By Steven Mew, the Australian Science Media Centre

Combining standard fetal surgery with stem cell therapy before birth could be a safe strategy to help doctors treat babies with a severe form of spina bifida, according to a small early-stage clinical trial in the US published this week in The Lancet.

Spina bifida is a condition affecting some babies before they are born in which the spinal cord does not develop properly, leaving part of it exposed. Spina bifida can have lifelong implications including paralysis, walking difficulties, and issues with bladder and bowel control.

Currently, when a baby is detected with spina bifida, surgery to repair the spinal cord can improve leg function but most children are still unable to walk, so doctors and researchers have been seeking ways to improve outcomes for babies.

In the current study, which involved six babies detected with spina bifida, doctors conducted the standard fetal surgery, and applied a special stem cell gel made from donated placenta-derived stem cells directly onto the exposed spinal cord. The trial focused on determining the safety of the procedure.

Dr Lana McClements from the University of Technology Sydney told the AusSMC that the results of the clinical trial are remarkable.

“All babies (six) delivered after this procedure were healthy, with no signs of wound problems, infection, leakage of spinal fluid, or tumour growth.”

“This new treatment, which uses the regenerative power of stem cells alongside fetal surgery, holds great promise for giving affected babies a better chance at improved mobility and quality of life”, she said.

The researchers will continue to monitor the children as they grow over the next six years, with regular check-ups to determine the effectiveness of the treatment.

However, both Dr McClements and the authors of the study acknowledge that more research involving a larger group of participants is needed to determine the true effectiveness of the stem cell treatment.

A/Prof Kuldip Sidhu from CK Cell Technologies and the University of New South Wales also criticised the paper for providing minimal data on the level of spina bifida defect in each baby, as well as data on the specific quality and quantity of the stem cells used.

“Further controlled studies are required to differentiate the effects of surgery vs stem cells alone or their derived products as exosomes, and that may shift the focus from pure fetal surgery as protective to a complete regenerative paradigm,” he told the AusSMC.

You can read the full AusSMC Expert Reaction here.

This article originally appeared in Science Deadline, a weekly newsletter from the AusSMC. You are free to republish this story, in full, with appropriate credit.

Contact: Steven Mew

Phone: +61 8 7120 8666

Email: info@smc.org.au