Medical doctors have performed a first-ever brain surgery on a baby with a deadly genetic disorder while it is still inside the mother’s womb.
The yet-to-be-born baby was diagnosed with a vein of Galen malformation at 30 weeks and children born with this kind of condition have a 30 per cent chance of dying before age 11, Daily Mail reported on Thursday.
VGOM is a rare blood vessel abnormality inside the brain in which misshapen arteries in the brain connect directly with veins instead of capillaries, which slows blood flow, and high-pressure blood can rush into the brain, according to Boston Children’s Hospital.
The surgery which involved cutting into the womb, the baby’s skull and then operating on the developing brain was carried out by a ten-man team of doctors at the Boston Children’s Hospital and Massachusetts General Hospital, United States.
The operation requires slicing into the pregnant woman’s abdomen and using an ultrasound to identify the artery and guide the surgery.
The baby was born two days post-op with no birth defects and limited complications at 4.2 pounds, which is light for a newborn. Most babies are born full-term at 6 to 9 lbs, according to the American Pregnancy Association.
Three weeks after birth, the baby had no signs of abnormal blood flow shown in MRI scans and needed no cardiovascular assistance.
“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. There are no signs of any negative effects on the brain,” Orbach shared.
Researchers and the Food and Drug Administration (FDA) are working together to test the safety and effectiveness of the surgery through trials with the hopes of expanding use.
“As always, a number of these fetal cases will need to be performed and followed in order to establish a clear pattern of improvement in both neurologic and cardiovascular outcomes,” Gary M. Satou, M.D., FAHA, director of pediatric echocardiography at UCLA Mattel Children’s Hospital and co-director of the UCLA Fetal Cardiology Program who was not involved with the study, said. “Thus, the national clinical trial will be crucial in order to achieve adequate data and, hopefully, successful outcomes.”
“While this is only our first treated patient and it is vital that we continue the trial to assess the safety and efficacy in other patients, 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,” Orbach said. “This may markedly reduce the risk of long-term brain damage, disability or death among these infants.”
