Scientists in London, UK, completed the first-ever study of a novel medicine for Alzheimers disease that can safely and effectively decrease levels of the toxic tau protein believed to cause the illness.

The technique employs the medication BIIB080 (IONIS MAPTRx) to "silence" the tau protein-coding gene, known as the microtubule-associated protein tau (MAPT) gene. This inhibits the gene from being translated into the protein in a doseable and reversible manner, lowering protein synthesis and changing the course of the illness.

The phase 1 trial examined the safety of BIIB080, administered intravenously to 46 individuals with an average age of 66.

According to the findings published in the journal Nature Medicine, the medicine was well tolerated, with all patients finishing the treatment term and more than 90% completing the post-treatment period.

The researchers also looked at two types of tau protein in the CNS and discovered a higher than 50% drop in total tau and phosphor tau concentrations after 24 weeks in the two treatment groups that got the highest dose of the medicine. There are presently no therapies that target tau, however, the US Food and Drug Administration has authorized aducanumab and lecanemab for use in certain instances.

Researchers suggested that more study is needed to determine the extent to which the medicine can halt the course of physical symptoms of illness, as well as to examine the drug in older and bigger groups of people and more varied communities.

(Source: https://health.economictimes.indiatimes.com/news/pharma/worlds-first-gene-silencing-alzheimers-drug-shows-promise/99800018)