Update: Monday, June 10, 2013
Dr. Nancy Snyderman recently visited us from NBC’s Rock Center to learn more about Omegaven, the experimental drug we’re using to treat babies suffering from liver disease. Watch her video interview with me to find out more.
Additionally, we have now treated over 100 babies, including Noah, the infant described in the video. We’re happy to report that he has made a complete recovery, and his mom has captured his Omegaven story.
One of the most dreaded complications of intestinal illness in newborn infants is damage to the liver, called cholestasis. The liver damage seems to be related, at least in part, to the need for long-term intravenous nutrition for newborn babies with severe intestinal illnesses or complications of prematurity. When severe, cholestasis can lead to the need for an intestinal transplant or lead to fatal complications.
About 4 years ago, a team at Boston Children’s Hospital discovered that giving fish oil intravenously (omega-3’s) to babies with this problem could lead to remarkable improvements in the liver disease. Some babies thought to need a liver transplant had a near complete recovery of their liver disease and the transplant was canceled. Since then, this recovery from liver disease related to intravenous nutrition has been shown to occur in many babies.
However, as of now, the use of fish oil given intravenously is not approved by the Food and Drug Administration (FDA). Doctors and hospitals need to have special permission from the FDA to use it. It can only be given to babies who have severe liver disease where it is believed the patient is severely ill and there are no other effective treatments available. This is called “compassionate use.” At Texas Children’s Hospital, we have a protocol for use of this medicine and we have provided it to over 50 babies. Our results have been excellent with most of the babies doing very well and having an almost complete recovery of their liver disease.
Noah was a very premature baby whose intestine had been damaged as a complication of his prematurity. He was unable to tolerate any food for a prolonged time period and his liver was damaged and worsening. His parents read about omega-3 fatty acids given intravenously and asked his doctors to send him to Texas Children’s for it. After coming here, we used this medication for about 8 weeks while he went through multiple surgeries and gradually was able to tolerate food. Meanwhile, his liver kept getting better and now he is at home eating a full diet and he has had almost complete healing of his liver damage.
Newborns with problems like necrotizing enterocolitis, short gut syndrome and related problems who develop liver problems may be eligible for this medicine under our compassionate use protocol. Families are welcome to ask their doctor about it, read about it on the internet looking for it under the trade name “Omegaven” (Fresenius, Germany) or contact us directly for information. We patiently await the FDA approving this medicine as an orphan drug for general use, but for now, we welcome sharing the currently available information with families or physicians.