David Spencer, MD, director of the Comprehensive Epilepsy Center and professor of neurology at Oregon Health and Science University, talks about the first-in-human trial of interneuron cell therapy for treatment of focal epilepsy and explains the next steps.
The first phase of a first-in-human trial of NRTX-1001, an intraneuron cell therapy treatment for epilepsy, focused on safety, and next will come a randomized, controlled trial to assess efficacy, according to David Spencer, MD, director of the Comprehensive Epilepsy Center and professor of neurology at Oregon Health and Science University.
How long is the clinical trial for interneuron cell therapy for treatment of focal epilepsy, and what are the next steps if it proves to be successful?
The cell therapy approach for treating epilepsy that we're exploring is a major commitment to the participants who've taken a big step to join into this study. It's a long-term approach. From the perspective of a person entering into the trial, the main part of the study lasts 2 years, but there's an extension study that continues beyond that for up to 15 years, where we'll continue to follow people and track their outcomes.
From the perspective of the overall study, It has 2 phases. The first phase we’re especially focusing on the safety of doing the cell implants, and so we're going at a cautious pace. After the first implant was completed last June, there was a waiting period, there was a lot of safety assessments and a committee met to ensure that the outcome looks good in that person before we moved on to doing an implant in the second person, which was in last fall of 2022. We're anticipating 5 participants receiving the cell implants, another pause in assessments, a second 5 potentially at a higher dose of cells.
And then at that point in the trial, we'll shift into a hybrid approach continuing to look at safety, but also looking more closely at efficacy. The second phase will be a small, randomized controlled trial, so some people will get cell therapy initially, others will get delayed therapy, and we'll try to look very carefully at the responses that are very specific to cell implants.