GA therapy endpoints: Should tissue preservation be considered?

Author(s):

As novel therapies in the pipeline are aiming to decrease the rate of GA expansion—the endpoint Karl Csaky, MD and Fredrick Ferris III, MD, identified in 2007—Dr. Csaky reinforces on the importance of preserving the central retinal tissue.

At Retina World Congress 2022, Karl Csaky, MD, presented a talk entitled, “Interpreting Reported GA Treatment Effects to Patient Care: Concept of Tissue Preservation.” He discussed how far research in geographic atrophy has come since he embarked on a project to determine provable endpoints for geographic atrophy (GA) in 2006 and 2007. Now that treatments in the pipeline are aiming to decrease the rate of GA expansion—the endpoint Dr. Csaky and Dr. Ferris identified in their research—he doubles down on the importance of preserving the central retinal tissue.

Dr. Karl Csaky serves as the T. Boone Pickens Director of the Molecular Ophthalmology Laboratory and Clinical Center of Innovation for Macular Degeneration. Additionally, Dr. Csaky is the Chief Executive and Medical Officer at Retina Foundation of the Southwest.

Video transcript

What we're trying to do is, you know, many years ago, I led with Rick Ferris, and a symposium with the Food and Drug Administration on endpoints for various trials and retinal diseases. And back in 2006-2007, we worked on and came to an agreement that the decrease in the rate of GA expansion could be a provable endpoint. And that's been now the gold standard in the community.

And here at this meeting, and throughout other meetings, we are hearing about treatments that may be achieving that endpoint. However, when we really think about what's critically important for patients, while reducing GA expansion is definitely a way to assess the therapy overall, what I was trying to focus this talk on is the what's critical for patients. And for patients. It's really the central retinal tissue within the macula and fovea that needs to be preserved.

So when we think about these therapies potentially coming to the clinic, we really need to be thinking about them in a slightly different, more focused approach. Which is trying to understand as we apply these treatments: How much truly important, critical tissue is being preserved? Which will allow for the patients to have better outcomes in terms of driving, seeing and reading. And so this talk is simply a focus on that subject.

Note: This transcript has been lightly edited for clarity.