Retinitis Pigmentosa (RP) generally first affects people in their teens. An early symptom is impaired night vision, followed by progressive loss of peripheral vision and eventually in most patients, loss of visual acuity and color/central vision. Most people become legally blind by the time they reach their 40s. RP affects an estimated 1 in 3500 people in the U.S. Although rare, this disease can arise from a combination of hundreds of genetic mutations and has multiple subtypes. So far, there is no cure.
Dr. Henry Klassen, associate professor in ophthalmology research at UCI, has spent the last decade developing a stem cell-based therapy to stop retinal cell degeneration. His research at UCI was supported by multiple grants from the California Institute of Regenerative Medicine (CIRM).
In 2012, Klassen and his co-investigator, Dr. Jing Yang, also a UCI faculty member, founded jCyte, located in Newport Beach, California. Paul Bresge, now CEO, and his colleague Terry O’Neal, COO, along with the founders, have grown jCyte by establishing a team of dedicated individuals, raising needed capital, obtaining the necessary regulatory approvals, and pushing the treatment towards clinical trials as quickly as possible.
jCyte’s stem cell therapy, jCell, was granted orphan drug designation by both the FDA and the European Medicines Agency, making the company eligible for certain patent protections, tax breaks, and grant funding. jCell was accepted into the competitive Therapeutics for Rare and Neglected Diseases (TRND) program at the NIH National Center for Advancing Translational Sciences (NCATS).
How the treatment works
The jCell treatment involves the intravitreal injection of immature progenitor stem cells, which can develop into various types of retinal cells, into the eye behind the retina. This investigational therapy is conducted as an outpatient procedure under topical anesthesia. Researchers think that the progenitor cells release protective growth factors that help preserve existing retinal cells from further degeneration. These progenitor cells may integrate into the retina, but how exactly, and by how much is still unclear.
The eye is a favorable site for stem cell therapy because there is less risk of cells migrating elsewhere. The eye is considered “immune privileged,” meaning that it tolerates the transplanting of foreign cells better than other parts of the body, so it is less likely to require anti-immune rejection drugs. jCyte believes that its cells are less likely to induce tumors, as they are already differentiated into retinal cells.
Testing a first-of-its kind therapy.
jCyte’s first-of-its-kind stem cell-based treatment received consent from the U.S. Food & Drug Administration (FDA) for use in a clinical trial in 2015. Enrollment and treatment of twenty-eight patients with advanced RP in the phase I/2a trial was conducted at the UCI Gavin Herbert Eye Institute as well as Retinal Vitreous Associates in Beverly Hills. This study will be completed in July 2017. The study has the primary goal of determining the safety of the experimental therapy, with potential vision improvement as a secondary measure.
Patients in the study had human retinal progenitor cells injected into their worst eye, and are being followed clinically for one year afterward. The trial underwent four reviews by a safety monitoring board. Results to date have shown that jCell therapy appears to be safe and well-tolerated by patients.
On May 2, 2017, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for jCell. The FDA RMAT designation could accelerate jCell’s path from clinic to patients, as it facilitates expedited reviews and approvals for stem cell and other cellular and tissue therapies that manage serious or life-threatening conditions.
In April 2017, jCyte announced a phase 2b trial to investigate jCell’s efficacy. This trial, recruiting about 70 patients, is supported by an $8.3 million CIRM grant. The trial will be conducted at UCI’s Gavin Herbert Eye Institute, Retinal-Vitreous Associates Medical Group, and the UC San Francisco Department of Ophthalmology.
In an outpatient procedure, patients will receive an injection of human retinal progenitor cells in the eye with the worst vision, and will then be followed for one year after treatment. The control group will get a sham procedure. After a year, if results are favorable, the control group will have the option of receiving treatment.
The study will assess primarily whether jCell improves eyesight, measuring visual acuity, mobility, color recognition and contrast sensitivity. The study will also examine the
safety of the treatment and how well the cells are tolerated.
Other stem cell techniques as well as gene therapy are being investigated by startups and research institutions. jCyte hopes that this approach may conceivably be used in other degenerative eye diseases in the future.
For more information, visit www.jcyte.com