First ’gene silencing’ drug for Alzheimer’s disease shows promise

tau protein - A world-first trial at UCL and UCLH has found a new genetic therapy 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 consultant neurologist Dr Catherine Mummery (UCL Queen Square Institute of Neurology & the National Hospital for Neurology and Neurosurgery), 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- MAPTRx ) , which is an antisense oligonucleaotide (used to stop RNA producing a protein), to 'silence' the gene coding for the tau protein - known as the microtubule-associated protein tau (MAPT) gene. This prevents the gene from being translated into the protein in a doseable and reversible way. It will also lower the production of that protein and alter the course of disease. Further trials will be needed in larger groups of patients to determine whether this leads to clinical benefit, but the phase 1 results published in Nature Medicine - with results from 46 patients - are the first indication that this method has a biological effect. There are currently no treatments targeting tau.