Key Points
A world-first trial at UCL and UCLH has found a new gene therapy for Alzheimer's disease that is able to safely 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 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 oligonucleotide (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..
It involved the UCL Dementia Research Centre, was supported by the NIHR UCLH Biomedical Research Centre, was supported by the NIHR UCLH Biomedical Research Centre, and took place at the Leonard Wolfson Experimental Neurology Centre at NHNN.. 46 patients, with an average age of 66, were enrolled in the trial -- which took place from 2017 to 2020..
But 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...
