Letting the Good Times Roll in Retina

American Academy of Ophthalmology 2013

Carl D. Regillo, MD; Allen C. Ho, MD; Sunir J. Garg, MD


December 04, 2013

Editorial Collaboration

Medscape &

In This Article

When Will We Have Gene Therapy?

Dr. Regillo: Gene therapy is a real niche field. We heard a number of talks about gene therapy, either to replace a missing gene or to promote the development of a therapeutic in the eye for wet AMD, for severe retinal degenerations like RPE65. What is happening there?

Dr. Ho: Gene therapy will be a part of our treatment toolbox for patients with a variety of conditions. Al Maguire's[12] work on Leber congenital amaurosis is fascinating. Jeff Heier's[13] work introducing a gene that will produce an anti-VEGF that might reduce the need for injections is very clever as well. We need more information, but it's showing that there will be some traction with gene therapy.

Dr. Regillo: This is not on the horizon, though, is it? We are making progress, but are we talking 5 or 10 years? What sort of timeline do you anticipate?

Dr. Ho: Within the next 2-3 years, we could have a gene therapy available.

Safety Profile of Ocriplasmin

Dr. Regillo: That is a big development. We have had a new therapeutic for about a year now, and we are starting to get some real-world experience with ocriplasmin[14] for treating vitreoretinal interface disorders. We have heard about the phase 3 studies. But now, at this meeting, we are starting to hear about postmarketing, commercially available use of the drug. More than 4300 injections were done in the United States this past year.

We heard a little bit about the experience -- the positive, the side effects, and so forth. What are we hearing at this meeting, Sunir?

Dr. Garg: These data are really helpful. They basically confirmed what we found in the phase 3 trial, which is that the drug works and it's quite safe. A few patients might experience some transient vision loss, but most of them recover vision very quickly. In an occasional patient, it seems as though the vision loss lasts a little bit longer, and we need longer follow-up on those patients. These data encourage retina specialists to continue to use this modality as a new treatment option for their patients.

Dr. Regillo: This surveillance is important because the phase 3 study only had 6 months of follow-up. For patients who were still having some ongoing issues --reduced vision or visual symptoms to some degree, even with successful treatment with the drug -- longer-term follow-up is important. It's like vitrectomy surgery for a macular hole. We see continued vision improvement well beyond 6 months. That sort of phenomenon could exist here too.

That is all we have time for today. We have had a lot to talk about, and we will have many more meetings sorting out these interesting points and new developments.

I would like to thank Dr. Sunir Garg and Dr. Allen Ho from Wills Eye. I'm Dr. Carl Regillo for Medscape Ophthalmology and Wills Eye Hospital. Thank you for joining us.