Allogeneic Umbilical Cord-Derived Mesenchymal Stromal Cells in Severe Systemic Lupus Erythematosus: A Phase 1 Trial
Allogeneic Umbilical Cord-Derived Mesenchymal Stromal Cells in Severe Systemic Lupus Erythematosus: A Phase 1 Trial
Author or authors of report : Prof Dominique Farge, MD, Lucie Biard, PhDd, Ben Weil, EngDe, Virginie Girault, PhD, Pauline Lansiaux, PhD, Ingrid Munia, MS, Séverine Loisel, PhD, Catney Charles, BSca, Judikael Saout, PhD
Date of report : 2024-12-17
Lupus (SLE)
Fondation du Rein, ANR eCellFrance
Summary of the Main Points/Findings
Background and Objective:
This phase 1, dose-escalation trial aimed to assess the safety and preliminary efficacy of allogeneic umbilical cord-derived mesenchymal stromal cells (UC-MSCs) in patients with severe SLE. The objective was to explore a novel therapeutic approach for patients who had not responded to standard treatments. The Lancet
Methods and Findings:
Participants received a single infusion of UC-MSCs at doses of either 2 × 10⁶ cells per kg or 4 × 10⁶ cells per kg. The primary outcome was safety, while secondary outcomes included changes in disease activity and B-cell profiles. The study found that the treatment was safe and well-tolerated, with no serious adverse events reported. Preliminary efficacy data suggested improvements in disease activity and modulation of B-cell populations.The Lancet
Conclusion:
The infusion of allogeneic UC-MSCs appears to be safe in patients with severe SLE. Placebo-controlled trials are needed to confirm clinical efficacy and further explore the role of B-cell modifications in the therapeutic benefit observed.New England Journal of Medicine+4The Lancet+4The Lancet+4
