Clinical trial of a prion disease drug candidate begins enrolling participants
“Eric and Sonia are visionaries who are not only top-notch scientists, but have enormous ethical conviction. I’m honored to have an opportunity to work with them,” said Khvorova, professor in UMass’s RNA Therapeutics Institute and Program in Molecular Medicine. “Their commitment to transparency is paving a new path for running a clinical trial, so we’re not only advancing therapy for prion disease, it’s advancing the field as a whole.”
The initial phase of the trial will enroll 15 symptomatic patients who will each receive one dose of the siRNA by lumbar puncture, and new patients enrolled over the course of the trial will receive larger doses than patients enrolled earlier. The trial will also have an observational arm consisting of 15 additional participants who will not receive any treatment. Minikel says they hope to add pre-sympomatic participants in the future. They also plan to share safety data as soon as they are able.
“Standard drug development is, for all sorts of reasons, not well set up to be a learning system,” Vallabh said. “But we can’t afford to treat each shot on goal as a self-contained unit – everything needs to feed the mission. I wish the absolute best for this exact molecule, and I also know that however it performs in the clinic, this experience must move us forward. It’s our job to figure out how.”
This is now the second clinical trial that Vallabh and her team at Broad have helped to enable. The first one, initiated by Ionis Pharmaceuticals, began in 2023 for drug candidate ION717 and is ongoing. In their lab, Minikel and Vallabh are pursuing other possible therapeutic approaches for prion disease, including epigenetic and gene editing.
To learn more about PRiSM, visit ClinicalTrials.gov and CureFFI.org, where Minikel shares information for the prion patient and scientific communities. For patient resources, Minikel and Vallabh recommend visiting the CJD Foundation.
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