London: UK scientists have conducted the first-ever trial of a new drug for Alzheimer’s disease that is able to safely and successfully lower levels of the harmful tau protein known to cause the disease.
The trial, led by a team at the University College Lon- don, represents the first time that a “gene silencing” approach has been taken in dementia and Alzheimer’s disease. The approach uses a drug called BIIB080 (IONIS-Marx) to “silence” the gene coding for the tau protein – known as the microtubule-associated protein tau (MAPT) gene. Th- is prevents the gene from being translated into the protein in a dose able and reversible way. It will also lower the pro- duction of that protein and alter the course of disease.
“We’ll need further research to understand the extent to which the drug can slow progression of… the disease,” said consultant neurologist Dr Catherine Mummery at UCL. “But the results are a signify- cant step forward,” she said.
The phase 1 trial looked at the safety of BIIB080, what it does in the body, and how well it targets the MAPT gene. In all, 46 patients, with an average age of 66, were enrolled in the trial which took place from 2017 to 2020. The trial loo- ked at three doses of the drug.
The research team also looked at two forms of the tau protein in the central nervous system (CNS) – a reliable indicator of disease-over the duration of the study. The results, published in Nature Medicine, found a greater than 50% reduction in levels of total tau and phosphor tau concentration in the CNS after 24 weeks in the two treatment groups that received the e of the drug.