WEINHEIM, Germany, Dec. 13, 2011 /PRNewswire/ -- For the first time in the Midwest, a baby with urea cycle disorder (UCD) has received an investigational liver cell therapy as part of a clinical trial sponsored by international biotechnology firm Cytonet. The liver cell transplantation performed at Children's Memorial Hospital in Chicago is the second of its kind in the United States.
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 can be an extremely difficult procedure for very young children and neonatal patients. Additionally, there is a shortage of suitable organs available for transplantation.
The clinical trial that will enroll 20 patients across 14 centers in the U.S. and Canada was approved by the U.S. Food and Drug Administration (FDA) in 2010, following an analysis of interim results of an ongoing trial in Germany involving newborns with UCD.
"We hope to improve the landscape of UCD treatment and are committed to continuing to identify patients who might benefit from this kind of therapy," says Dr. Wolfgang Rudinger, CEO and CSO of Cytonet. "A challenge is enrolling patients since UCD only affects about 400 babies born in the United States each year and diagnosis poses several challenges."
The baby treated at Children's Memorial has a form of UCD called citrullinemia. The baby received six liver cell infusions on six consecutive days in early November and is receiving follow-up care at Rady Children's Hospital-San Diego, which is a follow-up care site for the trial and is where the baby received her diagnosis. "It is fortunate this baby was referred to us and that a diagnosis was made," said Bruce Barshop, M.D., Ph.D., professor of pediatrics and co-director of the Biochemical Genetics Laboratory at the University of California, San Diego School of Medicine, a research partner of Rady Children's. "Unfortunately, because of the rarity of this disease, it's non-specific symptoms and because newborn screening panels do not catch all cases, many babies with severe forms of the disease are not diagnosed or the diagnosis comes too late."
Liver cell therapy involves collecting healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) which are then gently isolated and undergo complex processing. These cells are infused into the portal vein over six days.