Orgenesis Technology Rationale
Pursuing cell therapy in future treatment of various degenerative diseases seems inevitable. Among all forms of cell therapy modalities, autologous cell replacement (AIP) therapy seems to be of the highest benefit.
AIP seems to be safer than other options as it does not alter the host genome but only alters the set of expressed genetic information which seems to be highly specific to the reprogramming protocol. It provides an abundant source of therapeutic tissue, which is not rejected by the patient; which does not have to be treated by immune suppressants. It is highly ethical since no human organ donations or embryo derived cells are needed. The proposed AIP therapeutic approach does not need cellular bio-banking at birth, which is both expensive and could not be used for patients born prior to 2000 (when the first cord blood cell preservation was initiated by a very few individuals), and is very rare even today.
Using Orgenesis technology, individuals can use their own tissues at any given age.
Within the last decade, many thousands of studies published in leading scientific journals confirmed the capacity of reprogramming adult cells from many of our mature organs to either alternate organs or to "stem like cells.”
Orgenesis' technology is the pioneer in this field which has established the groundwork for the entire field of cell therapy to date.
Few examples of this technology are already in clinical use. The most widely used autologous cell replacement protocol is the one used for autologous implantation of bone marrow stem cells. This protocol is widely used in patients undergoing massive chemotherapy sessions which destroy their bone marrow cells. A sample of their own bone marrow stem cells is collected and stored prior to the aggressive chemotherapy session, and the stored cells are introduced back into the same patient to initiate the restoration of their blood cells including cells that belong to the immune system. In some of the protocols, cells are propagated prior to their in vivo administration. However, the cell therapy protocol for cancer patients delineated above does not require extensive cell culture, in vitro.
Despite advances in materials science and innovations in knee repair, no current therapy can mimic the extraordinary biomechanical properties of cartilage. This notion drives initiatives in cell-based replacement technologies, such as Autologous chondrocyte implantation (ACI).
For years, the concept of harvesting stem cells and re-implanting them into one's own body to regenerate organs and tissues has been embraced and researched in animal models. In particular, mesenchymal stem cells have been shown in animal models to regenerate cartilage]. Recently, there has been a published case report of successful cartilage growth in human knees using autologous mesenchymal stem cells. An advantage to this approach is that a person's own stem cells are used, avoiding transmission of genetic diseases. It is also minimally invasive, minimally painful and has a very short recovery period.
Orgenesis cell replacement technology, if proven clinically successful, appears ideal as a therapy for diabetes – a new technology for regeneration of functional insulin-producing cells, thus enabling normal glucose regulated insulin secretion, via cell therapy. By using a therapeutic agent (i.e., PDX-1, or additional pancreatic transcription factors in adenovirus-vector) that converts a sub-population of liver cells into pancreatic islet phenotype and function.
Orgenesis' technology aims to allow the diabetic patient to be the donor of his or her own therapeutic tissue.
Orgenesis’ major competitive advantage is in its cell transformation technology.
Prof. S. Ferber, Orgenesis’ founder established a solid proof-of-concept demonstrating the capacity to induce a shift in the developmental fate of liver cells and converts them into ‘pancreatic beta cell-like’ cells. Furthermore, those cells were found to be resistant to the autoimmune attack.
The Orgenesis technology will provide 7 main advantages over any other IDDM therapy approach currently studied:
- It releases the patient from the daily involvement in the monitoring of blood glucose levels, numerous insulin injections and watching food intake and exercise.
- It allows continuous control of blood glucose levels which prevents diabetes related complications.
- It overcomes the shortage in tissues available for islet cells transplantation.
- It is inserted through an autologous transplant, thus avoiding the risk of transplant rejection.
- No recurrent auto-immune attack on the transplanted beta-cells, thus avoiding the need of immunosuppressant treatments.
- The procedure is only minimally invasive.
- Recovery is expected to be fast.