Midwest’s First Liver Cell Transplant for Baby with Life-Threatening Urea Cycle Disorder Performed at Children’s Memorial
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. Risks include portal thrombosis and systemic shunting of liver cells.
“We are pleased to be participating in this trial because it has the potential to provide a treatment option for children with a rare and complicated diagnosis for which there are few options now,” said Barbara K. Burton, MD, a pediatrician and director of the PKU program at Children’s Memorial.
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. For further information, please visit Cytonet’s website at http://www.cytonetllc.com/
About Urea Cycle Disorders
According to the National Urea Cycle Disorders Foundation (NUCDF), a urea cycle disorder is a genetic disorder caused by a deficiency of one of the six enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. These include carbamoyl phosphate synthetase I (CPS I) deficiency, N-acetylglutamate synthetase (NAGS) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase (ASS) deficiency (which is also known as citrullinemia), argininosuccinate lyase (ASL) deficiency and arginase 1 deficiency (hyperargininemia). The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea. Normally, the urea is transferred into the urine and removed from the body. In urea cycle disorders, the nitrogen accumulates in the form of ammonia, a highly toxic substance, and is not removed from the body resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it causes irreversible brain damage, coma and/or death.
Urea cycle disorders are included in the category of inborn errors of metabolism. Inborn errors of metabolism represent a substantial cause of brain damage and death among newborns and infants. Because many cases of urea cycle disorders remain undiagnosed and/or infants born with the disorders die without a definitive diagnosis, the exact incidence of these cases is unknown and underestimated. It is believed that up to 20 percent of Sudden Infant Death Syndrome cases may be attributed to an undiagnosed inborn error of metabolism such a urea cycle disorder. Experts estimate the incidence of the disorders at 1 in 10,000 births. This represents a significant increase in case identification and diagnosis in the last few years. Research studies have now been initiated to more accurately determine the incidence and prevalence of UCDs. For more information, please visit NUCDF at www.nucdf.org
Cytonet is an internationally active biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, NC in the U.S. The company develops and produces cell therapeutic products. Cytonet’s goal is to provide alternatives to existing therapies for many diseases with a particular emphasis on liver diseases. Cytonet is a pioneer and leader in the field of regenerative medicine. For the past several years, Cytonet has worked with internationally-leading metabolism and neonatal centers to study its liver cell therapy, which uses healthy and metabolically functional human liver cells collected from donated livers not suitable for transplant for infusion to treat urea cycle disorders in children. For more information, please visit Cytonet’s website at http://www.cytonetllc.com/
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