International Stem Cell Corporation, a company specializing in the therapeutic applications of human parthenogenetic stem cells (hpSCs), today reported positive top line efficacy results from its pre-clinical in vivo liver study. The main goal of this study was to demonstrate the therapeutic equivalence of human parthenogenetic stem cell (hpSC)-derived hepatocytes to adult liver cells as a prerequisite for using such cells in the treatment of metabolic liver diseases, including Crigler-Najjar Syndrome (NYSE:CNS).
CNS is a rare inherited disorder in which bilirubin cannot be broken down by the liver. The build-up of this toxic compound can result in damage to the brain, muscles, and nerves, as well as eventually cause death. Current treatment paradigms for CNS include phototherapy and blood transfusions, but these do not treat the underlying cause of the disease. Hepatocyte transplantation has emerged as a therapeutic strategy, and has been successfully applied to treat patients with CNS, however the extremely limited availability of human livers and therefore of donated primary hepatocytes makes a stem cell based approach attractive.
The results of this efficacy study demonstrate that the hpSC-derived hepatocytes engraft in the liver of Gunn rats and perform in a similar manner to primary human hepatocytes. The Gunn rat is a well-established model of CNS and has been used extensively to study bilirubin toxicity and hepatocytes transplantation. Furthermore, the study indicates that a single intrasplenic injection of hpSC-derived hepatocytes results in a change in the plasma indirect bilirubin level equivalent to that achieved by injecting primary hepatocytes. Establishing the equivalence of hpSC-derived and donor-derived hepatocytes in their ability to metabolize bilirubin supports the thesis that hpSC-derived hepatocytes can be used therapeutically as a substitute for donated primary liver cells.
"Achieving this milestone is very encouraging," stated Dr. Andrey Semechkin, CEO and Co-chairman of ISCO. "These results suggest that hpSC-derived hepatocytes could be a well suited alternative to donated primary hepatocytes as a source of cells in clinical applications including the treatment of Crigler-Najjar Syndrome."
For more information on the company and its hpSC technology, visit internationalstemcell.com.
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