Funded by the California Institute for Regenerative Medicine (CIRM), the trial has successfully undergone four reviews by the Data Safety Monitoring Board (DSMB). The first patient enrolled in the study, in which allogeneic human retinal progenitor cells were injected into one eye, has now been followed clinically for one year post-treatment. Interim (six to12 month) safety results from this patient and the eight subsequent trial participants enrolled in 2015 are encouraging.
“We are pleased with the results,” said jCyte co-founder Henry Klassen, MD, PhD, an associate professor of ophthalmology at UCI and a member of the Sue & Bill Gross Stem Cell Research Center, where much of the pre-clinical work took place. “Retinitis pigmentosa is an incurable retinal disease that first impacts people’s night vision and then progressively robs them of sight altogether. This is an important milestone in our effort to treat these patients.”
The cell-based approach taken by jCyte is intended to rescue sick and dying retinal photoreceptor cells (rods, cones) in the diseased retina. Nonclinical studies have shown that transplanted retinal progenitor cells tend not to provoke an immunological rejection response, even when sourced from unrelated donors. Because of this, patients in the study are not immunosuppressed, as is often done in traditional organ transplantation. In addition, the procedure is relatively simple to perform and can be completed in an office setting.
The ongoing Phase I/IIa safety trial is being conducted at the Gavin Herbert Eye Institute at UCI and at Retina Vitreous Associates in Los Angeles. Worldwide, almost 1.5 million people suffer from RP, making it the leading cause of heritable blindness. Currently, there is no cure or effective treatment.
Based on the interim results from the current trial, further clinical studies are being planned.
About jCyteBased in Orange County, California, jCyte is committed to developing therapies for incurable retinal diseases. The company was founded by physician researchers Henry Klassen, MD, PhD, and Jing Yang, MD, PhD in 2012. Their work has been supported by the California Institute for Regenerative Medicine (CIRM), the National Institutes of Health (NIH) and other organizations. For more information visit www.jcyte.com.
About the Sue & Bill Gross Stem Cell Research CenterOne of the largest and most technologically advanced stem cell research facilities in the world, UCI’s Sue & Bill Gross Stem Cell Research Center was established, in part, through a $10 million gift from Sue and Bill Gross. For more than 40 years, UCI scientists and research and graduate assistants have worked to unlock the potential of stem cells in treating and curing about 70 major diseases and disorders. Advances have led to the world’s first clinical trial of a human neural stem cell-based therapy for chronic spinal cord injury and the first FDA-approved clinical trial using human embryonic stem cells. For more information, visit http://stemcell.uci.edu/.
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