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Promising Alzheimer’s drug utilizes ‘gene silencing’ breakthrough

First-ever trial of gene-silencing Alzheimer’s drug successfully lowers harmful tau protein, offering potential disease-altering effects.

In a groundbreaking study led by University College London, UK scientists conducted the first-ever trial of a new drug for Alzheimer’s disease. This drug, called BIIB080 (IONIS-MAPTRx), utilizes a “gene silencing” approach to target the microtubule-associated protein tau (MAPT) gene, responsible for producing the harmful tau protein linked to the disease.

The trial, which took place from 2017 to 2020, involved 46 patients with an average age of 66. The drug was administered via intrathecal injection, directly targeting the nervous system. The results, published in Nature Medicine, showed that the drug was well tolerated, with no serious adverse events reported.

Significantly, the highest dose of the drug led to a more than 50% reduction in levels of total tau and phosphor tau concentration in the central nervous system (CNS) after 24 weeks. This reduction demonstrates the drug’s potential to alter the course of the disease by targeting tau accumulation, a hallmark of Alzheimer’s.

Consultant neurologist Dr. Catherine Mummery, from UCL Queen Square Institute of Neurology & the National Hospital for Neurology and Neurosurgery, commented on the findings: “The results are a significant step forward in demonstrating that we can successfully target tau with a gene silencing drug to slow – or possibly even reverse — Alzheimer’s disease, and other diseases caused by tau accumulation in the future.”

This breakthrough study opens up new possibilities for treating Alzheimer’s and other tau-related diseases. While further research is needed to evaluate the drug’s efficacy in larger and more diverse populations, the successful targeting of the tau protein is a significant milestone in the fight against Alzheimer’s, a disease for which there are currently no tau-specific treatments available.


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