Promethera Biosciences, a Belgian biotechnology company developing Promethera HepaStem, a cell-based therapy for the treatment of liver-based metabolic diseases including Crigler-Najjar Syndrome and Urea Cycle Disorders, announces it has treated its first two patients with its innovative treatment based on allogeneic adult liver stem cell technology (HepaStem). These patients were treated and are followed at the Cliniques Universitaires Saint-Luc (Brussels, Belgium).
The trial, a prospective, open label multicenter Phase 1/2 study testing Promethera HepaStem is designed to evaluate the safety and the preliminary efficacy of Promethera HepaStem in Urea Cycle Disorders and Crigler-Najjar Syndrome patients.
“HepaStem is an innovation in the field of liver-based metabolic diseases,” said Dr. Beatrice De Vos, chief medical officer of Promethera Biosciences. “This clinical trial will be the very first to apply allogeneic liver progenitor cells. This treatment with progenitor cells will contribute to restore a deficient enzyme function in Urea Cycle Diseases or in a Crigler-Najjar syndrome.”
The major innovation of Promethera HepaStem resides in the simplicity of the treatment. This consists of a simple injection into the vein leading to the liver, which may make it possible to avoid radical and invasive surgery such as a liver transplant. Promethera HepaStem could be used to treat a wide variety of liver pathologies. Thanks to the industrial development, if this therapy proves to be safe and effective, as many as a hundred patients could be treated from a single liver, thus largely overcoming the organ shortage problem.
“There is a real need for medical innovation to treat metabolic diseases in children; too many diseases are still intractable,” said Professor Etienne Sokal, co-founder and chief scientific officer at Promethera Biosciences, paediatric hepatologist at Cliniques Universitaires St Luc and director of UCL’s cell therapy research lab. “This first study represents a major step in the development of Hepastem towards a therapy that could transform positively the prognosis of these diseases.”
Date: May 14, 2012
Source: Promethera Biosciences
Filed Under: Drug Discovery
