Using a Cancer Drug to Treat a Rare Pediatric Liver Disease

Principal Investigator: Dr. Evan Nadler, Dr. Ai-Xuan Holterman

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Disease: Biliary Atresia

Research Description: Biliary atresia (BA) is a rare disease, occurring in approximately 1 in 10,000-20,000 live births, and is more prevalent in Asia than the US. Despite current treatment efforts, BA is the most serious and costly liver disease in infants. It is the leading cause of chronic end-stage liver disease in children, and the leading indication for liver transplantation in pediatric patients. BA is lethal, with a median survival of 10 months. Prompt surgical treatment by 2 months of age relieves biliary obstruction and improves BA mortality. Yet long-term survival after surgery without liver transplantation is low, and most surgical patients eventually require liver transplantation. Granulocyte-Colony Stimulating Factor (GCSF), a drug used in cancer treatment, has been shown to improve liver function in mice and in other adult liver conditions. These data suggest that GCSF may be used as innovative support therapy for BA, both in surgical patients and in patients who did not have the benefits of surgery. This project will test the dosing and safety of repurposed GCSF therapy in newborns and infants with BA at two clinical sites: one in Washington, DC and one in Viet Nam. Data from this project will be the basis for a future multi-national, multi-institutional trial of GCSF therapy in children.

Funding Partners: Goldman Philanthropic Partnerships

CWR funding role: Primary funder

Completed: 2019