Closer to Cures: Diana Farmer discusses clinical trials for spina bifida

/ California Institute for Regenerative Medicine (CIRM)

written by Holly Alyssa MacCormick

UC Davis Health's Diana Farmer, MD, presenting at the CIRM Independent Citizens’ Oversight Committee in March 2026.

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 aims to correct the congenital disability early using a stem cell patch. The patch is surgically attached to a baby’s spine while they are still in their mother’s womb.

Diana Farmer, MD, surgeon-in-chief at the Children’s Hospital at the University of California, Davis (UC Davis) co-leads the CuRe Trial. Dr. Farmer, also the chief of pediatric surgery at Shriners Children’s Northern California, leads the clinical trial with Aijun Wang, PhD, the co-director of UC Davis’s Center for Surgical Bioengineering.

More on spina bifida and CuRe Trial

Spina bifida is a congenital disability that affects spinal cord development and closure. In severe forms, known as myelomeningocele, a kinked bit of spinal cord or nearby nerves protrudes 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. The update was part of a presentation to the CIRM governing board during the Closer to Cures speaker series. She discussed the study’s findings, published in The Lancet in 2026, which reported preliminary results from six babies. CIRM’s support throughout all phases of the CuRe Trial helped the work progress from early preclinical research to clinical trials, making 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, uncertainty for financial support remained. The uncertainty came from a legal challenges that disrupted federal funding for stem cell research in the U.S.

“My journey with CIRM started with a phone call from a NIH program officer,” said Farmer. “They said, ‘withdraw your grant … the word fetus is in there.’ 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 moved along. California led the way.”

A devastating cost

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

“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 fell between the cracks.”

If you look at the history of the research, from the 1930s to the 1980s, no one studied it, Farmer added.

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 a grant. An NIH-sponsored clinical trial that began in 2000. (This was four years before the formation of CIRM.) That lasted 9 years and took place at 3 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.”

The trial stopped early for efficacy, which doesn’t happen often.

“That’s pretty unusual, to stop a trial early because it works,” said Farmer.

From the barn to the bedside

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

Currently, the CuRe Trial plans to enroll up to 35 patients in a phase 1/2a study. CIRM and Shriners Children’s helped pay for the trial. This study will follow the progress of the tiny participants from birth to age 6. The work will help the researchers better understand the safety and long-term effects of stem cell therapy 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 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.”

The Closer to Cures speaker series 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 committee’s meetings are public and livestreamed via the CIRM official YouTube channel.  

• Promising Approach to Curing Spina Bifida get $5.6 Million from Stem Cell Agency

• A little history in the making by helping the tiniest patients

• Stem cell stories that caught our eye: spina bifida, review of heart clinical trial

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