Amir Kashani, MD, PhD: Stem-Cell Based Therapy for Dry Age-Related Macular Degeneration

Amir Kashani, MD, PhD, Assistant Professor of Clinical Ophthalmology, Vitreoretinal Surgeon and Retina Specialist, USC Roski Eye Institute:

We talked about a biosynthetic or bioengineered RPE patch, and those were the results of actually a decade's worth of work by a number of people, that have resulted in a artificial RPE patch that is potentially going to be used to treat vision loss in geographic atrophy. So it was a collaborative study, or several years of collaborative work, between the researchers in University of Southern California, University of California Santa Barbara, Caltech, and it was funded by the California Institute of Regenerative Medicine, which is really important because they've been a major supporter of this work, and sponsoring company was Regenerative Patch Technologies.

The idea is that we have a population of patients who have macular degeneration, and they have a form of it that's called a dry form of macular degeneration. We've had a lot of people have neovascular or wet macular degeneration and there's a treatment for that that's very effective at preventing vision loss, on even reversing vision loss. But the dry form of macular degeneration is much more common, and it can cause severe vision, loss legal blindness, or worse and it's actually one of the leading causes of vision loss in the Western world. So there's no treatment for those people because they go blind, because a single cell layer in there eye called the retinal pigment epithelium essentially degenerates. And there's lots of debate as to why that happens, but that's the ultimate result.

So what we've designed is a patch, essentially a single monolayer of RPE cells that are derived from human embryonic stem cells, and we insert that into patients who have this area of atrophy. And the idea is that it might be restore vision or prevent further vision loss.

This was an IND approved, that means the FDA is kind of monitoring it, phase 1-2A study which means we're testing essentially for the safety of the patch. And we're enrolling about 20 subjects in this study, over the next year or so, we're about halfway enrolled. And the idea is that we're going to make sure it's safe, so it doesn't cause anybody to get worse before we go on to phase 2 studies.