My Diabolical Plan: Stem Cell Transplant for Dry AMD

If you listen to the news, you know everything in the world right now is not rosy. Far from it. But for those of us with dry macular degeneration (AMD), there is a glimmer on the horizon: Stem cell transplant.

New dry AMD treatment research

Pegcetacoplan clinical trial

Believe it or not, in April I will complete the first year of the long-term trial with APL-2, now called Pegcetacoplan. That means we are a third of the way through the study designed to show how safe and tolerable this treatment actually is when administered over the long term.

Another development with APL-2 is the upcoming Food and Drug Administration (FDA) review sometime this summer. If the drug passes the FDA criteria it will be the first treatment for dry AMD ever in the world.

There is, however, the possibility the FDA will not give approval. While the phase 2 study was promising, the phase 3 study results are said to be less so. Only one of the 2 groups in the phase 3 study reached the desired end goal of efficacy. Depending upon the ruling of the FDA, APL-2 may either be approved or get sent back for further study.

Being an optimistic sort, I am going to bet on the FDA approving APL-2. Not only will that mean people with geographic atrophy can start slowing the growth of their visual lesions, it also means I will be one more step along the way in my diabolical plan.

Stem cell transplant for dry AMD

Like the A Team, I love it when a plan comes together. And so far, my plan is coming together just fine. My friend sent me an article about another piece of my plan just this week.

According to an article from the National Eye Institute entitled Clinical Trial Highlights Stem Cell Transplant for Dry AMD, they are starting trials in which they implant retinal pigment epithelial into the eyes of folks suffering from dry AMD.¹

My diabolical plan

My thought – and my plan – is that this research will be far enough along in 2 years that Apellis, the pharmaceutical company for the study I am in, will want to see how well stem cells will "take” in eyes treated with their drug. I intend to be first in line when that happens.

Why can’t I just jump the line and go with the stem cell study? I would not be accepted because I have been in a study. The study advertised - and may other studies - is looking for what they call treatment naive subjects. That means you have had no prior interventions.¹

Interest in more information?

If that is you and you are interested, you can find more information here. Since they are just starting, they are only taking people with really bad vision, but they might be able to get people with better vision into a later trial. Might as well try to be the solution; yes?¹

Just a caveat, don’t expect to get your vision back with retinal pigment epithelial stem cells. Retinal pigment epitheliums (RPEs) are the “servant cells” and only make the environment good for photoreceptors.

If your photoreceptors are dead, like many of mine are, RPEs are not going to restore your vision. You need new photoreceptors.¹

More steps in my plan

Bringing me to the last step of my diabolical plan. A quick look at the recent research just now confirmed they can grow photoreceptors but still cannot connect them into the neural network. No connection, no sight. Just the same, there is now optimism about some things they are doing in mouse research.¹

Hope for the future

Two years for the long-term study to finish. A couple of years in the RPE stem cell study. Photoreceptors replacement should have made great progress by then; right?

I love it when a plan comes together.

Editor's Note: As of August 2023, 2 drugs known as complement inhibitors — Syfovre® and Izervay™ — have been approved by the US Food and Drug Administration (FDA) to treat geographic atrophy (GA).