Landmark Phase 1 Trial in The Lancet Confirms Safety of First In-Utero Stem Cell Therapy for Fetal Spina Bifida Repair

The Lancet publishes the first successful use of live stem cells during fetal surgery for spina bifida. Learn how placental stem cells are improving infant outcomes.

By: AXL Media

Published: Feb 27, 2026, 4:10 AM EST

Source: The information in this article was sourced from The Lancet

A Major Milestone in Prenatal Regenerative Medicine

A groundbreaking study published in The Lancet has reached a major milestone in the treatment of congenital birth defects. For the first time, researchers have successfully used live stem cell therapy on a fetus's damaged spine to treat myelomeningocele—the most severe form of spina bifida. This phase 1 trial, known as the CuRe Trial, demonstrates that combining cellular therapy with standard in-utero surgery is a safe approach that could potentially offer superior neurological outcomes compared to traditional surgical methods alone.

The Limitations of Current Spina Bifida Treatment

Spina bifida occurs when the spinal cord fails to develop properly, leaving delicate nerve tissue exposed to amniotic fluid. This exposure often leads to permanent paralysis, bowel and bladder dysfunction, and significant mobility challenges. While standard fetal surgery to close the spinal opening during pregnancy has been available for years, it frequently fails to prevent all neurological damage. The addition of stem cells aims to go beyond a simple "closure" by actively promoting the healing and protection of the exposed nerve tissue before birth.

Utilizing the Power of Placental Stem Cells

In this trial, surgeons performed standard fetal surgery on six pregnant women but added a critical extra step: the application of placenta-derived mesenchymal stem cells (PMSCs) directly onto the exposed spinal cord. These specific cells were selected for their unique ability to secrete growth factors that reduce inflammation and encourage the regeneration of damaged nerves. The study hypothesized that by treating the injury while the fetus is still developing, the "government-created danger" of amniotic fluid exposure could be mitigated more effectively.