Many types of diseases, whether of genetic origin or not, present a severe dysfunction or generalized dysfunctions of the liver, which can only be treated by liver transplant. The transplantation of hepatocytes, the functional cells of the liver, is a promising alternative to liver transplant but faces a shortage in human hepatocytes to be transplanted. One of the objectives of the CESTI IHU, is to develop innovative therapies: its tender “support for innovative projects” has selected the StemHepTher project coordinated by Dr. Tuan Huy Nguyen (Inserm UMR 1064 / ITUN).
This particularly promising project proposes to develop an innovative therapy medicinal product made from embryonic stem cells, an unlimited potential source of human hepatocytes. It will, at first, evaluate the efficacy and biosafety of human hepatocytes generated from a grade GMP (Good Manufacturing Practices) embryonic stem cell which already exists. Then, in collaboration with clinicians of Nantes University Hospital and Paris Public Hospitals (AP-HP), Dr. TH Nguyen wants to set up a clinical trial that will involve patients with inherited metabolic liver disease linked to lack of a liver enzyme. This treatment with cell therapy could eventually involve all liver diseases which require a liver transplantation.
This project, whose purpose is to meet the medical needs resulting from the liver graft shortage, brings together regional, national and international expertise and infrastructures which are recognized in the fields of pluripotent stem cells, cell therapy and cell therapy product production.
Meanwhile, the project received the support of the French National Research Agency (ANR), Atlanpole and “SATT Ouest Valorisation” for the proposed creation of a start-up company based on the production technology of human hepatocytes from pluripotent stem cells. Winner of the national contest I-LAB 2015, contest for the creation of innovative technology companies, which was launched by the ministry in charge of research.