News and milestones
PRiSM trial opens to people at genetic risk
PRiSM (NN112), the first-in-human trial of PrP-siRNA, now also enrolls pre-symptomatic people with a genetic variant that carries a high lifetime risk of prion disease, alongside patients in early symptomatic stages. PrP-siRNA is a divalent siRNA given once by lumbar puncture, designed to lower the amount of prion protein made in the brain. This is a single-dose phase 1 trial: it looks for a dose that is safe and lowers prion protein in spinal fluid, and is not designed to show a benefit. The trial is sponsored by the Broad Institute, funded by the US National Institutes of Health, and runs at 5 sites in the United States only; the drug is not available outside the trial. People interested should talk with their own physician and contact a study site listed on ClinicalTrials.gov (NCT07444580), whose update is still under review. More in the trial FAQ, on CureFFI and on Prion Alliance.
CJDbio goes online
The biobank has a new international home at cjdbio.org, with information for researchers and families. A secure research data platform is in preparation.
A second gene-silencing trial begins
A first-in-human phase 1 trial of an siRNA that lowers prion protein production started at sites in the United States, funded by the US National Institutes of Health. It tests safety and the effect on prion protein levels after a single injection.
PrionAtRisk meets in Vienna
The international PrionAtRisk consortium, which the foundation joined through its biobank, met to plan its search for early biomarkers and treatment targets.
First antisense trial in prion disease
A human trial of an antisense oligonucleotide designed to lower prion protein began at 13 centres worldwide, including patients from our community.