NEWS | November 21, 2018

Promising approach to curing spina bifida gets $5.6 million

Grant enables UC Davis Health team to pursue FDA approval for clinical trials

(SACRAMENTO)

The California Institute for Regenerative Medicine (CIRM) has awarded a $5.66 million grant to UC Davis Health researchers who are pursuing a new approach to repairing the birth defect that causes spina bifida.

Diana Farmer, with Aijun Wang (center) and their research team in 2015. Diana Farmer, with Aijun Wang (center) and their research team in 2015.

Led by Diana Farmer, professor and chair of surgery, and Aijun Wang, associate professor of surgery and biomedical engineering, a UC Davis research team will have the funding to continue its work with mesenchymal stem cells, taken from a donor placenta, and a form of biomaterial scaffold that is placed over the condition’s injury site in the womb. Animal modeling has shown this approach capable of repairing the defect caused by spina bifida and preventing the paralysis associated with it.

­­In spina bifida, the spinal cord doesn’t form properly and in many cases it leaves a section of it open, exposing tissues and nerves. The condition is the most common cause of lifelong paralysis and frequently leads to other serious health problems affecting the bowel and bladder. The current standard of care is surgery, which still leaves almost 60 percent of children unable to walk independently.

Three years ago, the state stem cell agency provided Farmer and Wang with an initial $2.2 million preclinical development award to advance their innovative work to help address what is a devastating condition for babies born with the disorder and the families who care for them. In 2017, the team announced that two bulldog puppies were the first “patients” to be successfully treated with their unique stem cell therapy.

“CIRM has funded this important work from its earliest stages and we are committed to working with Dr. Farmer’s team to moving this work to the stage where it can be tested in patients.” said Maria T. Millan, and CEO of CIRM, in a statement announcing the award.

This latest grant provides funding will enable the UC Davis team to perform final testing and preparations needed for FDA approval to start a human clinical trial.

Farmer, Wang and their research team have been working on their novel approach for more than 10 years, and they are excited about being able to take it to the next step.

“There were many times of frustration, many times when cell types we explored and worked with didn’t work,” Farmer said. “But it’s the patients, seeing them, talking to them and working with them, that keeps me motivated to do the science, to keep persevering.”

Farmer, Wang and their UC Davis colleagues note that if their therapy is successful it will have an economic benefit for California and beyond. Because spina bifida is a lifelong condition involving multiple surgeries, many stays in the hospital and, in some cases, the lifelong use of a wheelchair, it has enormous financial and psychological impacts on families.

“This work is so fulfilling, and we are grateful to CIRM for enabling it to continue,” added Wang, who also is co-director of the university’s Surgical Bioengineering Laboratory and a principal investigator at the Institute for Pediatric Regenerative Medicine. “Because this is a rare disease, funding can be very difficult. California’s stem cell agency has been a perfect partner in helping bring our approach, blending stem cell therapy and tissue engineering, together to help these patients and their families.”