NIH Study to Test Treatment for Fatty Liver Disease in Children

Results from a small pilot study using cysteamine in 11 children with NASH suggest that it improves liver enzymes by reducing toxins that can damage the liver.
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BIO publishes quarterly newsletters for its members which focus on the therapeutic areas of member companies. The newsletters include updates from biotech stakeholders around Washington, including Congress, FDA, NIH, and patient organizations. The following is an excerpt from the "Focus on Nephrology/Endocrinology/Metabolism/
Gastroenterology" newsletter. To find funding opportunities and learn about what Congress and the federal agencies are doing for biotechnology, please click here to read the “Focus on Nephrology/Endocrinology/Metabolism/Gastroenterology" newsletter.

With the launch of a new clinical trial supported by NIH, 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. More than 90% of the children are expected to be overweight or obese. Participants need a baseline biopsy that confirms severe NAFLD to be eligible. Children with poorly managed diabetes, heart disease, & 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. 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 NIDDK. “Fatty liver disease affects about 17% of children in the U.S. This rise in the number of children with NAFLD most likely mirrors the increase in obesity, which affects more than 16% of American children and teens,” Dr. Doo said.

Results from a small pilot study using cysteamine in 11 children with NASH suggest that it improves liver enzymes by reducing toxins that can damage the liver. Cysteamine is approved to treat cystinosis, a genetic disease that causes the amino acid cystine to accumulate in the kidneys, liver, eyes, brain, and white blood cells. Modest weight loss through diet and physical activity may help some children with fatty liver disease, but it is a treatment option that seldom helps people meet their goals. “We know that following a weight loss plan for many children and adults can be daunting, especially if they have limited access to healthy food options that are low in fat, added sugars, and calories, and infrequent opportunities for physical activity,” said Dr. Joel E. Lavine. “Hopefully, this trial will move us closer to finding a safe and effective treatment that helps children with fatty liver disease.”

NAFLD can be a precursor to NASH, which may progress to cirrhosis, liver failure and liver cancer. NAFLD may also increase a patient’s risk of developing heart disease. “We are concerned that the disease may advance as children become adults and increase their risk for cirrhosis, liver failure, liver transplantation, and death as adults,” said Dr. Stephen P. James. “This multicenter, double-blind trial offers researchers and NIDDK an opportunity to rigorously assess how safe and effective cysteamine is in treating children with NASH, as well as to reveal new avenues worthy of scientific study.”

For more information on this study, please click here.

For more information, please click here to read the “Focus on Nephrology/Endocrinology/Metabolism/Gastroenterology” newsletter.

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