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Saturday, September 25, 2021

UC Davis Medical Center launches stem cell clinical trial for spina bifida treatment

Fetal Surgery Department and researchers collaborate to combine surgery and placental stem cell therapy

A UC Davis Health research team, in collaboration with the Fetal Surgery Department at the medical campus, recently announced the first-ever stem cell clinical trial to treat in-utero patients with a birth defect called spina bifida. 

Dr. Aijun Wang, a trained bioengineer and an associate professor of the department of surgery for the UC Davis School of Medicine, describes the birth defect, the focus of a bioengineered therapy project he has been working on for over a decade.

“Spina bifida is a birth defect, specifically a spinal cord injury that happens early in gestation,” Wang said. “During normal fetal development the spinal neural tube folds early in gestation to cover the spinal cord and protect it. But spina bifida causes the exposure of a part of the spinal cord, usually in the lumbar area. The most severe form of spina bifida, called myelomeningocele, causes an extrusion of the spinal cord during gestation and even after birth.” 

For most patients, the babies are born paralyzed and rendered unable to walk. The current standard treatment of care for babies detected with spina bifida had been solely fetal surgery. However, the surgery alone did not serve as a guaranteed cure.

In close collaboration with Wang on this project, Dr. Diana Farmer, the chair for the department of surgery as well and a trained fetal surgeon, is the principal investigator of the clinical trial and describes the significance of the stem cells to be applied for the first time on fetal patients. 

“We use placental stem cells because we think and have been able to show that they have unique and special properties compared to bone marrow-derived stem cells and other stem cells,” Farmer said. “They are specially cultured and engineered to secrete growth factors important to nerve growth and development.”

Dr. Jan Nolta, the director of the stem cell program at the UC Davis gene therapy center, describes her role in aiding Farmer and Wang’s clinical trial.

“The stem cells come from the placenta of different qualified donors, whom we have tested their blood, screened and made sure they do not have any viruses,” Nolta said. “Here at UC Davis, we are very lucky to have a Good Manufacturing Practice facility, where we can take a small amount of those placental stem cells and grow them into a big batch of stem cells. That also gets qualified, tested, cleaned and purified from which we thaw out a small amount from that batch for fetal surgery.” 

Farmer further highlighted the properties of these stem cells and how exactly they participate in the healing of the fetus.

“The placental stem cells have regenerative and antiinflammatory properties that help reverse apoptosis, cell damage,” Farmer said. “If you think about it, the fetus is constantly making new cells–eyeballs, fingernails, heart cells, skin and everything in nine months. We think the fetus is the perfect target for this particular regenerative stem cell therapy because the fetus is always regenerating itself.”

The preparation of this project, through previous animal studies that both Farmer and Wang have conducted and through analysis and qualification of these placental stem cells will continue to pave the way for future stem cell research. 

“We hope that we can cure spina bifida—that these children will walk,” Farmer said. “That is what we saw in our animal studies, and we worked with the veterinary school to treat dogs born with spina bifida and we saw improvement there as well. So we are very optimistic, hoping that every win in the stem cell therapy arena will advance other stem cell research and therapy for other diseases. I think it is a very promising field for future scientific work.”

Wang further highlighted the optimistic future for stem cell therapy and what he hopes people will gain from this pivotal stem cell clinical trial. 

“This is a very important study, being able to apply this lab-grown technology from the bench to the bedside,” Wang said. “Spina bifida is, relatively speaking, a rare condition. But the paralysis associated with this disease is more like many other conditions that can cause spinal cord injury. If we can apply this to neuron injuries and damages, it could have a pretty broad impact on many patients actually, including those with neurological degenerative diseases.”

Written by: Brandon Nguyenscience@theaggie.org

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