Medics in Japan have successfully transplanted liver cells derived from embryonic stem cells into a newborn baby, in a world first that could provide new treatment options for infants.
The newborn was suffering from urea cycle disorder, where the liver is not capable of breaking down toxic ammonia, but the six-day-old was too small to undergo a liver transplant, generally not considered safe until a child weighs about 6kg.
Medics at the National Center for Child Health and Development decided to try a “bridge treatment” until the baby was big enough, injecting 190 million liver cells derived from embryonic stem (ES) cells into the blood vessels of the baby’s liver.
Following the treatment, “the patient did not see an increase in blood ammonia concentration and was able to successfully complete the next treatment,” namely a liver transplant, the center said in a statement.
The baby received a liver transplant from its father and was discharged from hospital six months after birth.
“The success of this trial demonstrates safety in the world’s first clinical trial using human ES cells for patients with liver disease,” the statement said.
It noted that in Europe and the US, liver cells are often available after being removed from brain-dead donors, but the supply in Japan is more limited.
That has created difficulties in managing the health of small children as they wait to grow big enough for liver transplants.
ES cells are harvested from fertilized eggs and using them has raised ethical issues because the embryos are subsequently destroyed.
The center works with eggs whose use has been approved by both donors having already completed fertility treatment, it said.
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