UCLH trial of world-first treatment for Alzheimer’s disease progresses into larger trial

UCLH’s trial of the world’s first ‘gene silencing’ drug for Alzheimer’s disease has progressed into a larger and later stage trial, after promising early results.

UCLH has recruited the first participant in the UK into the phase 2 trial which is looking at whether an investigational drug called BIIB080 can slow progression of Alzheimer’s disease in people with mild cognitive impairment or mild dementia.

Dr Catherine Mummery is leading the trial which is taking place at the NIHR UCLH Clinical Research Facility.

Results from an early stage trial, published in Nature Medicine in April 2023, showed the drug successfully lowered levels of the harmful tau protein known to cause the disease, with no serious adverse side effects.

The aim of the drug is to ‘silence’ the gene which codes for the tau protein. This prevents the gene being translated into the protein, thus reducing protein levels. It is the first ‘gene silencing’ drug being trialled in Alzheimer’s disease.

The study, sponsored by Biogen, will be conducted in two parts. In the first part, the research team will compare BIIB080 at different doses with a placebo. In the second part of the study, all participants will receive the drug, and researchers will assess the safety of the drug, and how it affects participants’ daily living, thinking, and memory abilities in the longer term.

Dr Mummery said: “It is very exciting to move this trial into a later stage, following the promising results we saw in the initial trial. Our early-stage trial showed that we can successfully target tau with a gene silencing drug, and the next stages of the trial will give us vital data on the extent to which the drug is effective and who could benefit from it.”