UC Davis Health leads world’s first stem cell treatment for spina bifida

The procedure marks the first time the paralysis has been successfully cured in utero

By BRANDON NGUYEN — [email protected]

For the first time in the world, UC Davis Health successfully cured paralysis and predicted neurological abnormalities associated with spina bifida for babies in utero during fetal surgery.
According to the UC Davis Health website, spina bifida, also known as myelomeningocele, is not uncommon, affecting 1,500 to 2,000 children in the United States each year. This neurological condition is diagnosed through a blood test and ultrasound scans when spinal tissue fails to fuse properly during the early stages of pregnancy, which can lead to a host of permanent disabilities related to cognitive function, mobility and movement. urinary and intestinal.

Until now, fetal surgery alone has not guaranteed recovery for these patients, but with the rise of stem cell technology, hope for a cure is much closer to reality. Dr. Diana Lee Farmer, chair of the department of surgery and a fetal surgeon trained at UC Davis Health, launched the landmark CuRe trial, which stands for cell therapy for in utero repair of myelomeningocele, in the spring of 2021 to begin recruiting patients. to test their placental stem cells during fetal surgery.

Farmer has spent more than 20 years searching for a cure for this disease, but she explained that regenerative stem cell therapy has only recently become a feasible solution.
“For spina bifida, kids don’t die, but it’s terrible,” Farmer said. “They’re born paralyzed for the rest of their lives, and there’s really nothing anyone can do at that time. They have fluid in their brains; they can’t pee or poop. They have to have special devices to facilitate urinary and bowel movements This is not fatal, but it is devastating to patients and families and the cost to society.

According to Farmer, the stem cells her team extracted came from the placenta, which she says is a cell-rich organ that constantly regenerates but is often rejected after a baby is delivered.

“We discovered that these unique placenta-derived stem cells had special characteristics,” Farmer said. “They sort of secrete what I call ‘magic stem cell juice’. In addition to protecting the spinal cord – at least in our animal models and how our bulldogs react to it – it seems to have reversed some of the damage already done, because we recovered fully in these experimental models, so we worked hard to get donor placentas, extract the cells and engineer them […] and finally got FDA approval to do the first human studies to see if it was going to be safe for humans.

Dr. Aijun Wang, a qualified bioengineer and associate professor in the Department of Surgery at the UC Davis School of Medicine, helped Farmer develop the stem cell patch for use in the surgery. Preliminary work by the two showed that prenatal surgery combined with stem cells derived from the human placenta, held in place with a biomaterial scaffold to form a “patch”, helped lambs with spina bifida walk without noticeable disability.
“When the baby sheep who received stem cells were born, they were able to stand at birth and they could run almost normally; it was amazing,” Wang said.

The team also modified the technique used on the lambs which combined surgery with stem cells for canines. When the lamb and dog models restored the animals’ ability to walk, Farmer and his team began a human trial.

One patient who participated in the CuRe trial was Michelle Johnson, who flew with her husband Jeff Maginnis from Portland, Oregon to receive placental stem cells during fetal surgery. Johnson detailed the roller coaster of emotions she felt upon hearing the diagnosis.
“I remember around the twentieth week of the gestation period, I got a call from my radiologist after my ultrasound,” Johnson said. “His voice was shaking and he told me my baby had a spinal defect called spina bifida. I hung up the phone thinking ‘what is spina bifida?’ It’s not genetic; it is very random in the presentation. I had no control over it, so why me and why my child?

Johnson said the diagnosis left her feeling like she had no options, but the CuRe trial at the time became the miracle she needed.

“I was desperate to give our baby the best possible quality of life, and that’s when I heard about the CuRe trial,” Johnson said. “We could have lost our careers at great risk when we moved to Davis, but it was worth giving our son the ability to walk. Tobi is a small miracle; he is about to crawl and he is the happiest baby in the world.
For Farmer, reversing the effects of spina bifida paved the way for a possible cure for other neurological diseases.

“We’re starting to work with team members on a variety of other neurodegenerative or neural diseases that might be related to inflammation,” Farmer said. “Magic Stem Cell Juice is truly magical. If you think about it, the placenta is a very interesting organ. It goes from nothing to being done and fully formed and basically dying in nine months. The organ makes large blood vessels and protects the fetus from being rejected. So I think there will be more potential in placenta research as well as other stem cell applications.

Written by: Brandon Nguyen — [email protected]

About Antoine L. Cassell

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