Human amniotic fluid stem cells show promise in treating spina bifida

Erna West, CELVI Founder and CEO

Human amniotic fluid stem cells can protect the spinal cord of a fetus from myelomeningocele (MMC).

Spinal neural tube defects (NTDs) result from failure of neural tube closure that normally occurs between 15-28 days after conception. According to Fieggen SAMJ 2014, there are no recent figures for prevalence in South Africa, but previous studies have shown an incidence of 0.77 – 6.1/1 000 live births, with higher incidences in rural areas.1

The most severe form of spina bifida, myelomeningocele (MMC), results when there is a problem with the neural tube closure before birth. It can result in a child developing significant disabilities, including partial or full paralysis, difficulty with bowel and bladder control, hydrocephalus and developmental delay.

In April 2019, BizCommunity3 reported on Africa’s first in-utero spinal surgery on a fetus with spina bifida. Dr Nicolai and his team worked with Professor Mike Belfort from the United States.  It was noted that repairing spina bifida in-utero may slow or stop the inflammatory process that leads to the damaged nerves and that, if successful, may be the only surgery required.

A study, published in STEM CELLS Translational Medicine in August 2019, provides compelling evidence of how an injection of human amniotic fluid stem cells can protect the spinal cord of a fetus from MMC.

“Despite the poor prognosis associated with MMC, the options for prenatal treatments are still limited,” said the study’s lead investigator, Daigo Ochiai, M.D., Ph.D., of the Keio University School of Medicine, Tokyo, Japan. “Recently, however, cellular therapy delivered to the fetus while in the womb has shown promise for treating birth defects.”

“This led us to investigate whether human amniotic fluid stem cells (hAFSCs) might be used to treat fetal MMC, especially since fetal MMC can be diagnosed during early stage of pregnancy and this gives us an opportunity to isolate hAFSCs from those patients and use them for in utero therapy. To the best of our knowledge, this is the first study to do this,” he added.

Pregnant rats were treated to induce fetal MMC, then injected with hAFSCs in each amniotic cavity. “Overall, we examined 116 rat fetuses with MMC,” said Dr. Ochiai. “Results showed that the hAFSCs exerted their effect on fetal MMC via two different mechanisms: by migrating to the lesions and covering the exposed spinal cords, and by secreting hepatocyte growth factor to protect neural elements and promote neural regeneration.”

Yushi Abe, M.D., also of Keio University School of Medicine, was the first author of the study. “MMC is a disease that causes many disorders, but there is no effective treatment,” he noted.

“This study shows us that in utero therapy with hAFSCs may be an innovative treatment for fetal MMC. As such, we would like to continue research for human clinical application in the future.”

“The ability to use cell therapy to treat a condition like spina bifida prenatally with minimal risk to the fetus would be a major advance in treatment,” said Anthony Atala, M.D., editor-in-chief of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. “We look forward to seeing the development of this important preclinical research.”


  1. K Fieggen et al. Aetiology and antenatal diagnosis of spina bifida. SAMJ March 2014, Vol. 104, No. 3
  2. YUSHI ABE et al. In Utero Amniotic Fluid Stem Cell Therapy Protects Against Myelomeningocele via Spinal Cord Coverage and Hepatocyte Growth Factor Secretion. STEM CELLS Translational Medicine 2019; 00:1–10.

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The treating doctor will determine the use of cord blood for treatment, depending on many factors, including the patient’s medical condition, the quality of the cord blood sample, if the patient’s own cord blood can be used or an adequately matched donor’s cord blood.The use of cord blood has been established in stem cell transplantation and has been used to treat more than 80 diseases. The use of cord blood in regenerative medicine is still being researched and there is no guarantee that treatments being studied in the laboratory, clinical trials, or other experimental treatments will be available in the future.The use of cord tissue stem cells is still in early research stages, and there is no guarantee that treatments using cord tissue stem cells will be available in the future. Cord tissue stem cells are found in the cord tissue which is stored whole. Additional processing will be required to isolate the stem cells from the tissue for use. CELVI (Pty) Ltd outsources all cord blood and tissue processing and storage activities to Next Biosciences in Midrand, South Africa, a licensed and AABB accredited facility.

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