NIH study to test treatment for fatty liver disease in children

Guess which kids get fatty liver disease? Nutritionally abused ones.

With the launch of a new clinical trial supported by the National Institutes of Health, researchers are working to determine whether treating children diagnosed with the most severe form of fatty liver disease with a drug called cysteamine will help improve the liver.

The trial, called Cysteamine Bitartrate Delayed-Release for the Treatment of Nonalcoholic Fatty Liver Disease in Children (CyNCh),will enroll 160 boys and girls ages 8 to 17 with nonalcoholic fatty liver disease (NAFLD). The participants will receive cysteamine or placebo by mouth twice a day for a year. There are no weight cutoffs or percentiles for the children participating in CyNCh. However, more than 90 percent of the children are expected to be overweight or obese. Participants need a baseline biopsy that confirms severe NAFLD to be eligible for the study. Children with poorly managed diabetes, heart disease, and other chronic liver diseases will be excluded.

NAFLD covers a range of severity from simple liver disease without injury, called steatosis, to the more concerning nonalcoholic steatohepatitis, or NASH, which includes fat accumulation, inflammation, and liver injury. Most children with fatty liver disease are overweight and resistant to insulin, a hormone that regulates energy. The only way to distinguish NASH from other forms of fatty liver disease is with a liver biopsy.

"We did not see fatty liver disease in children until recently," said Edward Doo, M.D., NASH Clinical Research Network project scientist and director of the Liver Diseases Program at NIH's National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), which is funding the study in collaboration with Raptor Pharmaceutical of Novato, Calif., which makes the drug and will provide it to the trial. "Fatty liver disease affects about 17 percent of children in the United States. This rise in the number of children with NAFLD most likely mirrors the increase in obesity, which affects more than 16 percent of American children and teens," Dr. Doo said.

And that's no doo.