News Releases & Research Results Clinical trial with human ES cells for congenital urea cycle disorder - World's first transplantation of human ES cell-derived hepatocytes into humans!

News Releases & Research Results


The results of research conducted by Executive Director Mureo Kasahara, Deputy Director Seisuke Sakamoto, and Medical Director Akinari Fukuda of the Organ Transplantation Center, Medical Director Reiko Horikawa of the Division of Endocrinology and Metabolism, Director Akihiro Umezawa of the Center for Regenerative Medicine of the National Center for Child Health and Development.

The key results of research are as follows:

  • Human embryonic stem (ES) cell-derived hepatocytes (HAES) prepared by the National Center for Child Health and Development were successfully transplanted into a 6-day-old newborn with urea cycle disorder, who could not decompose toxic ammonia, for the first time in the world (as a "bridging treatment" until 3-5 months of age before liver transplantation).
  • Subsequent liver transplantation was completed safely probably because HAES transplantation suppressed the increase in a blood ammonia level in the patient.
  • The method of treatment developed by this research project should have additional effects on the conventional treatment by preventing hyperammonemia attacks for a waiting period before liver transplantation performed as radical treatment.

This research project was conducted with the support of the Research Center Network for Realization of Regenerative Medicine and the Research Project for Practical Applications of Regenerative Medicine by AMED.


Last updated 05/21/20