One-step beyond – Embryonic stem cells for drug discovery and treatment of degenerative diseases

Prof. Benjamin Reubinoff, M.D., Ph.D. Chairman of the Department of Obstetrics and Gynecology.

Human embryonic stem cells (hESCs) are derived from the early in vitro fertilized embryo. Stem cells are unique as they are pluripotent, i.e., capable of giving rise to all different cell types of the human body, and can self-renew infinitely in culture. Thus, given the unique properties of pluripotent stem cells, they may serve as an unlimited source of human cells for transplantation therapy for the replenishment of malfunctioning or dying cells. Moreover, these cells may serve to model human diseases and the development of novel therapeutic modalities and drugs. hESCs have the potential to cure diseases associated with degeneration of cells or loss of their function. These conditions may include neurodegenerative disorders such as Parkinson's disease, multiple sclerosis, cerebrovascular accidents and spinal cord injuries, as well as heart failure, diabetes mellitus, and others.

Prof. Benjamin Reubinoff has been among the world’s pioneers in the development of hESCs and the first to show their potential to give rise to various cell types in culture. Towards further development of hESCs for clinical applications, the Hadassah hESC Research Center was established in 2003 within the Goldyne Savad Institute of Gene Therapy in collaboration with the Department of Obstetrics and Gynecology. The Center is directed by Prof. Benjamin Reubinoff from the Department of Gynecology and includes a staff of seventeen scientists, PhD students and technicians. The hESC Research Center at Hadassah is among the world’s pioneers in developing hESCs for regenerative therapy. The Center developed new hESCs under high quality conditions that allow their use as starting material for clinical cell therapy applications. The clinical-grade hESCs are included in the NIH hESC lines registry and have been provided to multiple groups in Israel and worldwide for the development of cell therapy for a variety of conditions, including retinal and neurological degenerations and diabetes mellitus.

At the Hadassah hESC Research Center, Prof. Reubinoff and research team are focusing on the development of stem cell therapy for retinal and neurological degenerative conditions. These projects are conducted in collaboration with Prof. Eyal Banin and Prof. Tamir Ben-Hur from the departments of Ophthalmology and Neurology, respectively. Age-related macular degeneration (AMD) is the leading cause of blindness in the Western World and is caused by malfunction and death of the retinal pigmented epithelial (RPE) cells. Until now, there has been no cure for AMD. There are about 1.6 million new cases of dry-AMD in the US annually. Prof. Reubinoff and collaborators have developed a novel approach to direct hESCs to mature into RPE cells for the potential replenishment of the dying cells in these patients. They have initiated a clinical transplantation trial in AMD patients and are among the world’s pioneers in translating hESCs into the clinical setting. They are also developing hESCs for transplantation therapy in multiple sclerosis.

Another goal of the Hadassah hESC Research Center is to use pluripotent stem cells for modeling diseases and developing novel therapeutic approaches, specifically modeling and studying cerebral palsy (CP) and amyotrophic lateral sclerosis (ALS). These studies may pave the way to the discovery of new drugs for these devastating and untreatable conditions.

In summary, due to the role that pluripotent stem cells may play in the treatment of deleterious untreatable conditions, the Hadassah hESC Research Center led by Prof. Reubinoff is at the forefront of world science in exploiting these promising cells for clinical therapeutic applications.