Stem cell therapy performed on unborn babies 'could help reverse spina bifida brain abnormalities'

Surgeons took stem cells from donated placentas and applied them to the exposed spinal cord of babies during surgery.

Researchers are now hopeful that the procedure has the potential to alter the lifelong health of people with the condition.

Spina bifida happens when a baby's spine does not develop fully in the womb and is usually diagnosed during pregnancy or soon after birth.

There are two main types of the condition; open spina bifida and closed spina bifida.

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Open spina bifida, known as myelomeningocele and meningocele, is less common but more serious, and causes the spinal cord to push out of a gap in the spine.

Symptoms can include problems with walking or paralysis, incontinence, a loss of feeling in the legs, a curved spine, and a build up of fluid in the brain which can cause learning difficulties.

In some cases, specialists can perform fetal surgery to close open spina bifida while the baby is still in the womb.

However, researchers say this "does not fully address" the problems with mobility that can happen when the lower motor neurons - which connect the brainstem and spinal cord to skeletal muscles - are damaged.

The trial, carried out in California, included six women between 24 and 25 weeks pregnant.

Each of their babies had been diagnosed with myelomeningocele and were shown to have hindbrain herniation, which are brain abnormalities associated with spina bifida.

All six patients had fetal surgery to correct their baby's condition.

In an extra step, stem cells from donated placentas were also applied directly to the exposed spinal cord of the fetus.

The six babies were delivered between July 2021 and December 2022, and all were born with spinal repairs in tact and no signs of infection or abnormal tissue growth.

An MRI scan performed after the birth confirmed that the hindbrain herniation had been reversed.

Researchers said the study - published in The Lancet - "establishes a scalable and clinically feasible platform for the targeted delivery of biological therapeutics to the fetus".

They added: "The successful integration of cell-based therapy into fetal surgery, under stringent regulatory oversight and within a standardised surgical workflow, shows the feasibility and safety of delivering rationally designed biologics during gestation.

"By intervening at early stages of development, this approach has the potential to alter lifelong health trajectories, reducing the burden of chronic disability and minimising the long-term social and economic impact of these conditions."

The six children involved in the trial received routine care, but will also be followed-up at intervals until they are six to assess the long-term safety of the treatment.

The findings from the phase one trial have also allowed experts to proceed with the enrolment of 35 patients on another study.

Spina bifida affects about one in 1,000 births in the UK every year.

Kate Steele, chief executive of the charity Shine which supports people with Spina bifida, said: "Shine has been aware of the trial, applying stem cells to the spine prior to closure of the spina bifida lesion in utero.

"The advances in stem cell science that have enabled this first-in-human trial to be possible are remarkable, and we are delighted for the team at UC Davis School of medicine that the safety of the procedure has been demonstrated.

"Having seen the difference foetal surgical repair is making to the lives of children with myelomeningocele, we are excited to see what the next phase of this study finds. Improvements in bladder and bowel function will be particularly welcome."