August 7, 2018 — The following is excerpted from a story published by WGBH:
The FDA approved a drug last month that provides nutrition to children born with a scary condition. That approval is the result of 16 years of work by doctors and researchers at Children’s Hospital in Boston.
When Gib Brogan’s daughter Ellie was born, he says at first everything seemed perfect. But things changed quickly.
“Soon after she was born, early, early one morning, she got very ill. She started vomiting dark green,” Brogan said.
Ellie was rushed to the neonatal ICU, where doctors quickly realized something was very wrong.
“They told us she had short bowel syndrome. She was missing 90 percent of her small intestine and 30 percent of her large intestine,” Brogan said.
The NIH says short bowel syndrome affects about three out of every million people. Ellie had surgery to connect her intestines, but there wasn’t enough there to really work right.
“On that first day the surgeon told us that she can be fed by I.V., but there is a bit of a race that comes when you feed an infant through an I.V.,” Brogan said.
That race is to get them eating regularly as soon as possible. Because while you can feed newborns through an I.V, over an extended period of time, the fat that they’re given, which comes from soybean oil, can damage their livers, meaning they’ll likely need a transplant.
By the time Ellie was transferred to Boston Children’s Hospital, at just a few weeks old, she already had the yellowed skin and eyes of jaundice — a sign of liver damage.
“And one day we were sitting at Children’s and a doctor knocked on the door and he came in and said, ‘My name is Dr. Mark Puder. I have something I’d like to talk to you about,'” Brogan remembered.
“And I approached the family,” Puder said, “because we had developed something in the laboratory that we’d already given to some patients and had reversed that liver injury.”
“He sat down and he told us that there were a little over 20 kids that they tried using fish oil with,” Brogan said, “and that in that limited number of kids they had seen great things with their livers — that the damage had not progressed, and in some cases it had cleared up.”
The Brogans agreed to be part of the trial. And it worked.