In a revolutionary medical achievement, brain surgery has been successfully performed on a fetus still in the womb, potentially preventing life-threatening complications post-birth.
This historic procedure was part of an ongoing clinical trial conducted at Boston Children’s Hospital and Brigham and Women’s Hospital, under the careful oversight of the U.S. Food and Drug Administration (FDA).
The focus of the trial is Vein of Galen malformation (VOGM), a rare prenatal condition affecting fetuses. In VOGM cases, arteries carrying high-pressure blood are improperly connected to a major vein at the base of the brain, deviating from the normal fetal development where they should connect to smaller capillaries, regulating blood flow and oxygen delivery to surrounding tissue.
The groundbreaking procedure took place when the expectant mother was 34 weeks pregnant, utilizing ultrasound technology. The surgical intervention aimed to address potential damage to vessels and mitigate the risk of heart failure or stroke for the unborn child.
The unnamed baby, the first treated patient in the trial, was delivered via induced labor two days later due to a broken membrane. Although the infant spent several weeks in the neonatal intensive care unit, both mother and baby are now at home, marking a significant success in the experimental treatment.
Lead author, Professor Darren Orbach of Boston Children’s Hospital, said, “In our first treated case, we were thrilled to see that the aggressive decline usually seen after birth simply did not appear.”
Post-birth assessments revealed marked improvements in cardiac output through repeated echocardiograms. Scans exhibited normal heart and brain function, showcasing the effectiveness of the pioneering procedure.
“We are pleased to report that at six weeks, the infant is progressing remarkably well, on no medications, eating normally, gaining weight and is back home,” said Dr. Orbach in a statement.
“There are no signs of any negative effects on the brain,” he shared.
Stroke experts lauded the approach and results as “pioneering” in the American Stroke Association’s peer-reviewed journal, Stroke. Subsequent to the intervention, the premature newborn displayed exceptional outcomes, requiring neither cardiovascular support nor surgery. The infant underwent a normal neurological examination, revealing no signs of strokes, fluid accumulation, or hemorrhage in brain MRI scans.
“This approach has the potential to mark a paradigm shift in managing Vein of Galen malformation where we repair the malformation prior to birth and head off the heart failure before it occurs, rather than trying to reverse it after birth,” said Dr. Orbach.
“This may markedly reduce the risk of long-term brain damage, disability, or death among these infants,” he added.
The potentially life-saving intervention becomes particularly crucial for fetuses with VOGM, the most prevalent congenital vascular brain malformation, occurring in approximately one in every 60,000 births. Typically diagnosed through prenatal ultrasound scans during the late second or third trimester, the condition demands immediate attention to prevent severe complications post-birth.
The current standard of care for VOGM involves post-birth treatment with embolization, a catheter-based procedure closing off direct artery-to-vein connections and restricting excess blood flow to the brain and heart.
However, this method carries inherent risks and may not consistently reverse heart failure. Moreover, it might not prevent irreversible brain damage, leading to lifelong cognitive disabilities or even fatal conditions for the infant.
Experts, including Professor Gary Satou, a fetal cardiologist at the University of California, Los Angeles, who was not involved in the study, have emphasized that this type of intervention could be “very impactful” in a specific patient group.
“As always, a number of these fetal cases will need to be performed in order to establish a clear pattern of improvement in both neurologic and cardiovascular outcomes. Thus, the national clinical trial will be crucial in order to achieve adequate data and, hopefully, successful outcomes,” he said.
Professor Colin Derdeyn, a neuro-interventional radiologist at the University of Iowa, also cautiously welcomed the breakthrough, acknowledging its potential to reshape the landscape of prenatal care and treatment.
The success of this pioneering surgery opens doors to new possibilities in addressing congenital conditions and underscores the importance of advancing medical techniques to safeguard the health of unborn children.
“The key advance here is to intervene before the physiologic events of birth can cause life-threatening heart failure. There are caveats; one successful case is not enough experience for us to conclude that the risks of this procedure are worth the benefits,” said Professor Derdeyn.
“However, the positive hemodynamic changes that they observed in utero and after birth —reduction in flow, reduction in size of the draining vein, reversal of the abnormal reversed flow in the aorta—are really encouraging,” he added.
“These are some of the most exciting and surprising aspects of this case report. This is pioneering work being done in a very careful and responsible way,” he said.
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