Improving Outcomes for the Rare Liver Disease Biliary Atresia with a Cancer Drug in Vietnam

Principal Investigator: Dr. Anh-Hoa Pham Nguyen​

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Disease: Biliary atresia

Research Description: Biliary atresia (BA) is a rare congenital liver disease that causes bile duct inflammation and destruction, with a higher disease burden seen in developing countries. BA is the leading cause of chronic end-stage liver disease in children and is the leading indication for liver transplantation in pediatric patients. While prompt surgical treatment by 2-3 months of age relieves biliary obstruction and reduces deaths caused by BA, most surgical patients still eventually require liver transplantation. Granulocyte-Colony Stimulating Factor (GCSF) is a biologic medicine used during cancer treatment, and previous research has been shown that GCSF can improve liver function in adults. GCSF has a long safety history and is relatively inexpensive. The research team has already completed a Phase 1 safety/dose determination study for GCSF in BA patients (partially funded by Cures Within Reach). This multi-site Phase 2b study will be conducted at 3 pediatric hospitals in Vietnam and will determine if giving BA patients GCSF following surgical treatment improves outcomes. After successful completion of this clinical trial, GCSF could be given to BA patients in developing countries and beyond to improve patients’ medical condition, nutritional status and quality of life; reduce hospital readmissions; and reduce or delay the need for liver transplantation.

Funding Partners: ReGRoW Funders

CWR funding role: Participating funder