The clinical trial is designed to evaluate the safety and efficacy of liver cell therapy in infants to children up to age 5 with urea cycle disorders (UCD). Two centers in Canada join the 12 medical centers in the United States taking part in the trial: the Alberta Children's Hospital in Calgary and the Hospital for Sick Kids in Toronto.
UCDs are congenital and often life-threatening disorders of ammonia metabolism in the liver. Neurotoxic ammonia accumulates in the body and may lead – depending on the severity of the disease – to massive damage of the nerves and the brain and can be fatal.
For the past several years Cytonet has worked with internationally-leading metabolism and neonatal centers to study its liver cell treatment. This therapy uses healthy and metabolically functional human hepatocytes for infusion to treat the metabolism disorder. Healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) are isolated and undergo complex processing. These cells are infused into the hepatic portal vein over six days.