Baby with liver disease receives Japan’s 1st ES cell transplant
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A hospital in Tokyo said it successfully transplanted liver cells derived from human embryonic stem (ES) cells to a baby with a potentially life-threatening disease, marking the first time ES cells have been used to treat human diseases in Japan.
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The National Center for Child Health and Development (NCCHD) announced on May 21 that it carried out a clinical trial to transplant the cells into a baby with a severe liver disease. The transplant was a success, and the baby’s condition is now stable, according to the NCCHD.
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The center said it was the world’s first transplant of liver cells derived from ES cells.
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The baby developed a type of urea cycle disorder called citrullinemia type 1 in October 2019. The baby was just two days old.
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The disease prevents the body from breaking down toxic ammonia due to a congenital lack of liver enzymes. An increase in the concentration of ammonia in the blood can cause permanent brain damage and may lead to death.
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Citrullinemia type 1 is an intractable hereditary disease. About one in 530,000 people develop the disease. Fewer than 100 people are estimated to have the disease in Japan.
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Treatment requires a liver transplant. But from a safety standpoint, it is difficult to transplant livers to babies until they reach the age of 3 to 5 months, when they weigh at least 6 kilograms.
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The cellular transplant was conducted as a “bridge treatment” to improve liver function until the baby became old enough to receive a new liver.
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The liver cell transplant procedure was performed when the baby was just six days old. Medical experts injected 190 million ES cell-derived liver cells into the baby’s abdomen over two days.
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The baby was discharged from the hospital after the transplant. Then, around six months after birth, the baby underwent a living liver transplant from the father.
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The administration of immunosuppressants prevented the baby’s body from rejecting the new liver, allowing the patient to be discharged from the hospital the following month.
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The NCCHD aims to transplant ES-derived liver cells to five patients by 2022 to confirm the efficacy and safety of the treatment.
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“Regenerative medicine will become a great blessing for patients with a liver disease,” said Mureo Kasahara, head of the Center for Organ Transplantation at the NCCHD.
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