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Nadler headshotInvestigator: Dr. Evan Nadler

Disease: Biliary Atresia

Research Description: Biliary atresia (BA) is a rare disease, occurring in approximately 1 in 10,000-20,000 live births. It is the leading cause of chronic end-stage liver disease in children, and the leading indication for liver transplantation in pediatric patients. In BA, obliteration of the biliary tract and complete obstruction of bile flow leads to progressive liver fibrosis and cirrhosis. 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. The majority of surgical patients eventually require liver transplantation. Despite current treatment efforts, BA is the most serious and costly liver disease in infants. 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, and will be the basis for a future multi-national, multi-institutional trial of GCSF therapy in children.

CWR funding role: Primary funder

Start date: January 2018

Most recent report: This project is anticipated to start in January 2018. 

Anticipated next steps:

→ Data published in a scientific journal and/or presented at a scientific conference 

→ Data will be used in a larger clinical trial

→ Data leveraged into larger philanthropic or government funding for this treatment

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