Fetoscopic regenerative therapy for severe pulmonary hypoplasia - a feasibility pre-randomized controlled trial study

When a baby’s lungs are not adequately developed at birth, severe complications and even death can result. Dr. Ruano brings expertise in a technique that uses a minimally invasive procedure to intervene while the fetus is still in the uterus. He will participate as one investigator in a larger clinical trial to see if this procedure can regenerate the process of lung growth and development before birth.

Year One Progress Report:

Congenital diaphragmatic hernia (CDH) is a developmental defect of the developing baby’s diaphragm which causes the abdominal tissues to be pushed into the chest cavity, thereby inhibiting the ability of the lungs to grow normally. This condition occurs in 1 in 2,400 births and is associated with 70-90% mortality after birth without immediate intervention in severe forms because of abnormal lung development (small lungs, known as pulmonary hypoplasia). A new approach is Fetoscopic endoluminal tracheal occlusion (FETO), a procedure performed in utero for severe cases of CDH with the objective of promoting lung growth by regenerating lung tissue growth. During a FETO procedure with ultrasound guidance, a balloon is placed in the fetal airway causing a blockage that leads to fluid build-up in the lungs allowing them more space to develop. The balloon is removed prior to delivery with the goal of achieving better survival rates and shorter hospitalization stays after delivery compared to those that do not undergo intervention. With MN Regenerative Medicine funding, we are confirming that this procedure is safe and feasible for mothers and their children as well as training other young physicians on this procedure using plastic models. We are also confirming that FETO procedure is a clinical example of regenerative medicine therapy. Proficiency in treating CDH will advance the health of future Minnesotans and bring people in from surrounding cities to have this highly specialized procedure.

Final Report:

Congenital diaphragmatic hernia (CDH) is a developmental defect of the baby’s diaphragm which causes the abdominal tissues to be pushed into the chest cavity inhibiting the ability of the lungs to grow normally. This condition occurs in 1 in 2,400 births and is associated with 70-90% mortality after birth without immediate intervention in severe forms because the lungs are so small (known as pulmonary hypoplasia). A new approach is Fetoscopic Endoluminal Tracheal Occlusion (FETO), a procedure performed in utero for severe cases of CDH with the objective of regenerating lung tissue growth. During a FETO procedure with ultrasound guidance, a balloon is placed in the fetal airway causing a blockage that leads to the build-up of fluid in the lungs allowing themmore space to develop. The balloon is removed prior to delivery. The data demonstrates better survival rates and shorter hospitalization stays after delivery in cases that undergo intervention compared to those that do not. With MN Regenerative Medicine funding, we are confirming that this procedure is safe and feasible for mothers and their children as well as training other young physicians on this procedure simulation models. Proficiency in treating CDH will advance the health of future Minnesotans and bring people in from surrounding Midwest communities to have this highly specialized procedure.