Two Baby Girls.

Where the modern world was born.

---

1. An exciting new genetic therapy for Alzheimer’s disease is safe and successfully lowered levels of the harmful tau protein known to cause the disease, in a world first trial at UCLH and UCL. The trial, led by consultant neurologist Dr Catherine Mummery at the National Hospital for Neurology and Neurosurgery, represents the first time that a ‘gene silencing’ approach has been taken in dementia and in AD. In this approach, the aim is to use a drug to ‘silence’ the gene coding for the tau protein – the microtubule-associated protein tau (MAPT) gene – with a drug called BIIB080, which is an antisense oligonucleotide. This prevents the gene being translated into the protein in a dose-able, reversible way. This can then 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 then leads to clinical benefit, but the phase 1 results published today in Nature Medicine are the first indication that this method has a biological effect. (Sources: uclh.nhs.uk, rcp.ac.uk, nature.com)