In a first, six babies with damaged spines treated using stem cell therapy

A Phase 1 clinical trial published in The Lancet has found that combining stem cell therapy with standard fetal surgery before birth is a safe and promising approach to treating myelomeningocele, a severe form of spina bifida.

Researchers said this marked the first time live stem cells had been applied directly to a fetus’s damaged spine. The approach could potentially improve health outcomes compared with conventional fetal surgery alone.

India has one of the highest rates of spina bifida globally, with studies indicating prevalence ranging from 1.9 per 1,000 births and in some regions even higher.

Spina bifida is a congenital condition in which the spinal cord does not develop properly, leaving part of it exposed. It can lead to lifelong complications, including paralysis, difficulty walking and problems with bladder and bowel control. At present, treatment typically involves surgery during pregnancy to close the spinal opening. While this can reduce certain complications, it does not always prevent neurological damage.

In the trial, six pregnant women carrying fetuses diagnosed with spina bifida underwent standard fetal surgery. Surgeons additionally applied placenta-derived stem cells directly to the exposed spinal cord during the procedure. These cells are known to reduce inflammation, promote healing and protect nerve tissue.

All six babies, born between July 2021 and December 2022, had intact spinal repairs and showed no signs of infection, abnormal tissue growth or tumour formation. Postnatal MRI scans confirmed that hindbrain herniation, a brain abnormality commonly associated with spina bifida, was reversed in all cases, according to the study.

Researchers added that larger, long-term clinical trials were under way to further refine surgical techniques and treatment protocols.