Fetal Neuroprotection and Neuroplasticity Program

Better diagnostic capabilities, surgical advances and specialized long-term care have greatly improved survival rates for children with congenital heart disease (CHD), congenital diaphragmatic hernia (CDH) and other anomalies associated with pulmonary hypoplasia such as giant omphalocele (GO). However, with improved early outcomes, we’re learning that children with these conditions have a high risk of neurodevelopmental disabilities as they grow up.

Building on the growing evidence of the interaction between heart disease and brain development during the prenatal period (see the American Heart Association statement), Children’s Hospital of Philadelphia has established the Fetal Neuroprotection and Neuroplasticity Program to investigate innovative prenatal therapies to protect fetal brain development and prevent brain injury as early as possible before birth.

A joint effort of the Hospital’s Cardiac Center, the Fetal Heart Program, the Center for Fetal Diagnosis and Treatment, and the Division of Neurology, the Fetal Neuroprotection and Neuroplasticity Program is dedicated to improving the long-term quality of life for patients with CHD, CDH, GO and other congenital anomalies by better understanding the link between these anomalies and brain development in utero, and developing new treatment options that can be administered before birth.

Protecting the brain before birth

In the United States, approximately 1 in every 120 newborns is diagnosed with congenital heart disease, making it the most common birth defect. Many of these newborns will require either corrective or palliative open heart surgery. As recently as the 1960s, only 20 percent of newborns with critical CHD survived to adulthood. Today, thanks to better diagnostic capabilities, including prenatal diagnosis, advances in surgery, and improved postoperative care, survival is more than 90 percent.

Despite successful surgical treatments and improved early outcomes, many children will have long-term problems, such as attention problems, learning difficulties, problems with gross and fine motor skills, speech and language difficulties, social interaction problems, and problems with vision and hearing. Neurodevelopmental disability is now recognized as the most common complication of critical CHD patients, and has the most negative impact on quality of life, academic performance and opportunity for independence as an adult.

Most studies of treatments to prevent brain injury in children with CHD have focused on preventing injury after birth. However, there is convincing evidence that risk for brain injury begins even before birth.

This fetal neurodevelopment research, much of which was developed at CHOP, shows that in utero brain development is abnormal in fetuses with CHD, leading to delayed maturation, poor growth and increased risk for brain injury. These findings suggest that in order to prevent brain injury and improve outcomes, treatment to protect the brain must begin before birth.

In addition, there is increasing evidence that fetal brain injury and altered development are not limited to children with CHD. Infants with congenital diaphragmatic hernia (CDH) and other anomalies associated with pulmonary hypoplasia such as giant omphalocele (GO) have been found to have very similar brain developmental delays, suggesting that conditions that alter blood flow to the fetal brain may also put babies at increased risk.

If prenatal neuroprotective approaches are proved to benefit patients with CHD, similar strategies might be used to treat other fetuses at risk for abnormal brain development and brain injury.

The focus of the Fetal Neuroprotection and Neuroplasticity Program is to:

  1. Investigate the underlying causes of abnormal brain development in fetuses with CHD and other congenital anomalies.
  2. Conduct clinical trials to investigate potential new prenatal therapies that may prevent or improve the associated brain growth impairment and injury, with the goal of improving neurodevelopmental outcomes.

Our research

The Fetal Neuroprotection and Neuroplasticity Program will conduct pilot clinical trials of fetal interventions to learn whether new prenatal treatments may reduce brain injury and improve neurodevelopmental outcomes in newborns with CHD and other congenital anomalies.

Participation is voluntary, and each study will also have specific criteria.

For more information about studies currently enrolling:

  • Call 267-844-1155
  • Questions can also be directed to 1-800-IN-UTERO (1-800-468-8376)

Next Steps