Dr. Darren Orbach, co-director of the Cerebrovascular Surgery & Interventions Center at Boston Children’s Hospital aims to transform the treatment of Vein of Galen Malformation (VOGM), a rare but life-threatening cerebrovascular disorder affecting approximately 1 in 50,0000 births.
VOGM results in excessive blood flow to the brain, leading to severe complications such as heart failure and neurological damage in newborns. The current standard of care involves high-risk, postnatal interventions that frequently yield poor outcomes, with a mortality rate nearing 40% and many survivors facing lifelong disabilities.
Dr. Orbach and his team propose a novel, in utero surgical intervention designed to mitigate these risks by treating the malformation before birth. Using ultrasound-guided techniques, surgeons will perform a minimally invasive embolization procedure, reducing abnormal blood flow while the fetus remains protected by the placenta. This approach aims to prevent the onset of heart failure and brain injury, significantly improving survival and developmental outcomes.
“In our ongoing clinical trial, we are using ultrasound-guided transuterine embolization to address the vein of Galen malformation before birth, and in our first treated case, we were thrilled to see that the aggressive decline usually seen after birth simply did not appear,” said Dr. Darren Orbach. “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.”
The study has already demonstrated remarkable success and has gained widespread media attention in news outlets such as CNN, The Washington Post, New Scientist and The Times, highlighting the global significance of this advancement in fetal medicine. The first patient, treated in March 2023, showed immediate physiological improvements, including a 40% drop in fetal cardiac output and a reduction in the brain malformation’s size. After birth, the baby remained stable, required no cardiac support or neonatal embolization, and was discharged home neurologically intact.
With the additional funding, the study will expand its patient cohort, enrolling 18 high-risk fetuses to further evaluate the efficacy and safety of this procedure. This will allow for extensive follow-up assessments, tracking the long-term neurodevelopmental outcomes of treated infants. This research offers hope to families facing a devastating diagnosis, and by intervening before birth, it sets a new and exciting standard of care for fetal medicine.
