Clinical trial of a prion disease drug candidate begins enrolling participants

The Broad Institute, in collaboration with the National Institutes of Health (NIH), has initiated a clinical trial for a new drug candidate to treat prion diseases, which are rare and fatal neurodegenerative disorders. The trial is expected to enroll 20 patients with sporadic fatal insomnia (sFI), a type of prion disease that causes severe insomnia and eventually leads to death. The trial will be conducted at the NIH Clinical Center in Bethesda, Maryland, and will involve a randomized, double-blind, placebo-controlled study. The drug candidate, known as Tafamidis, has shown promise in preclinical studies in slowing the progression of sFI. The Broad Institute's Dr. Matthew Schrag is leading the trial, and the NIH's Dr. David M. Holtzman is serving as a principal investigator.