Cytonet initiates clinical trial using liver cell infusion to treat urea cycle disorders in children

International biotechnology firm Cytonet recently launched the first ever clinical trial in the United States using an investigational liver cell infusion to treat urea cycle disorders (UCD) in children. The first patient in the Phase II trial, which will seek to enroll 20 patients in 12 medical centers, is a child with citrullinemia, which is a hereditary UCD.  He received six liver cell infusions on six consecutive days at Yale School of Medicine in New Haven, Conn.

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. Children who remain untreated rarely experience normal physical and mental development. The only cure is liver transplantation, which is an extremely difficult procedure for very young children and neonatal patients. Additionally, there is a shortage of suitable organs available for transplantation.

"Liver cell infusion may provide a new treatment option for children," said Sukru H.  Emre, MD, the lead investigator and Section Chief, Transplantation Surgery at Yale-New Haven Transplant Center.  "We are looking forward to conducting this clinical trial."  

The clinical trial, called SELICA III, is designed to evaluate the safety and efficacy of liver cell therapy in infants to children up to age 5 with UCD.  The Investigational New Drug Application (IND) was submitted to the U.S. Food and Drug Administration (FDA) in mid-2010, following an analysis of interim results of an ongoing trial in Germany involving newborns with UCD.  Cytonet received an Orphan Drug designation from the FDA's Office of Orphan Products Development.

Liver cell therapy involves collecting healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) which are then isolated and undergo complex processing.  These cells are infused into the hepatic portal vein over six days.  Risks include portal vein thrombosis and systemic shunting of liver cells.

"We are excited to help advance the landscape of UCD treatment through this milestone and are committed to continuing to identify patients who might benefit from this kind of therapy and find success," says Dr. Wolfgang Rudinger, CEO of Cytonet Germany.