UCI scientists are poised to break ground in efforts to combat retinitis pigmentosa, a degenerative eye disease.
The procedure, pioneered by a team of collaborators that included Dr. Henry Klassen and Dr. Jing Yang, received approval late last week from U.S. Food & Drug administration to begin clinical trials.
Those with retinitis pigmentosa (RP) suffer from a degeneration of their rod photoreceptor cells, resulting in dimmed and compromised peripheral vision. Currently, there are no procedures to treat RP because of its irreversible nature.
Using stem cells, the procedure intervenes in the degeneration of the photoreceptor cells while they can still be protected. The scientists are hoping that initial clinical trials will lead to further breakthroughs that will allow them to go one step further and fully reactivate the rods.
Because the procedure hasn’t been tested before, the main purpose of these initial trials are to determine the safety of injecting the stem cells into the eyes of patients with severe retinitis.
Currently, the UC has a patent pending on the technology. jCyte Inc., a start-up cofounded by Klassen and Yang is set to administer the trials by licensing research rights from the university.
Although approved by the FDA, the trial is still seeking approval from UCI’s own institutional review board. Once approved, the clinical trials will take place at the Gavin Herbert Eye Institute. Up to 16 patients will be enrolled for initial studies, with a possibility of expanding to a second site.
“This milestone is a very important one for our project,” said Klassen, an associate professor of ophthalmology affiliated with UCI’s Sue & Bill Gross Stem Cell Research Center and Gavin Herbert Eye Institute. “It signals a turning point, marking the beginning of the clinical phase of development, and we are all very excited about this progress.”
The beginning of these clinical trials are the result of over a decade of research supported by the California Institute for Regenerative Medicine, which was founded in 2004 with funds from Proposition 71.
“Without the backing of CIRM and the people of California, we would have never made it this far this quickly,” Klassen said. “To the patients and their families who have been waiting all these years, I am delighted to finally be taking our research out of the lab and into the clinic.”