First generation of human retinal cells in the laboratory from human embryonic stem cells

Sep 23 2004

Advanced Cell Technology reported today the first generation of human retinal cells in the laboratory from human embryonic stem cells.

Some of these retinal tissues such as Retinal Pigment Epithelium (RPE) may provide a novel therapeutic strategy for such devastating diseases as macular degeneration, retinitis pigmentosa, and other retinal degenerative diseases.

The research, which will appear in the Fall issue of the peer-reviewed journal Cloning and Stem Cells, by ACT and its collaborators at Wake Forest University and the University of Chicago, describes for the first time the isolation and characterization of putative RPE cells from human embryonic stem cells, as well as the first application of transcriptomics (gene expression profiling) to comparatively assess stem-cell derivatives vs. their in vivo counterparts.

"Millions of patients with retinal degeneration might conceivably benefit from these cells in the future," said Robert Lanza, Medical Director at ACT, and senior author of the study. "An important next step will be to test the ability of these cells to restore visual function in both humans and animal models. With therapeutic cloning, or the creation of banks of stem cells generated using parthenogenesis (perhaps as few as 40-400 lines), a limitless supply of immune-matched cells could be generated for most of the population."

This study describes for the first time, a differentiation system that does not require co-culture with animal cells or products, thus allowing the production of pathogen-free RPE cells suitable for transplantation into patients. "We differentiated several human embryonic stem cell lines into RPE," said Irina Klimanskaya, Senior Scientist at ACT, and first author of the study. "These cells expressed RPE-specific molecular markers and were capable of phagocytosis, an important RPE function. Gene expression profiling further confirmed their similarity to RPE from natural sources."

In addition to retinal cells, other stem-cell derivatives are likely to play an important role in the future of regenerative medicine. Evaluation and comparison to their in vivo counterparts will be critical for assessment of their therapeutic potential. This is the first application of transcriptomics to assess ES cell derivatives and their in vivo counterparts -- a 'differentiomics' outlook. Age-related macular degeneration (ARMD) alone affects over 30 million people worldwide and is the leading cause of blindness in patients over 60 in the United States. The use of multiple hES-RPE lines in these studies will allow further correlation between function and gene expression. Differentiomics could also be a valuable predictive tool for quality assessment of other ES cell derivatives based on their molecular signature.

"Human embryonic stem cells are so exciting because they provide a pathway of making many desperately-needed cell types for use in medicine," said Michael D. West, President & C.E.O. at ACT and an author on the paper. "In the ongoing debate over embryonic stem cells some have argued that the cells have not demonstrated their potential value in medicine. No one can reasonably say that anymore. Besides use in the aging retina, human embryonic stem cells have now demonstrated the potential to change the face of several age-related degenerative diseases, perhaps one day allowing physicians to prescribe young cells they way they dispense pills today."

The researchers of the paper from Advanced Cell Technology, collaborated with scientists from the Institute of Regenerative Medicine at Wake Forest University School of Medicine, Winston-Salem, North Carolina, and the University of Chicago, Chicago, Illinios. The paper's other authors are Jason Hipp and Anthony Atala of Wake Forest University, and Kourous A. Rezai of the University of Chicago.

Advanced Cell Technology is a leading biotechnology company in the emerging field of regenerative medicine.