CIRM-Funded Therapy for Blinding Disease Treats First Patients


August 11, 2015

A clinical trial using stem cells to treat people who have lost their vision due to retinitis pigmentosa (RP) has treated its first four patients. CIRM, California’s stem cell agency, is funding the trial, which has FDA consent.

“We are delighted to be moving into the clinic after many years of bench research,” says Dr. Henry Klassen, the project director at the University of California, Irvine (UCI).

The four patients are legally blind because of retinitis pigmentosa, a degenerative disease that slowly destroys cells in the retina, the light sensitive area in the back of the eye that is essential for vision. The patients were each given a single injection of retinal progenitor cells. It’s hoped these cells will help protect the photoreceptors in the retina that have not yet been damaged by RP, and even revive those that have been damaged but not yet destroyed by the disease.

“This is an important step for Dr. Klassen and his team, and hopefully an even more important one for people battling this devastating disease,” says Jonathan Thomas, Ph.D., J.D., Chair of the Board of CIRM. “Our mission at CIRM is to accelerate the development of stem cell therapies for patients with unmet medical needs, and this certainly fits that bill. That’s why we have invested almost $19 million in helping this therapy reach this point.”

Altogether 16 patients will be enrolled in this first phase. All will receive a single injection of retinal progenitor cells into the most affected eye. The patients will then be followed for 12 months to determine if the therapy is safe and if it has any beneficial effects on vision compared to the untreated eye.

The treatments are being administered at the Gavin Herbert Eye Institute in Irvine and at Retina Vitreous Associates in Los Angeles. This is one of the first clinical trials being carried out as part of CIRM’s new Alpha Stem Cell Clinics Network, which brings together experts in developing and delivering these new therapies to patients.

Worldwide almost 1.5 million people suffer from RP. It is the leading cause of inherited blindness in the developed world. There is no cure and no effective treatment.

To learn more about the study or to enroll contact the UCI Alpha Stem Cell Clinic at 949-824-3990 or by email at stemcell@uci.edu.

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