written by Holly Alyssa MacCormick

The California Institute for Regenerative Medicine (CIRM) helped fund a clinical trial that involved an experimental stem cell treatment for a frequently paralyzing form of spina bifida. Today, several of those babies are now able to walk and run unassisted.

Babies with severe spina bifida are often treated with surgery after they are born. This therapy is aimed at correcting the birth defect early via a stem cell patch that is surgically attached to a baby’s spine while they are still in their mother’s womb.

The clinical trial, called the CuRe Trial, is co-led by Diana Farmer, MD, surgeon-in-chief at the children’s hospital at the University of California, Davis (UC Davis), and chief of pediatric surgery at Shriners Children’s Northern California, and Aijun Wang, PhD, the co-director of UC Davis’s Center for Surgical Bioengineering.

Spina bifida is a birth defect that affects spinal cord development and closure. In severe forms, known as myelomeningocele, a kinked bit of spinal cord or nearby nerves is exposed through a “window” in the spine that failed to close during development. This can cause changes in brain structure, leg weakness, or bladder and bowel dysfunction.

Recently, Farmer gave updates on the progress of this research in a presentation to the CIRM governing board as part of the Closer to Cures speaker series. She discussed the study’s findings, which were published in The Lancet in 2026, and described preliminary results from six babies. She also shared how CIRM’s support during all phases of the CuRe Trial, from early preclinical research to clinical trials, helped make this experimental stem cell therapy for spina bifida a reality.

Given the recent results of the CuRe Trial, it is hard to imagine not supporting this study. But early on, financial support for the trial was uncertain because legal challenges had disrupted federal funding for stem cell research in the U.S.

“My journey with CIRM started, essentially, with a phone call from a NIH program officer almost a decade ago,” said Farmer. “They said, ‘withdraw your grant … the word fetus is in there. … good luck to you.’ And that’s how I got introduced to CIRM.”

At the time, “there was a lot of confusion…a lot of fear about stem cells,” said Farmer. “I’m happy to say that society has moved along, in no small part, I think, to the way California led the way.”

A devastating cost

Approximately four babies are born in the U.S. every day with spina bifida, and an even higher proportion of infants have the disease in lower- and middle-income countries. In the U.S., the annual cost for a child born with spina bifida is estimated to be $791,900.

“There is a huge annual cost to care for these children who often need multiple operations over the course of their lifetime,” said Farmer. “And the disease is somewhat neglected because no discipline owns it… So, it kind of fell between the cracks, and really, no work was done, if you look at the history of the research, from the 1930s to the 1980s.”

Early surgery-based treatments for spina bifida focused on closing the spine over the exposed area of the spinal cord and nerves after the baby had been born. These surgeries had limited success.

Farmer’s spina bifida studies took a different approach.

“We asked a very simple question,” said Farmer. “If we close that defect before birth … could we get an improvement in the outcome for these children?”

This question led her to submit the grant that led to an NIH-sponsored clinical trial that started in 2000 (four years before CIRM was formed), which lasted nine years and took place in three research centers in the United States.

“This was a randomized controlled trial,” said Farmer. “Parents had to pull a number out of a hat to decide whether or not they’d have surgery before birth or after birth. … But it was stopped early for efficacy. That’s pretty unusual, to stop a trial early because it works, because they think it’s unethical to not allow this therapy for other people,” said Farmer.

From the barn to the bedside

CIRM has helped fund additional phases of the CuRe Trial for over a decade, including studies in sheep, which later led to studies in English bulldogs funded by Shriners Children’s. English bulldogs are commonly born with a form of spina bifida, and several such dogs had their spinal injuries repaired by Farmer’s team at UC Davis using stem cells.

Currently, the CuRe Trial is enrolling up to 35 patients in a phase 1/2a study funded by CIRM and Shriners Children’s. This study will follow the progress of the tiny participants from birth to six-years of age, helping the researchers better understand the stem cell therapy’s safety and long-term effects on movement, bladder, and bowel function.

“All the babies have been born being able to wiggle their toes, move their ankles,” said Farmer. “We don’t [yet] know that wiggling your toes when you’re born means you’re going to walk when you’re two-and-a-half…but we are, incredibly optimistic that we might be able to get all these kids out of a wheelchair. And that would be pretty exciting.”

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Closer to Cures is a speaker series that features presentations from two CIRM-funded grantees at each of the California Institute for Regenerative Medicine’s governing board meetings, known as the Independent Citizens Oversight Committee (ICOC). The ICOC meetings and this speaker series are open to the public and are livestreamed via the CIRM official YouTube channel.