First-of-its-kind clinical trial for retinitis pigmentosa is enrolling first patients for safety study

The University of California, Irvine (CA, USA) is enrolling its first patients for a Phase I/IIa clinical trial to treat retinitis pigmentosa

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Aug 19, 2015

Participants are being enrolled in the first clinical trial that tests the use of retinal progenitor cells to treat retinitis pigmentosa (RP). These retinal progenitors are the product of stem cell research at University of California, Irvine (UCI; CA, USA) and are similar to stem cells in terms of potential regenerative properties, but are specific to the retina.

To date, four participants with RP have received the cell injections, either at the Gavin Herbert Eye Institute in Irvine or at Retina Vitreous Associates in Los Angeles (CA, USA). This effort is in conjunction with the California Institute for Regenerative Medicine’s new Alpha Stem Cell Clinic network, of which UCI is a founding member. “We are delighted to be moving into the clinic after many years of bench research,” commented Henry Klassen, project director of UCI’s Gavin Herbert Eye Institute.

Retinitis pigmentosa is a rare genetic condition associated with progressive loss of retinal photoreceptors (rods, then cones). The disorder progressively results in night blindness, tunnel vision and, ultimately, severe visual disability and blindness. There is no current treatment for RP, which is considered an 'orphan' disease by the US FDA, thereby conferring advantages in terms of the regulatory pathway necessary for drug approval.

The open-label Phase I/IIa trial is designed to evaluate the safety of retinal progenitor cells injected into the vitreous cavity at two different dosage levels in patients with late-stage RP. The total enrollment will involve 16 patients all of whom will receive a single injection of cells into the most affected eye under topical anesthesia. No systemic immune suppression will be utilized and participants will be followed for 12 months to monitor safety and efficacy parameters.

This first-of-its-kind stem cell-based treatment for RP was created by Klassen and Dr Jing Yang, who co-founded jCyte to sponsor the development of the investigational therapy. The therapy is intended to preserve vision by intervening at a time when degenerating photoreceptors in the patient’s retina can be protected and potentially reactivated, as opposed to replacing lost cells, which is very challenging.

The initiation of this clinical trial represents the culmination of a research project stretching back more than a decade. This project was accelerated by support from the state’s stem cell agency, the California Institute for Regenerative Medicine, which granted the team US$17 million for the current phase of the project.

To learn more about the trial or to enroll, contact the UCI Alpha Stem Cell Clinic at 949-824-3990 or

Source: University of California, Irvine press release:

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Elena Conroy

Contributor, Future Science Group

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