World-first stem cell therapy trial treats spina bifida before birth

2022-10-06

In a world-first clinical trial, three babies have been born after receiving stem cell treatment for spina bifida. The treatment involves administering a stem cell patch to the fetus’ spine while still developing in the womb, and early results are promising one year on.

Spina bifida is a birth defect in which the spine fails to develop properly, which can lead to weakness or paralysis of the lower limbs, cognitive issues, and urinary and bowel dysfunction. While there’s no cure, post-birth surgery can improve the symptoms in some cases.

But a new clinical trial aims to intervene earlier. Because signs of spina bifida can appear very early on in the pregnancy, there’s time to treat it while the fetus is still developing, potentially improving the outcomes. That was the goal of the Cellular Therapy for In Utero Repair of Myelomeningocele (CuRe) trial, conducted at UC Davis Health.

Patients enrolled in the trial undergo surgery midway through pregnancy, where a patch containing mesenchymal stem cells is carefully applied to the affected area of the fetus’ spine in utero. Previous studies in sheep and dogs with spina bifida have shown that the technique can prevent paralysis, helping these young animals walk without noticeable disability.

So far, three babies have been born out of the eventual 35 that will be enrolled in the CuRe trial. The first received the treatment in July 2021 at 25 and a half weeks gestation, and was born that September. If left untreated, it was expected that the baby would have been born with leg paralysis – and yet, she was seen to be kicking and wiggling her toes right away.

Over a year later and things seem to still be going well, but the team remains cautious about jumping to conclusions. The scientists will monitor the babies until they’re six years old, and there’s a particular milestone at 30 months of age to check how well they’re walking and toilet training.

New Atlas, 6 October 2022
; https://newatlas.com