In a groundbreaking trial conducted at University College London (UCL) and University College London Hospitals (UCLH), a new genetic therapy for Alzheimer’s disease has been discovered, which effectively lowers the levels of the harmful tau protein, a known contributor to the disease. The trial, led by consultant neurologist Dr. Catherine Mummery, is the first to employ a ‘gene silencing’ approach in dementia and Alzheimer’s disease.
The genetic therapy uses a drug called BIIB080 (IONIS-MAPTRx), an antisense oligonucleotide, to silence the gene coding for the tau protein, preventing its translation and production in a doseable and reversible manner. Results from the phase 1 trial, involving 46 patients, published in Nature Medicine, show that the drug was well tolerated and led to a significant reduction in total tau and phosphor tau concentration in the central nervous system after 24 weeks in the treatment groups receiving the highest dose of the drug.
The successful trial paves the way for further research to evaluate the drug’s impact on slowing or even reversing the progression of Alzheimer’s disease and other diseases caused by tau accumulation. This marks a significant advance in the treatment of Alzheimer’s and opens new opportunities for targeting tau with gene silencing drugs in the future.