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Stem Cell Treatment for Spina Bifida

Spina bifida is a congenital disability that happens when the spinal cord and spine do not develop correctly. This condition is a form of neural tube defect. The neural tube is essential for the developing fetus as it is the structure that goes on to develop into the baby’s spinal cord, brain, as well as tissues that enclose these major organs. Usually, the neural tube starts to form in the early stages of pregnancy itself and already closes by the 28th day from conception. However, in babies who have spina bifida, there is a part of the neural tube that does not close properly or fails to develop correctly. This causes problems in the proper development of the spinal cord and the other bones of the spine.(1234)

A new treatment has now successfully used stem cells to treat spina bifida and even reverse the problems caused by this condition, including paralysis in newborns. Here’s everything you need to know about stem cell treatment for spina bifida.

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Overview of Spina Bifida

Spina bifida is a congenital disability that develops when the spinal cord and bones of the spine do not form correctly. This happens because of problems with the neural tube during the early days of pregnancy. Spina bifida can be of different types, and it also ranges from mild to severe depending on the size, location, complications, and kind of neural tube defect. Surgery is usually the only early treatment available for spina bifida, but even surgical intervention is not able to completely get rid of the condition.(56)

Signs and symptoms of spina bifida also vary depending on the type of the condition and also from person to person. A person does not typically experience any symptoms of the condition in spina bifida occulta, where the spinal nerves are not involved. However, even in this type of spina bifida, you can see some symptoms on the baby’s skin just above where the spinal problem is, including a birthmark, a small dimple, or a tuft of hair.(78)

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In another type of spina bifida known as meningocele, the child experiences problems with bowel and bladder function. In myelomeningocele, which is a severe form of the disease, the spinal canal in newborns remains open at various places, especially in the middle or lower back. At birth, both the spinal cord and membranes also tend to protrude, forming a sac. Even the nerves and tissues are commonly exposed, though in some cases, they may be inside a sac and covered by skin.(910)

The exact treatment for spina bifida depends on the type of and severity of the condition. While spina bifida occulta does not require any treatment, other types of spina bifida have to be treated, and in most cases the treatment involves surgery before birth.

Can Stem Cell Therapy Be Used For Treating Spina Bifida?

In recent years, stem cell treatments have become quite popular for a variety of conditions, including spina bifida. Stem cell therapy works by introducing adult stem cells into the damaged tissue to treat various congenital disabilities in babies. The same treatment technique is also used for spina bifida injuries. In most cases, the umbilical cord of the baby with the blood cells is harvested and frozen until such a time when the cells will be extracted from this and used. However, in just October 2022 itself, US doctors successfully carried out surgery on babies with spina bifida in the womb to repair spinal defects with the use of a specially designed therapeutic stem cell patch technique.(11)

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According to the clinical trial that is currently underway at UC Davis Health in the United States, this new stem cell treatment seems to be effective at reversing the many problems caused by spina bifida in newborns, including paralysis.(12)

In the trial, three babies were administered this one of its kind treatment, and the unique part about this procedure was that it was carried out while the fetus was still in the womb. After the treatment, when the baby was delivered, there was no sign of spina bifida. The child was born kicking her legs and wiggling her toes.

This clinical trial was launched at the start of 2021, and it is formally called the CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele.(13) Myelomeningocele is a severe form of spina bifida in which the spinal canal remains partially or fully open before birth. If the spinal canal does not close completely before birth, it can cause damage to the spinal cord. Due to the spinal canal not closing, children born are usually paralyzed and are unable to regulate their bowel movements. The Centers for Disease Control and Prevention estimates that this type of spina bifida affects nearly 1500 to 2000 children in the US every year. The condition is diagnosed during pregnancy during a routine ultrasound checkup.(14)

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The current treatment for myelomeningocele is known as fetal repair, and it requires surgery to close the spinal canal opening during pregnancy itself. It was in accordance with this technique of fetal repair that the team of researchers at UC Davis developed a specialized stem cell treatment that is derived from the placenta itself and applied to the fetus during the surgery to help heal the opening. It is believed that after the stem cells are applied, they start working on repairing and restoring the damaged spinal tissue.
The early stages of the clinical trial are looking to research the safety of this type of stem cell treatment in 35 participants. The control group in this study is newborns who also undergo fetal repair surgery but without the use of stem cells. The research team has plans to continue to monitor the babies until at least six years of age, scheduling a comprehensive checkup at 30 months to check if they have started potty training and walking.

