The trial, led by Dr. Catherine Mummery, has used a ‘gene silencing’ technique for the first time in treating Alzheimer’s disease and dementia.
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 phase 1 trial looked at the safety of BIIB080, what it does in the body, and how well it targets the 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. The trial looked at three doses of the drug, given by intrathecal injection (an injection into the nervous system via the spinal canal), compared with the placebo. Results show that the drug was well tolerated, with all patients completing the treatment period and over 90% completing the post-treatment period.
Patients in both the treatment and placebo groups experienced either mild or moderate side effects — the most common being a headache after injection of the drug. However, no serious adverse events were seen in patients given 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.