Foetal surgeries found that brain abnormalities associated with spina bifida were reversed in every case<\/p>\n
Combining stem cell therapy with standard foetal surgery before birth is a safe and promising approach to treat a severe form of spina bifida, a new trial has found.<\/p>\n
The study, published in The Lancet, is the first time live stem cells have been used on a foetus\u2019 damaged spine, which could potentially lead to better health outcomes for babies, compared to traditional foetal surgery.<\/p>\n
Clinicians involved in the trial now aim to establish this stem cell therapy as a safe and standard option for foetal repair of spina bifida, providing new hope for families affected by this condition worldwide.<\/p>\n
The phase one clinical trial involved six pregnant women with foetuses diagnosed with myelomeningocele \u2013 a severe form of spina bifida where the spinal cord and nerves protrude through an open, unclosed section of the backbone.<\/p>\n
Developing in the first month of pregnancy, it can cause permanent neurological damage, often resulting in paralysis, loss of sensation, and issues with bladder and bowel control.<\/p>\n
The participants underwent standard foetal surgery plus an additional step where surgeons applied cells from the placenta\u2014called placenta-derived mesenchymal stem cells (PMSCs)\u2014directly onto the exposed spinal cord during the surgery. These cells are known for their ability to reduce inflammation, promote healing, and protect nerve tissue.<\/p>\n
All six babies, who were born between July 2021 and December 2022, had their spinal repairs intact and showed no signs of infection, abnormal tissue growth, or tumour formation. Post-birth MRI scans confirmed that brain abnormalities associated with spina bifida, called hindbrain herniation, were reversed in every case.<\/p>\n
\u201cThis trial establishes a scalable and clinically feasible platform for the targeted delivery of biological therapeutics to the foetus,\u201d concluded the researchers.<\/p>\n
\u201cThe successful integration of cell-based therapy into foetal surgery, under stringent regulatory oversight and within a standardised surgical workflow, shows the feasibility and safety of delivering rationally designed biologics during gestation.<\/p>\n
\u201cBy 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.\u201d<\/p>\n
The children in this trial will be carefully monitored, with regular check-ups and assessments, until they turn six. This long-term follow-up will help researchers confirm that the stem cell treatment remains safe, and improves children\u2019s mobility, health, and quality of life as they grow. So far, no serious adverse effects attributable to the stem cell treatment during the trial have been recorded.<\/p>\n
The trial was carried out in the University of California Davis Health Medical Center. Additional large-scale, long-term clinical trials are underway to further refine surgical techniques and treatment protocols. These studies aim to confirm that children who receive this therapy experience benefits at birth, improved mobility, and a better quality of life. Throughout this process, regulatory agencies will work closely with the research team to carefully monitor safety and effectiveness.<\/p>\n