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.
"Over a relatively short period of time her jaundice cleared and so she improved very nicely and she was not at risk for having to undergo a transplant," Puder said.
Ellie Brogan is now 12 years old. She enjoys summer camp and amusement parks. She has a permanent I.V. line that she has to be careful not to get wet. And there’s still plenty of things she can’t eat. But for the most part, Ellie’s just a normal 12 year old.
The fish oil that saved her liver, and quite possibly her life, is called Omegaven. And it hasn’t been easily available to most kids with her condition — a fact that isn't lost on Ellie.
"My parents say there's people and they, the doctors won't let them have this. And I'm like, 'Why won't they have this?'” she said. “It drives me crazy."
"Since Ellie was born we we've met dozens of kids around the country and around the world that have not had the benefit of Omegaven and we've seen them get into life threatening situations,” said her father.
Patients have been traveling to Boston Children's from around the country to get the fish oil, which the hospital provides to them for free. And the trial was opened up to other centers around the country. But it was still out of reach to many patients, because it hadn’t gotten FDA approval for use in infants, except on an experimental basis. That changed a few weeks ago, when Omegaven was approved.
"This means that other children will have access to a medication, that they don’t have to go to a research facility, they can stay local,” said Kathy Gura, a clinical pharmacist at Boston Children’s who’s been working on the drug since the beginning. “It means that that there will be less children dying of a condition that now was preventable. They'll be able to grow up."
"The short bowel families around the world are cheering this one and looking forward to more kids that are going to be saved by this," said Brogan.
Every year, Puder invites Ellie Brogan to come speak to his medical students about short bowel syndrome and the drug that changed her life.
"She comes in, skips school, comes in and teaches at Harvard," Puder said.
And, he added, Ellie always does a great job.
