Clinical trial of a prion disease drug candidate begins enrolling participants

A phase 1 clinical trial has begun enrolling 15 patients with prion disease to test a novel small interfering RNA (siRNA) drug candidate designed to reduce production of the disease-causing prion protein in the brain. This marks the first time this potential treatment has been tested in humans, with the trial assessing safety, dosing, and whether the drug warrants advancement to larger studies. Prion diseases are currently fatal neurodegenerative disorders with no cure, but animal research has shown that lowering prion protein levels can delay onset and slow progression.