A team of medical scientists in Japan successfully transplanted liver cells derived from embryonic stem cells into a newborn baby with a liver condition.
This achievement is a scientific first worldwide, and its success represents new treatment options for babies born with serious illnesses.
Liver cells from embryonic stem cells
The newborn was diagnosed with a urea cycle disorder, a condition in which the liver is unable to break down toxic ammonia, leading to its accumulation in the body.
But at six days old, he was too young to have a liver transplant. This procedure is not considered safe until a child weighs about six kilograms, a weight that reaches between three and five months of age.
In view of this huge hurdle, doctors at the National Center for Child Health and Development decided to take a risk and try a different treatment, a “bridge treatment” that the baby could live to be old enough for surgery.
They then injected 190 million embryonic stem cell-derived liver cells (ES cells) through the umbilical cord blood vessels to the baby’s liver.
Successful liver transplant
And the positive result: the level of ammonia in the blood fell to normal levels. Nine weeks later, the baby was able to leave the hospital to wait to meet the conditions for the next treatment, that is, a liver transplant.
“The patient did not see an increase in the concentration of ammonia in the blood and was able to successfully complete the following treatment,” said the National Center for Child Health and Development in a press release.
For the procedure, the baby received a liver transplant from his father in March this year, and was released from the hospital six months after his birth. And the latest reports indicate that he is in good health.
A viable treatment option
The institute also noted that in Europe and the United States, liver cells are available after being removed from brain-dead donors. But these supplies are more limited in Japan, which increases the difficulties in managing diseases like these in newborns while they wait to grow a little more to be suitable for a transplant.
“The success of this trial demonstrates safety in the world’s first clinical trial using human ES cells for patients with liver disease,” said the institute.
In addition, they plan to carry out three three more cell transplants before the end of the current fiscal year in order to obtain more evidence to establish it as a viable treatment, according to the Japanese portal NHK.