This morning, Ionis Pharmaceuticals posted a small but important update to NCT06153966: the first trial site in Cleveland, OH is now recruiting:

For those just tuning in now, Sonia and I are not affiliated with Ionis. We are scientists at the Broad Institute working to develop a drug for prion disease, for which Sonia is at risk. For several years we’ve been collaborating with Ionis Pharmaceuticals on an antisense oligonucleotide to lower PrP, for 2 months we’ve known that a first-in-human trial was imminent, and for 2 weeks we’ve known a few details of that upcoming trial. Read those posts first for background. As of 2 weeks ago, the trial was posted but “not yet recruiting”. Now, it’s recruiting at one site: University Hospitals Cleveland Medical Center. Although the posting does not say so, we can infer that the trial site PI is likely Brian Appleby, director of the national prion surveillance center in Cleveland and a deeply experienced clinician and researcher in prion disease.

So now, for the first time, for people looking to enroll their loved one in a trial, we have some place to direct you to other than just sitting and waiting. In the posting, the contact information provided for patients to enroll is still via Ionis, 844-221-3587 / PrionDisease@clinicaltrialmedia.com. We wish you the best of luck.

As a reminder, a clinical trial is just an experiment. This is a Phase 1/2a trial, meaning at the start, it’s a Phase 1, meaning the primary goal is to assess safety. We don’t know that the drug works, and for now at least, this trial isn’t even designed to tell us whether the drug works. It’s just designed to assess safety and identify a dose level to move forward with. The trial is for symptomatic patients. Pre-symptomatic at risk are not eligible at this time.

What’s next? Ionis’s other first-in-human trials are always multi-site trials, and we can guess that this one will be no different. I expect that over the coming weeks to months, we will see several other trial sites around the world added to the ClinicalTrials.gov posting. I won’t write a blog post about each and every one, but at some point, I might circle back to analyze how they’ve done in terms of catchment area and coverage. I don’t know whether we’ll hear any announcement when the first patient has been dosed, or if we’ll just have to infer that such a milestone has likely been reached once enough time passes. But either way, there will surely be lots more to say as the trial takes shape, and at some point it will be worth reflecting on all the unknowns and the wide spectrum of possible outcomes of this trial, and where we now stand in our journey to develop a drug for prion disease. For today, I just wanted to share this happy update.