The research team has been working on this new treatment approach using stem cells during fetal repair surgery for over ten years, with work revolving around finding the suitable stem cells to use and also finding the most appropriate techniques to then use the cells in dogs and sheep. The team achieved success as when the baby sheep who were given stem cells were born, they were able to stand up at birth and were also able to run around almost normally. The researchers used human placenta-derived mesenchymal stromal cells, and a biomaterial scaffold was used to hold this in place to basically form a ‘patch’ that helped the newborn lambs with spina bifida walk almost normally and without any disability being overly apparent. After studying the treatment in sheep and dogs, the team moved on to conducting safety studies in humans.

The California Institute for Regenerative Medicine is funding the trial, and the research team has received a grant of $9 million. This clinical trial can substantially improve the treatment of spina bifida, thus enhancing the quality of life for so many babies.

Conclusion

The reason there is so much interest in the potential of stem cells in treating spina bifida is that these cells have the ability to transform into different types of cells. This is why they have the potential to help repair damaged tissues or even replace the cells that are not working well. While the initial results of this clinical trial show a lot of promise, more research and trials are needed to firmly understand the potential of stem cells in the treatment of spina bifida.

References:

  1. Mitchell, L.E., Adzick, N.S., Melchionne, J., Pasquariello, P.S., Sutton, L.N. and Whitehead, A.S., 2004. Spina bifida. The Lancet, 364(9448), pp.1885-1895.
  2. Copp, A.J., Adzick, N.S., Chitty, L.S., Fletcher, J.M., Holmbeck, G.N. and Shaw, G.M., 2015. Spina bifida. Nature reviews Disease primers, 1(1), pp.1-18.
  3. Bowman, R.M., McLone, D.G., Grant, J.A., Tomita, T. and Ito, J.A., 2001. Spina bifida outcome: a 25-year prospective. Pediatric neurosurgery, 34(3), pp.114-120.
  4. Fletcher, J.M. and Brei, T.J., 2010. Introduction: Spina bifida—A multidisciplinary perspective. Developmental disabilities research reviews, 16(1), p.1.
  5. Oakeshott, P. and Hunt, G.M., 2003. Long-term outcome in open spina bifida. British Journal of General Practice, 53(493), pp.632-636.
  6. Phillips, L.A., Burton, J.M. and Evans, S.H., 2017. Spina bifida management. Current problems in pediatric and adolescent health care, 47(7), pp.173-177.
  7. Iskandar, B.J. and Finnell, R.H., 2022. Spina Bifida. New England Journal of Medicine, 387(5), pp.444-450.
  8. Northrup, H. and Volcik, K.A., 2000. Spina bifida and other neural tube defects. Current problems in pediatrics, 30(10), pp.317-332.
  9. Juranek, J. and Salman, M.S., 2010. Anomalous development of brain structure and function in spina bifida myelomeningocele. Developmental disabilities research reviews, 16(1), pp.23-30.
  10. Sandler, A.D., 2010. Children with spina bifida: key clinical issues. Pediatric Clinics, 57(4), pp.879-892.
  11. The cure trial: Cellular therapy for in utero repair of myelomeningocele (no date) StudyPages. Available at: https://studypages.com/s/the-cure-trial-cellular-therapy-for-in-utero-repair-of-myelomeningocele-251856/?ref=gallery (Accessed: October 31, 2022).
  12. Stem cell therapy for spina bifida? (no date) Spina Bifida Resource Center. Available at: https://www.spinabifida.net/research/stem-cell-therapy-for-spina-bifida/ (Accessed: October 31, 2022).
  13. Cellular therapy for in utero repair of myelomeningocele – the cure trial – full text view (no date) Cellular Therapy for In Utero Repair of Myelomeningocele – The CuRe Trial – Full Text View – ClinicalTrials.gov. Available at: https://clinicaltrials.gov/ct2/show/NCT04652908 (Accessed: October 31, 2022).
  14. What is Spina Bifida? (2020) Centers for Disease Control and Prevention. Centers for Disease Control and Prevention. Available at: https://www.cdc.gov/ncbddd/spinabifida/facts.html (Accessed: October 31, 2022).

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