With CIRM funds, UCD stem cell ‘fairy godmother’ leads fight against spina bifida

The California stem cell agency had a $9 million moment last week that involved a “stem cell fairy godmother,” two English bulldogs named Darla and Spanky, four lambs and a baby from Texas named Robbie.

While it took the agency only moments to hand out the $9 million, it took the fairy godmother more than 25 years to make it happen.

Meet Diana L. Farmer, the fairy godmother and the world’s first female fetal surgeon. “I take care of my patients in the womb,” she says.

Farmer does her work at the University of California, Davis, where she and her collaborator, Aijun Wang, a professor of biomedical engineering, are developing a therapy for spina bifida.

“I think of it as delivering the magic stem cell juice,” Farmer said in an award-winning, UC Davis video documenting the story of her research.

The treatment is aimed at spina bifida, a devastating and costly birth defect that can lead to lifelong cognitive, mobility, urinary and bowel disabilities. It affects 1,500 to 2,000 children in the United States each year.

Robbie, whose parents hail from Austin, Texas, could have been one of the estimated 70,000 persons living with spina bifida. But her mother, Emily, learned of Farmer’s clinical trial at UC Davis the day that ultrasound tests revealed that Robbie’s spine was not growing properly. (UC Davis did not provide Emily’s last name.)

Emily enrolled in the trial, and Robbie became the first child to be born after being treated by Farmer’s team. That was in 2021. Robbie is doing just fine nowadays and is being monitored by UC Davis. Three more children have been treated.

Last week, it took stem cell agency directors only 40 seconds to approve Farmer’s latest award after seeing that the agency’s scientific reviewers had already voted 14-0 to give the funds. Reviewers said the treatment could be “one of the most impactful CIRM projects ever” among the 1,397 awards that the agency has handed out since 2005.

While it took the agency only moments to hand out the $9 million, it took the fairy godmother more than 25 years to make it happen.

The new, $9 million award is part of $25.8 million that Farmer has received over the years from the California Institute for Regenerative Medicine (CIRM), as the stem cell agency is officially known. The latest funding will go to treat 29 additional children as part of the clinical tests needed to qualify the treatment for broader, more general use.

Because spina bifida occurs more frequently among Latinos, UC Davis plans to enroll at least 10 Latina women in the trial (30 percent or more).

Shriners Hospital for Children, which has a facility on the UC Davis hospital campus in Sacramento, added $6 million to the CIRM award.

Farmer’s “magic stem cell juice” therapy is the first ever in-utero stem cell treatment for spina bifida. UC Davis Health has detailed the story of what it calls the CurRe (Cellular Therapy for In Utero Repair of Myelomeningocele ) in a compelling Northern California Emmy-winning documentary that takes the viewer into the operating room.  The first chapter in the documentary has run up nearly 16,000 views, the last 22,000.

“The surgery is performed during pregnancy. It uses human placenta-derived mesenchymal stromal cells that are held in place with a biomaterial scaffold to form a ‘patch’ on the spine,” said UC Davis.

“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.

The English bulldogs came during an earlier stage and can be seen frolicking post-surgery in a 2017 video. (They were adopted and went off to a home in New Mexico.)

“After Emily was placed under general anesthetic, a small opening 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,” a UC Davis account of the surgery said.

“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 patch was put in place at 25 weeks and five days into Emily’s pregnancy. At 35 weeks and five days, Robbie was born through a cesarean section and weighed five pounds, 10 ounces.

Emily was thrilled. “She shouldn’t be able to move anything,” Emily said shortly after Robbie was born. “So to have hips, knees and ankles and toes and to have good strength in all of them too is pretty, pretty wild, pretty unreal.”

As for Farmer, Emily, said, “She’s kind of like (Robbie’s) stem cell fairy godmother.” Farmer responded in kind. Clinical trials involve risk, and Emily is one of the women willing to take a risk in hopes of saving her child from a terrible affliction. “What an incredible gift they give to science,” Farmer said.

The research is several years away from being available to the general public outside of a clinical trial. Trials proceed in three stages, the first involving safety, before a proposed treatment is approved by the federal government. Ninety percent of proposed “conventional” treatments entering trials do not reach the marketplace. And stem cells are not “conventional.”

CIRM, which voters created in 2004, has yet to help finance a stem cell therapy that is available to the general public. It hopes that work like Farmer’s and that of other researchers will ultimately resonate with voters when CIRM needs refinancing with more billions in about nine years or less.

Here is a link to information about enrolling in the clinical trial.

Jensen is a retired newsman and has covered CIRM for 18 years on his newsletter, the California Stem Cell Report. He authored the book, “California’s Great Stem Cell Experiment,” in 2020.