Spina bifida is a devastating and costly birth defect that causes lifelong paralysis and is one of the most common birth defects worldwide. Spina bifida affects physical, educational, social, and psychological development, and most children require many operations throughout life. A recent randomized controlled trial showed for the first time that spina bifida can be improved by surgically repairing the defect before birth. However, improvements in the distal neurological consequences (such as paralysis and bladder incontinence) were less strong. This project aims to address these problems by developing a stem cell product from the fetus's own tissue and applying these cells during surgery before birth in order to take advantage of the unique immunology of the developing fetus. In this project we will complete the development of this stem cell product and establish its preliminary safety and efficacy in appropriate animal models. Successful use of stem cell therapy for spina bifida could potentially cure this devastating disease and would introduce the concept of using a fetus's own tissue to treat existing or later disease.
Statement of Benefit to California:
Spina bifida is one of the most disabling and costly birth defects compatible with life. California’s 5-year statewide rate for spina bifida was 6.8 per 10,000 live births between 1999 and 2003—significantly higher than the Healthy People 2010 target of 3 per 10,000 births. Average total lifetime cost to California is approximately $532,000 per child. For many children, the cost may be several millions of dollars, and these figures do not account for indirect medical costs, pain and suffering, and lost time of unpaid caregivers. There is no cure for spina bifida, and interventions that mitigate the negative consequences of the disease (lower body paralysis, bowel and bladder incontinence) are urgently needed. Hope was recently provided by a clinical trial which demonstrated, for the very first time, that the paralysis associated with spina bifida might be improved by repairing the defect before birth. The great promise of fetal therapy for spina bifida is that by intervening as early as possible—in the womb—the potential exists to correct the problem in time to prevent permanent damage. This proposal presents an innovative stem cell-based prenatal therapy that may further improve and possibly cure the neurologic deficits of spina bifida. A cure for spina bifida would relieve families and society of the tremendous cost burden of the disease and would be life-changing for future children afflicted with spina bifida.