SAN FRANCISCO & RALEIGH-DURHAM, N.C.--(BUSINESS WIRE)--Cytonet announced today that new research on its investigational liver cell therapy (LCT) was presented on July 27, 2014 at the 2014 World Transplant Congress (WTC) in San Francisco.
The presentation, titled “Clinical Outcome in Children with Urea Cycle Deficiencies after Liver Cell Therapy in Comparison to a Control Group,” demonstrates how investigational liver cell therapy may help temporarily stabilize pediatric patients with urea cycle disorders (UCD) as they await liver transplantation.
"Liver cell therapy seems to effectively help stabilize children until liver transplantation is possible,” said Sukru H. Emre, MD, lead author and director of the Yale-New Haven Transplantation Center. “This technique is especially promising for treating pediatric patients, for whom liver transplantation is still a very complex and difficult procedure.”
Data derived from an interim analysis suggest that liver cell therapy may be an effective tool for treating children with severe UCD until liver transplantation is able to be performed.
Liver cell therapy involves collecting healthy cells from donated livers not suitable for organ transplantation. These cells are infused into the portal vein in six sessions on six consecutive days. Cytonet currently has two ongoing multicenter clinical trials in U.S. and Canada (CCD05, formerly SELICA III), and in Germany (CCD02, formerly SELICA V), exploring the use of liver cell therapy for patients with UCD. In December 2013, Cytonet submitted a Marketing Authorization Application to the European Medicines Agency seeking approval for its liver cell therapy for the treatment of urea cycle disorders in children.
ABOUT UREA CYCLE DISORDERS
According to the National Urea Cycle Disorders Foundation (NUCDF), urea cycle disorders comprise a group of genetic disorders leading to 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 and represent a substantial cause of brain damage and death among newborns and infants.
Cytonet is an international biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, NC in the U.S. The Company develops and produces liver cell products for therapeutic purposes. 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 metabolic 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/