Backed by $17 million in cash from California’s stem cell agency, researchers at UC Davis this month are launching “the world’s first clinical trial using stem cells to treat spina bifida before the child is born.”
The effort is the culmination of more than a decade of work by Diana Farmer, who is the world’s first female fetal surgeon, and her colleagues, including Aijun Wang, co-director of UC Davis’ surgical bioengineering laboratory.
California’s $12 billion stem cell agency this month highlighted the Davis effort in a blog item headlined: “A little history in the making by helping the tiniest patients”
Kevin McCormack, senior director of communications at the California Institute for Regenerative Medicine (CIRM), as the agency is formally known, wrote, “Spina bifida is a birth defect caused when a baby’s spinal cord fails to develop properly in the womb. In myelomeningocele, the most severe form of spina bifida, a portion of the spinal cord or nerves is exposed in a sac through an opening in the spine. Most people with myelomeningocele have changes in their brain structure, leg weakness, and bladder and bowel dysfunction.
“While surgery can help, Dr. Farmer says it is far from perfect: ‘Currently, the standard of care for our patients is fetal surgery, which, while promising, still leaves more than half of children with spina bifida unable to walk independently. There is an extraordinary need for a treatment that prevents or lessens the severity of this devastating condition. Our team has spent more than a decade working up to this point of being able to test such a promising therapy.’
“The team at UC Davis – in a CIRM-funded study – will use a stem cell “patch” that is placed over the exposed spinal cord, then surgically close the opening, hopefully allowing the stem cells to regenerate and protect the spinal cord.”
Farmer said, “A successful treatment…would relieve the tremendous emotional and economic cost burden on families. We know it initially costs approximately $532,000 per child with spina bifida. But the costs are likely several million dollars more due to ongoing treatments, not to mention all the pain and suffering, specialized childcare, and lost time for unpaid caregivers such as parents.”
The trial is scheduled to take 30 months.
Editor’s Note: David Jensen is a retired newsman who has followed the affairs of the $3 billion California stem cell agency since 2005 via his blog, the California Stem Cell Report, where this story first appeared. He has published more than 4,000 items on California stem cell matters.