Metabolic Disorder Treatment Through Liver Cell Transplant

Hepatocyte Transplantation for Liver-Based Metabolic Disorders

Protocol Description

This research, originating at Children’s Hospital of Pittsburgh of UPMC, is being done to determine whether a new therapy can help patients with life-threatening, liver-based metabolic diseases. Intended for patients who are unlikely to survive without extensive medical therapy or transplant, the therapy involves partial irradiation of the liver combined with transplantation of liver cells, called hepatocytes. This treatment may prove to be an effective alternative to whole liver transplantation, which, despite encouraging survival statistics, continues to be associated with significant morbidity and mortality. Among other potential benefits, hepatocyte transplantation should be less stressful to the patient than whole liver transplantation and could, theoretically, provide restorative potential for the liver.

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting patients with a life-threatening liver-based metabolic disease for whom conventional whole liver transplantation is not immediately available.
Males: Up to 21 years
Females: Up to 21 years


Initially participants will receive radiation therapy to part of their livers to prepare for hepatocyte transplant. Hepatocytes from human donors will be matched with the patient’s blood type and injected into the liver through the portal vein. Blood tests will be done before, during and after transplantation of the liver cells. Follow-up care will include regular check-ups, use of immune system suppressants to prevent organ rejection, and use of antibiotics to prevent infections.
Visits: Requires hospitalization for transplant
Duration: Follow-up is ongoing

Status: Open to Enrollment

Source(s) of Support

Children’s Hospital of Pittsburgh of UPMC

Primary Investigator

Ira J. Fox, MD

Contact Information

For information, please contact:
Rachel Novosel Sada, MS, CIP