FDA Approves Innovative Liver Cell Therapy for Clinical Trial in the U.S.

WEINHEIM, GERMANY–(Marketwire – August 10, 2010) – On July 23, 2010 the liver cell preparation by Cytonet GmbH & Co. KG received an Investigational New Drug allowance (IND) from the U.S. Food and Drug Administration (FDA). This is the first time a later stage clinical trial with liver cell therapy can now be started in the US. The FDA-approval is partly based on interim results of an ongoing clinical trial in Germany with neonatal patients suffering from urea cycle disorders (UCD). Cytonet will immediately start the clinical trial SELICA (Safety and Efficacy of Liver Cell Application)-III. The objective of this open, prospective, group-matched, historic-controlled multi-center study is to investigate the safety and efficacy of liver cell therapy in children suffering from UCD. Five therapy centers, as well as 10 centers actively assigning patients and responsible for the after-care, are participating in the SELICA-III trial. 

This study is the second clinical study on liver cell therapy in children suffering from UCD. The first trial, SELICA-II, has been running in Germany for approximately 1 year. SELICA-III comprises a six-month treatment and observation phase and a subsequent 18-month follow-up. The study includes infants, toddlers and children up to the age of five, who are suffering from the most severe forms of the following UCDs: ornithine transcarbamylase (OTC) deficiency, carbamoyl­phosphate synthetase I (CPS I) deficiency or argininosuccinate-synthetase (ASS) deficiency, also called citrullinemia. Cytonet would like to acknowledge the National Urea Cycle Disorders Foundation for its assistance during the development of the program. For more information about urea cycle disorders, visit the National Urea Cycle Disorders Foundation at www.nucdf.org.

About urea cycle disorders and liver cell therapy
UCDs are severe and life-threatening disorders of the ammonia (NH3) metabolism of the liver. These include carbamoylphosphate synthetase I (CPS) deficiency, N-acetylglu­tamate-synthetase (NAGS) deficiency, ornithine-transcarbamylase (OTC) deficiency, argininosuccinate-synthetase (ASS) deficiency — also known as citrullinemia, arginino­succinate-lyase (ASL) deficiency and arginase 1 deficiency (hyper­argi­ninemia). UCDs are based on disorders of 6 known enzymes that are involved in NH3-detoxification. In UCD-patients, the neurotoxic NH3 is not metabolized into urea and excreted. Instead, it accumulates in the blood and tissue. Depending on the severity of the disease, it leads to massive damage of the nerves and the brain, and can be fatal. Children who remain untreated rarely have a normal physical and mental development.

The only currently available cure is the transplantation of a whole liver or a liver lobe. However, this treatment is highly problematic for very young children and neonatal patients. Additionally, suitable organs available for transplantation are very rare. A therapy using isolated and processed liver cells from non-transplantable donated livers (manufactured under GMP standards) has been investigated and further developed over the past years. Cytonet is working in close cooperation with internationally leading neonatal and pediatric metabolism centers. The overall goal is an effective compensation of the metabolism disorder by means of the infusion of healthy, fully metabolism-competent human liver cells into the portal vein, subsequently engrafting in the liver of the child with the disorder.

About Cytonet
The Cytonet Group is an international biotechnology company with sites in Weinheim, Heidelberg and Hannover, Germany and Durham, NC, USA. Currently Cytonet has 60 employees. The company develops, produces and markets cell-therapeutic products for many diseases. In the process developed by Cytonet, liver cells from donated livers are gently isolated and cleaned up in a complex procedure. The donor livers, which are obtained from Organ Procurement Organizations in the United States, are unsuitable for organ transplantation. Additionally, Cytonet provides blood stem cell and bone marrow preparations for the therapy of leukemia and other malignant diseases. Managing directors are Dr. Wolfgang Rüdinger and Dipl.-Kfm. (MBA) Michael J. Deissner. Cytonet was founded by the demerger of the Cell Therapy department from the Roche Group in April 2000. The Dietmar Hopp family owns the majority of shares.