALN-APP treatment brings hope for finding a solution to Alzheimer’s disease by targeting its underlying genetic causes. Continued research and testing will be crucial in determining its efficacy and safety for widespread use.
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