Date: 2014-03-11
Type of information:
phase:
Announcement: presentation of interim data at the Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) in Pacific Grove, California.
Company: Cytonet (Germany - USA)
Product: human heterologous liver cells (Heparesc)
Action mechanism: liver cell therapy
Disease: Urea Cycle Disorders (UCD) in children
Therapeutic area: Metabolic diseases - Genetic diseases
Country: Canada, Germany, USA
Trial details: In these studies, young children with UCD will be treated by repetitive application of human liver cells. In the last consequence, the aim of this new therapy option is to supply a sufficient amount of healthy liver cells to compensate for the metabolic defect and to reduce the risk of neurological deterioration while awaiting OLT (NCT01195753 and NCT00718627).
Latest
news: * On March 11, 2014, Cytonet has announced that interim data on the use of liver cell therapy (LCT) for the treatment of pediatric patients with Urea Cycle Disorders (UCD) were presented today at the Annual Meeting of the Society for Inherited Metabolic Disorders (SIMD) in Pacific Grove, California. Thomas Opladen, MD, from the University of Heidelberg presented data from a combined interim analysis of two studies performed in Germany, the U.S. and Canada. Patients less than six months of age with a confirmed diagnosis of UCD (OTCD, CPS1D or ASSD (citrullinemia) were treated with liver cell therapy and were compared to a historical control group treated with the current standard of care. Advantages for the treated group were seen for parameters such as event free survival time or incidence of moderate or severe hyperammonemic events. Reduction of such events is the objective of the liver cell therapy in order to avoid irreversible brain damage. The liver cell therapy was well tolerated. Cytonet’s 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. The Company is currently conducting two on-going multicenter clinical trials in U.S. and Canada (SELICA III), and in Germany (SELICA V).In December 2013, Cytonet submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval for its liver cell therapy for the treatment of Urea Cycle Disorders in children.
