Stem Cell Patch May Aid in Spina Bifida Repair in Utero

Key details

• Disabling congenital defect: Spina bifida is a birth defect which causes a baby’s spine to not fully close during development, leaving part of the spinal cord exposed. Even with surgical treatment, this condition can result in disability, impacting sufferers’ ability to walk as well as bladder and bowel control [1].
• Encouraging trial results: Results from a first-in-human phase 1 trial, called the CuRe trial, indicate that a patch of placenta-derived mesenchymal stem cells (PMSCs) is a safe addition to in-utero spina bifida repair surgery, and may provide superior results compared to surgery alone [2].
• Expanded trial enrolment: Researchers have now expanded recruitment to 35 patients in a phase 1/2 trial. Children will be followed up to age 6 to evaluate long-term safety as well as improvements in movement, bladder and bowel function[3].

What is spina bifida?

Spina bifida is a birth defect that leaves part of a baby’s spinal cord exposed and unprotected, due to the spinal column and spinal membranes, which normally protect it, not forming or closing properly. It occurs during the first four weeks of pregnancy, early in a baby’s development [1][4]. Spina bifida affects approximately 6 in 10,000 babies [5].

The severity of symptoms ranges from absent or mild to severe depending on the type of spina bifida, as well as where the opening is on the spine, with higher severity typically matching a higher position of the damage.[6] The mild type of spina bifida, called closed spina bifida or spina bifida occulta, often causes only minor symptoms such as pain or numbness, or no symptoms at all [1].

In the most severe form, called myelomeningocele, a sac of fluid containing part of the spinal cord and nerves protrudes through an opening in the baby’s back, resulting in nerve damage. This severe form is by far the most common, accounting for 75% of all cases of spina bifida.[7] The nerve damage means children with the condition are often born paralysed and unable to walk; it also can cause loss of bladder and bowel control, as well as hydrocephalus (an abnormal build-up of cerebrospinal fluid in the brain) and a type of brain abnormality called the Chari malformation, in which part of the brain pushes out from the back of the skull into the upper neck (hindbrain herniation)[2].

How is spina bifida treated?

Treatment usually involves surgery to close the opening in the spine and protect the spinal cord. This can be done either:

• during pregnancy (before birth), or
• shortly after the baby is born [1].

Research has shown that surgery during pregnancy can improve some outcomes compared with surgery after birth, although it also carries risks for both the mother and baby [8].

However, surgery does not reverse damage that has already occurred. Many children still experience long-term disability. For example, one study found that more than half of children who had surgery before birth were not walking independently at 30 months [2][9].

This means there is still a need for treatments that may improve outcomes further.

The CuRe trial for spina bifida

The CuRe trial is an early-stage clinical study designed to test whether adding stem cells to surgery before birth is safe and potentially helpful.

Researchers believe that damage in spina bifida may continue during pregnancy due to exposure of the spinal cord to surrounding fluid and movement. While surgery can help prevent further damage, it cannot repair existing injury [10].

The aim of CuRe trial was to support and protect the spinal cord during surgery.

• Choosing the right cells: Researchers first compared several types of cells before selecting placenta-derived mesenchymal stem cells (PMSCs). Compared with mesenchymal stem cells taken from adult bone marrow, PMSCs produce higher levels of substances that support nerve growth, including BDNF and HGF. In a sheep model of spina bifida, treatment with PMSCs was linked with improved movement and walking ability, suggesting they may have potential to support nerve repair in the future.[11]
• Application method: In this early clinical trial, PMSCs taken from donated placentas were placed onto a special biological “scaffold” that helps support and guide cell growth. This scaffold was already approved for use in people, making it suitable for the procedure. The finished stem cell patch was then trimmed to the right size and applied directly to the exposed spinal cord during surgery on the baby before birth to repair spina bifida.
• Trial size: Between 2021 and 2022, six patients were sequentially enrolled into the study, with each subsequent patient only being enrolled after the previous child was born and had been assessed for any complications.[2]

What were the results of the CuRe trial?

Key findings from the early CuRe trial, published in The Lancet, suggest the treatment was safe and well tolerated in this small group of babies [2].

• Safe use: The stem cell patch was successfully applied in all six cases, with no complications or need for emergency delivery.
• Good healing: All babies had fully healed surgical sites at birth, with no signs of infection, wound opening, or fluid leakage.
• No abnormal growth: Tests and scans found no evidence of tumours or unusual tissue growth.
• Positive brain changes: Before surgery, all babies showed signs of brain changes linked to spina bifida. After birth, these changes had resolved, and none of the babies needed treatment for fluid build-up in the brain (hydrocephalus).

