State stem cell agency funds clinical trial for spina bifida treatment
First human clinical trial combines surgery and cellular therapy
California’s stem cell agency (CIRM) today awarded a $9 million grant to Diana Farmer and Aijun Wang to help launch the world’s first human clinical trial using stem cells to treat spina bifida, a birth defect that occurs when the spine and spinal cord don't form properly.
Farmer, professor and chair of surgery at UC Davis Health, in collaboration with Wang, recently received approval from the U.S. Food and Drug Administration (FDA) for their groundbreaking treatment that combines fetal surgery with a special stem cell therapy.
Now, with the generous CIRM funding, the team will be able to launch their one-of-a-kind treatment in the coming months. It will be delivered while the baby is still in the mother’s womb (in utero). The complex procedure, with its unique use of a stem cell “patch,” could improve outcomes for children who are born with the severe form of spina bifida known as myelomeningocele.
Farmer and Wang will generate a type of stem cells -- mesenchymal stem cells -- from placental tissue. The cells are known to be among the most promising type of cells in regenerative medicine.
“This is truly an historic opportunity,” said Jan Nolta, director of the UC Davis Stem Cell Program and the university’s Institute for Regenerative Cures. “Drs. Farmer and Wang will be using stem cells that are known to be safe and helpful in repairing damaged tissues. We often call them the ‘paramedics’ of the body because they produce healing factors, which are crucial for treating something like spina bifida.”
Wang, an associate professor of surgery and biomedical engineering, and co-director of UC Davis’ surgical bioengineering laboratory, is a leader in developing cellular therapies that promote tissue regeneration. He and Farmer hope to successfully repair the spina bifida birth defect that occurs when the protective tissue around a baby’s developing spinal cord fails to fully close before birth.
Without treatment, an exposed spinal cord causes severe neurological damage. The resulting problems can be a range of lifelong cognitive, mobility, urinary and bowel disabilities. The birth defect affects approximately 1,500 to 2,000 children each year in the U.S.
Farmer has been working for years to gain approval and funding for a human clinical trial to address spina bifida. She launched the UC Davis Children’s Hospital Fetal Care and Treatment Center (with pediatric surgeon Shinjiro Hirose) and UC Davis Children’s Surgery Center several years ago. The new CIRM grant complements previous funding from the agency. The earlier grant enabled Farmer and Wang to manufacture and evaluate their specialized stem cells for safety and efficacy.
The UC Davis Health team is preparing to recruit pregnant women whose babies have been diagnosed with spina bifida to test the combined surgery and stem cell procedure. The clinical trial is expected to begin in early 2021.
Clinical trial participants will be treated at the centers Farmer helped establish. UC Davis Children’s Hospital and Shriners Hospitals for Children — Northern California will provide the follow-up care.