An infant with liver disease was treated with stem cells in Japan. (Illustration) - M.Libert / 20 Minutes

It is a world first that could pave the way for new options in pediatric medicine. Doctors in Japan successfully transplanted liver cells derived from embryonic stem cells into a newborn with liver disease last October.

The infant had a congenital urea cycle disorder that prevented his liver from degrading ammonia, a toxic constituent normally transformed into urea for elimination through the urine. But as young as six days old, the child was too small to have a liver transplant.

【Press Release】 Clinical trial implementation for congenital urea cycle disorder, using human ES cells
―The world's first successful transplantation of human ES cell-derived hepatocytes into humans ―https: //

- 国立 成 育 医療 研究 セ ン タ ー (@ncchd_pr) May 20, 2020

Therapy while waiting for a transplant

Doctors at the National Center for Child Health and Development (NCCHD) in Tokyo then decided to opt for transitional therapy, while waiting for the baby to grow enough to receive a classic liver transplant. They injected 190 million healthy liver cells made from human embryonic stem cells (ESF) into his liver's blood vessels.

After this treatment, "an increase in the concentration of ammonia in the blood was not noticed" in the baby, who was able to benefit a few months later from a transplant of part of his father's liver. He was released from the hospital six months after his birth.

An ethical dilemma

It is a "success" for the "first clinical trial in the world using ESCs for a patient suffering from a liver disease", welcomed the NCCHD in a press release published on Thursday. Human embryonic stem cells are harvested from fertilized eggs developed into "blastula", a cluster of about 100 cells, one of the very early stages of human life.

This promising area of ​​therapeutic research poses an ethical dilemma, however, because blastulas are destroyed in the process of extracting embryonic stem cells.


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  • Health
  • Japan
  • Medicine
  • Baby
  • Stem cells