What are the next steps?

Although it is still too early to know for certain whether the therapy is effective, particularly as positive results in small studies don’t always translate to benefits in larger cohorts, children who took part in the trial are able to walk, and researchers are cautiously optimistic [12][13].

The study is now expanding to include 35 patients to better assess long-term safety and to gather early information on how well the treatment may work. Families will be followed closely, with clinic visits at 3, 12, and 30 months of age, along with regular phone or video check-ins between visits. After 30 months, children will continue to be monitored once a year until they reach 6 years old [2][3].

If larger studies confirm the benefits, this approach could represent an important step forward in foetal surgery [10]. The research also adds to growing evidence that placenta-derived cells may have useful regenerative properties.

To learn more about the placenta and other sources of stem cells collected at birth, and how they may be stored for possible future use, you can request a free guide for more information.

References

[1] NHS. Spina Bifida. https://www.nhs.uk/conditions/spina-bifida/

[2] Farmer DL, Kumar P, Reynolds E, Lee SY, Powne AB, Pivetti CD, Zwienenberg M, McLennan AS, Nolta JA, Brown EG, Saadai P, Hirose S, Wang A. Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. Lancet. 2026 Feb 28;407(10531):867-875. doi: 10.1016/S0140-6736(25)02466-3. PMID: 41763744.

[3] The CuRe Trial: The First Stem Cell Therapy for Spina Bifida (Myelomeningocele). https://studypages.com/s/the-cure-trial-the-first-stem-cell-therapy-for-spina-bifida-myelomeningocele-251856/

[4] NICHD. About Spina Bifida. https://www.nichd.nih.gov/health/topics/spinabifida/conditioninfo

[5] Spina bifida: information for parents. GOV.UK. https://www.gov.uk/government/publications/spina-bifida-information-for-parents/spina-bifida-information-for-parents

[6] Cleveland Clinic. Myelomeningocele: What it is, causes, diagnosis & treatment. https://my.clevelandclinic.org/health/diseases/22813-myelomeningocele

[7] Myelomeningocele. Great Ormond Street Hospital. https://www.gosh.nhs.uk/conditions-and-treatments/conditions-we-treat/myelomeningocele/

[8] Adzick NS, Thom EA, Spong CY, Brock JW 3rd, Burrows PK, Johnson MP, Howell LJ, Farrell JA, Dabrowiak ME, Sutton LN, Gupta N, Tulipan NB, D’Alton ME, Farmer DL; MOMS Investigators. A randomized trial of prenatal versus postnatal repair of myelomeningocele. N Engl J Med. 2011 Mar 17;364(11):993-1004. doi: 10.1056/NEJMoa1014379. Epub 2011 Feb 9. PMID: 21306277; PMCID: PMC3770179.

[9] Farmer DL, Thom EA, Brock JW 3rd, Burrows PK, Johnson MP, Howell LJ, Farrell JA, Gupta N, Adzick NS; Management of Myelomeningocele Study Investigators. The Management of Myelomeningocele Study: full cohort 30-month pediatric outcomes. Am J Obstet Gynecol. 2018 Feb;218(2):256.e1-256.e13. doi: 10.1016/j.ajog.2017.12.001. Epub 2017 Dec 12. PMID: 29246577; PMCID: PMC7737375.

[10] Cortes MS. First steps of a potential new era in fetal intervention for spina bifida. Lancet. 2026 Feb 28;407(10531):827-829. doi: 10.1016/S0140-6736(26)00029-2. PMID: 41763729.

[11] Wang A, Brown EG, Lankford L, Keller BA, Pivetti CD, Sitkin NA, Beattie MS, Bresnahan JC, Farmer DL. Placental mesenchymal stromal cells rescue ambulation in ovine myelomeningocele. Stem Cells Transl Med. 2015 Jun;4(6):659-69. doi: 10.5966/sctm.2014-0296. Epub 2015 Apr 24. PMID: 25911465; PMCID: PMC4449103.

[12] Fieldhouse R. World-first stem-cell therapy shows promise for treating spina bifida in the womb. Nature. 2026 Feb 26. doi: 10.1038/d41586-026-00602-z. Epub ahead of print. PMID: 41748891.

[13] Moore T. “Miracle” boy expected to be paralysed due to spina bifida able to walk after ground-breaking surgery in the womb. Sky News. https://news.sky.com/story/miracle-boy-expected-to-be-paralysed-due-to-spina-bifida-able-to-walk-after-ground-breaking-surgery-in-the-womb-13512709