Patch up Spina bifida in womb-thanks to stem cell

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Robbie is the first born after receiving the world’s first spina bifida treatment combining surgery with stem cells.

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Stem cell application

In one-of-a-kind treatment, where the fetus is still developing in the mother’s womb, there is a chance which could improve outcomes for children with this birth defect

Robbie was diagnosed with spina bifida in the womb. Spina bifida, also known as myelomeningocele, occurs when spinal tissue fails to fuse properly during the early stages of pregnancy. Birth defects can lead to lifelong cognitive, mobility, urinary and bowel disabilities. Every year It affects 1,500 to 2,000 children in the U.S. It is often diagnosed through ultrasound. At this point, the scientists from “CuRe Trial” have come to the rescue. In one-of-a-kind treatment, where the fetus is still developing in the mother’s womb, there is a chance which could improve outcomes for children with this birth defect.

So let us first learn about the clinical trial known formally as the “CuRe Trial: Cellular Therapy for In Utero Repair of Myelomeningocele.” It was launched in the year 2021. So far three babies have been successfully born. In order to fully assess the procedure’s safety and effectiveness the babies will be monitored by the research team until 30 months of age

One of the patients Emily, who is a part of this CuRe trial endorses and feels this trial could enhance the quality of life for so many patients to come. Her daughter Robbie was born last October. “We didn’t know about spina bifida until the diagnosis. We are so thankful that we got to be a part of this. We are giving our daughter the very best chance at a bright future.”

It so happens that when surgery is performed post birth it help reduce some of the effects, but surgery pre-birth can prevent or lessen the severity of the fetus’s spinal damage, which worsens over the course of pregnancy.   

Credit goes to

Diana L. Farmer, an internationally renowned fetal and neonatal surgeon, is chair of the Department of Surgery at UC Davis Health, where she oversees more than 250 faculty, volunteer clinical faculty, post-doctoral fellows, residents, students and staff who provide highly skilled, speciality services in bariatric, burn, cardiothoracic, gastrointestinal, plastic and reconstructive, oncology, transplant, trauma and vascular surgery.

“I’ve been working toward this day for almost 25 years now,” said Diana Farmer, the world’s first woman fetal surgeon, professor and chair of surgery at UC Davis Health and principal investigator on the study. 

As a leader of the Management of Myelomeningocele Study (MOMS) clinical trial in the early 2000s, Farmer had previously helped to prove that fetal surgery reduced neurological deficits from spina bifida. Many children in that study showed improvement but still required wheelchairs or leg braces. 

How was the project carried out?

Aijun Wang a bioengineer was recruited by Farmer specifically to help take that work to the next level. As a team, they launched the UC Davis Health Surgical Bioengineering Laboratory to find ways to use stem cells and bioengineering to advance surgical effectiveness and improve outcomes. Several years ago Farmer had launched the UC Davis Fetal Care and Treatment Center with fetal surgeon Shinjiro Hirose and the UC Davis Children’s Surgery Center.

The team is working rigrously on stem cells in fetal surgery for over a decade. As the time progressed animal modeling has shown it is capable of preventing the paralysis associated with spina bifida.

It’s believed that the stem cells work to repair and restore damaged spinal tissue, beyond what surgery can accomplish alone.

Lamb Model was used

The Preliminary work by Farmer and Wang. When prenatal surgery was combined with human placenta-derived mesenchymal stromal cells, held in place with a biomaterial scaffold to form a “patch,” helped lambs with spina bifida walk without noticeable disability.

Wang expressed his gratitude by saying “When the baby sheep who received stem cells were born, they were able to stand at birth and they were able to run around almost normally. It was amazing,” Wang said.

Further the team refined their surgery and stem cells technique for canines, the treatment showed the improvement in mobility of dogs with naturally occurring spina bifida.

In their trial approach, a pair of English bulldogs named Darla and Spanky were the world’s first dogs to be successfully treated with surgery and stem cells. The hindquarters is usually affected in this breed, frequently leaves them with little function. Spina bifida, a common birth defec runs in this breed.

On keen observation these dogs post-surgery at 4 months old were able to walk, run and play.

The world’s first human trial to patch up spina bifidia

Robbie was born last October, with the surgery already being performed on her. Her parents never expected a complicated pregnancy. But the day that Emily learned that her fetus had spina bifida was also the day she first heard about the CuRe trial, it was a lifeline that they couldn’t refuse.

The Participation in the trial would mean that she would need to temporarily move to Sacramento for the fetal surgery and then for weekly follow-up visits during her pregnancy.

After screenings, MRI scans and interviews, Emily received the life-changing news that she was accepted into the trial. Her fetal surgery was scheduled for July 12, 2021, at 25 weeks and five days gestation.

Farmer and Wang’s team manufactures clinical grade stem cells — mesenchymal stem cells — from placental tissue in the UC Davis Health’s CIRM-funded Institute for Regenerative Cures. The cells are known to be among the most promising type of cells in regenerative medicine.

The lab is a Good Manufacturing Practice (GMP) Laboratory for safe use in humans. It is here that they made the stem cell patch for Emily’s fetal surgery.

“It’s a four-day process to make the stem cell patch,” said Priya Kumar, the scientist at the Center for Surgical Bioengineering in the Department of Surgery, who leads the team that creates the stem cell patches and delivers them to the operating room. “The time we pull out the cells, the time we seed on the scaffold, and the time we deliver, is all critical.”

The first successful operation in medical history

Emily was placed under general anesthetic.The surgeons used a microscope to carefully begin the repair. A small cavity was made in her uterus and they floated the fetus up to that incision point so they could expose its spine and the spina bifida defect.

Then the moment of truth: The stem cell patch was placed directly over the exposed spinal cord of the fetus. The fetal surgeons then closed the incision to allow the tissue to regenerate.

“The placement of the stem cell patch went off without a hitch. Mother and fetus did great!” Farmer said.

The team declared the first-of-its-kind surgery a success.

Birthday of Robbie

On Sept. 20, 2021, at 35 weeks and five days gestation, Robbie was born at 5 pounds, 10 ounces, 19 inches long via C-section.

“One of my first fears was that I wouldn’t be able to see her, but they brought her over to me. I got to see her toes wiggle for the first time. It was so reassuring and a little bit out of this world,” Emily said.

It was expected to be born with leg paralysis if Robbie remained untreated. “It was very clear the minute she was born that she was kicking her legs and I remember very clearly saying, ‘Oh my God, I think she’s wiggling her toes!'” said Farmer, who noted that the observation was not an official confirmation, but it was promising. “It was amazing. We kept saying, ‘Am I seeing that? Is that real?'”

For Farmer, this day is what she had long hoped for, and it came with surprises.

Both mom and baby are at home and in good health. Robbie just celebrated her first birthday.

The CuRe team is cautious about drawing conclusions and says a lot is still to be learned during this safety phase of the trial. The team will continue to monitor Robbie and the other babies in the trial until they are 6 years old, with a key checkup happening at 30 months to see if they are walking and potty training.

“This experience has been larger than life and has exceeded every expectation. I hope this trial will enhance the quality of life for so many patients to come,” Emily said. “We are honored to be part of history in the making.”

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Drafted by Nagama Nadaf
 A technophile who is crazy about technology and passionate about blogging. 
I care by sharing recent advancements in technology and try to reach out to the minds of people

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