Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study - PubMed
6 hours ago
- #stem cell therapy
- #myelomeningocele
- #fetal surgery
- The CuRe Trial evaluated the safety and feasibility of using live placenta-derived mesenchymal stem cells (PMSCs) for in-utero repair of myelomeningocele.
- Six pregnant women with fetuses diagnosed with myelomeningocele were enrolled, and the repair was performed using PMSCs seeded on an extracellular matrix.
- Primary safety endpoints included assessment of the repair site for healing, cerebrospinal fluid leak, infection, and abnormal tissue growth.
- All infants were born with intact repair sites, no cerebrospinal fluid leaks, infections, or abnormal tissue growth, and showed reversal of hindbrain herniation.
- No cell-mediated adverse events were reported, indicating the therapy was safe for proceeding to a phase 1/2a trial with 35 patients.
- The study was funded by the California Institute for Regenerative Medicine and Shriners Children's.