The abstract is read at the publisher; the summary is JClub's.
A single infusion of mivocabtagene autoleucel in antibody-positive adult stiff person syndrome improved timed 25-ft walk at week 16 by a mean of -7.69 seconds (P<.001).
- Why it matters: Stiff person syndrome is progressive and disabling, and prior immunomodulatory or immunosuppressive therapies are often inadequate, leaving a need for effective, durable options with manageable safety.
- What they did: In a single-arm, open-label phase 2 trial at 3 US sites (September 2024 to May 2025), 26 treated adults received fludarabine/cyclophosphamide lymphodepletion followed by 1×10^8 CAR T cells, with week 16 efficacy and safety as primary end points.
- The result: Week 16 improvements also occurred across modified Rankin Scale, Distribution of Stiffness Index, Heightened Sensitivity Scale, and Hauser Ambulation Index, while grade 3/4 neutropenia occurred in 5/26 (19.2%); cytokine release syndrome and neurotoxicity resolved without sequelae, and immunotherapy-free remission persisted at last follow-up.
The findingWhy it mattersWhat they didThe result
AI summary of the abstract · may contain errors · not medical advice · read the paper.