Disease Fact Sheet: Liver Disease
Clinical Status: Several cell-based clinical studies have been conducted for various types of liver disease, including acute and metabolic liver disease, as well as liver cirrhosis. Encouraging clinical results have emerged from hepatocyte transplantation for metabolic liver diseases such as Crigler Najjar, glycogen storage disease type 1, and urea cycle disorders. Currently, there is no clear evidence supporting the efficacy of cell-based therapies for liver cirrhosis. Therefore, the use of cell-based therapies for liver cirrhosis cannot be recommended outside properly funded clinical trials. There have been encouraging results from studies that have sought to grow liver cells in the laboratory from various sources including pluripotent stem cells (iPSC) and adult stem cells taken from healthy human livers. Recent research suggests that human hepatocytes and biliary cells can be expanded as organoids, potential allowing them to be used as cell therapies for treating metabolic and acute liver disease, as well as serving as a cell source for treating biliary disease. Cell-based approaches focused on modulating the liver’s innate immunoregulatory microenvironment have also gained attention. A recent first-in human Phase I trial of autologous macrophage peripheral infusion into cirrhotic patients has been performed and shown to meet its primary outcome of safety and feasibility, and a Phase II randomized controlled multi-center trial is ongoing9 . A Phase I safety study, Macrophage Therapy for Acute Liver Injury (MAIL), is currently underway to investigate the use of allogeneic macrophages from frozen stocks for treating acute liver injury.