Boldly Going: Gene and Stem Cell Therapies for Fetal and Neonatal Care

October 2018

Gene and stem cell therapies offer promise for previously untreatable diseases. Recent clinical trials have shown benefits for both single gene disorders and multifactorial disease. In the perinatal period, these strategies may reduce the impacts of prematurity and birth defects. Research is already underway using gene therapy to limit intrauterine growth restriction, and several academic and pharmaceutical research operations are developing therapies for single gene disorders that may be even more beneficial when delivered in the fetal or immediate postnatal periods.  Developing the capacity to offer pregnant patients next generation gene and stem cell therapies will be a crucial component of perinatal care over the next half century, and the Boston Children’s Hospital Advanced Fetal Care Center strives to be an innovator in this regard. Implementing this vision will require a bold, multidisciplinary approach, requiring expertise and collaboration between translational researchers, manufacturing specialists, maternal-fetal physicians, fetal surgeons, and neonatologists. At the early phase of this initiative, there are several key goals: (1) Bring key players together from diverse backgrounds to begin a collaborative dialogue, (2) Identify the barriers and challenges to success (3) Develop an implementation timeline and goals for each phase of development, and (4) Establish a monitoring mechanism to ensure that patient safety is primary.