In an extraordinary breakthrough, researchers have conducted the world’s first-ever in-utero surgical procedure to treat a potentially lethal developmental condition by fixing an aggressive vascular malformation in a fetus’s brain. The malformation, referred to as vein of Galen malformation (VOGM), causes heart failure, severe brain damage, and, in many cases, death soon after birth. According to research published in the Stroke journal, researchers carried out the procedure by embolizing the fetus at 34 weeks and 2 days gestational age, resulting in a significant drop in abnormal blood flow and improvements in heart function. The baby, who was delivered two days later by vaginal birth due to the premature rupture of membranes during the procedure, did not require any medication or surgery to treat the malformation, nor did it require cardiovascular support following the in-utero treatment. The baby had a healthy brain with no signs of negative effects.
VOGM is a prenatal condition where the blood flow from the heart to the brain goes directly into a major collecting vein situated at the base of the brain, instead of passing through capillaries. This phenomenon results in insufficient oxygen supply to surrounding brain cells due to slow blood flow. The high flow within this malformation causes severe effects on both heart and brain during and after birth. These effects exert enormous pressure on newborns’ hearts and lungs, leading to life-threatening conditions such as pulmonary hypertension or heart failure. This malformation is rare, occurring in about one out of 60,000 births, and is usually diagnosed by MRI during the late second or third trimester of pregnancy.
The study is part of an ongoing clinical trial being conducted at Boston Children’s Hospital and Brigham and Women’s Hospital, with oversight from the US Food and Drug Administration. Embolization is the current standard of care for VOGM after birth, a catheter-based procedure to close off the direct artery-to-vein connections in the malformation and block excess blood flow to the brain and heart. However, embolization itself is high risk and not always successful at reversing heart failure, and severe brain damage may have already occurred, leading to lifelong cognitive disabilities and life-threatening conditions for the infant, or even death.
Dr. Darren B. Orbach, co-director of the Cerebrovascular Surgery & Interventions Center at Boston Children’s Hospital, lead author of the study, and associate professor of radiology at Harvard Medical School, believes that the research has the potential to change the paradigm in managing VOGM and head off heart failure before it occurs rather than trying to reverse it after birth. Although this study has only been conducted on one patient, the team is excited about the possibilities and will continue the trial to assess its safety and efficacy in other patients.