Ocata Therapeutics Inc. (NASDAQ:OCAT), based in Marlborough, Massachusetts, formerly known as Advanced Cell Technology Inc., or ACT, have announced the publication of data in the open access journal, PLOS One, describing the methods of corneal endothelial cell production from stem cell sources. The research, conducted in collaboration with researchers at Bascom Palmer Eye Institute, University of Miami, the Shiley Eye Institute, University of California San Diego and the Byers Eye Institute, Stanford University, outline the methodology for the generation of human embryonic stem cell derived corneal endothelial cells (hESC-CECs). The cells, developed for transplantation in patients with corneal endothelial dystrophies, were reported to be morphologically similar to human adult corneal endothelial cells and to express a range of corneal endothelial cell markers and gene transcripts rendering them a potentially suitable alternative to donor-derived corneal endothelium.
The results indicate that hESC-CECs were almost identical to normal primary human corneal endothelial cells, including in the expression of zona occludens 1 (ZO-1) and Na+/K+ATPaseα1 (ATPA1), and in the production of key proteins of Descemet’s membrane, Collagen VIIIα1 and VIIIα2 (COL8A1 and 8A2). In addition, microarray analysis showed the hESC-CECs to be 96% similar to primary human adult CECs. Ocata is one of the key commercial entities at the coalface of developing stem cell technologies for the treatment of human diseases. The company’s primary focus on ophthalmic conditions includes Stargardt’s, dry AMD, myopic macular degeneration, retinitis pigmentosa, glaucoma and corneal blindness. The application of stem cell treatments to human disorders is a highly active field with both commercial and research scientist / clinician led trials seeking to demonstrate robust proof-of-concept.
Commenting on the achievement, Robert Lanza, M.D., Chief Scientific Officer of Ocata stated, “Disease and injury to the cornea are leading causes of blindness worldwide. Unfortunately, many people are left visually impaired or blind due to lack of available donor corneas. We have developed a simple, two-step method for generating amounts of hESC-CECs, in-vitro, which does not rely on donor corneas. The ability to manufacture these cells from a single, replenishable stem cell source may provide a potential solution to the worldwide shortage of transplantable corneal tissue.”