Stem Cells for the Treatment of Spina Bifida

Dr. Diana Farmer presented on World Cord Blood Day on a World First: Using Placental Stem Cells to potentially treat Spina Bifida (in utero). Dr. Farmer from UC Davis, UC Davis is the Chief of Pediatric Surgery at Shriner’s Hospitals and Founder of the Center for Surgical Bioengineering. She is conducting the CuRE Trial (Cellular Therapy for in Utero Repair of MMC). She specializes in treating disease before birth, which is called Fetal Therapy. Conditions found in fetuses include tumors, lung lesions, airway obstruction, congenital heart disease, congenital diaphragmatic hernia, urinary obstructions and spina bifida.
Spina Bifida, scientifically known as Myelomeningocele (MMC) is the first of the non-fatal fetal birth defects. It is the second most common neurological birth defect. MMC afflicts 4 children born in the US every day and is a lifelong disability with paralysis, incontinence, and cognitive disability. With MME, the spinal cord is exposed in utero which creates an amniotic fluid toxicity. Typically, a surgery is performed after birth to close the spinal cord, however the amniotic fluid toxicity cannot be undone.
In March 2002, a randomized clinical trial with 200 women was conducted to determine if prenatal surgery to repair the spinal cord exposure would impact the children’s outcomes after birth. The need for draining extra fluid on the brain was reduced by half and there was an improved motor function at 30 months of age. This sparked more research to find a solution that could yield better results.
This led Dr. Farmer and her team to investigate placenta derived mesenchymal stromal cells (PMSCs). Their research showed that these cells were largely expandable and reach a sufficient number within the time window for in utero treatment. PMSCs secrete paracrine, which binds and activates surrounding cells to induce healing or regeneration of nearby tissues. It protects nerve cells from damage. The original research was done on two lambs from the same mother. The one who received the PMSCs was able to walk immediately after birth, whereas, the other could not bear weight on his hind legs. These results were repeatable on both lambs and bulldogs.
This lead to the creation of the CuRe Trial: Cellular therapy for in utero repair of myelomeningocele, where living cells derived from the central tissue are implanted directly on the exposed spinal cord at the time of fetal surgery. Currently, they are in a Phase 1/2a study to determine safety and preliminary efficacy. The safety part has been completed and the FDA has granted permission to move forward with preliminary efficacy.  The first baby in the trial, Robbie, is a remarkable success story. She came out kicking her legs wiggling her toes. 
Dr. Farmer is excited about the results she’s witnessed and is optimistic about the future of PMSCs use in spina bifida patients. They are currently enrolling women into the CuRE trial who have a diagnosis of spina bifida and eligible for spinal surgery. You can find out more by visiting their website.