|
Listen to this article
 |
Some of you may know I can be persistent -very ! -and even manipulative. I get a plan in my head and make the moves to follow it through.
Ever since I was diagnosed as legally blind with geographic atrophy, advanced age-related macular degeneration, in February 2016, I have had a master plan, a DIABOLICAL plan, for recovering my eye sight sometime before I die [Linda here: curious how old she is? At the time of this article, she is 69. She’s determined to live a LONG time. ::smile::]. This plan started with presenting myself as a patient to the esteemed retinologist Dr. Carl Regillo from Wills Eye Hospital in Philadelphia. I then expressed interest in being in a study. I wanted a clinical trial hopefully involving stem cells.
After what seemed like dozens of trips to Bethlehem, Pennsylvania, my persistence paid off. I was offered a place in phase 3 of a study using a complement factor inhibiter that had been considered to have promise. While initially it seemed as if being offered this study was one step to the left of where I wanted to be, I decided to take it. It was my “in” and I was not guaranteed another opportunity.
As it turned out, I had the honor of being part of the trials that brought forth the first, FDA-approved treatment for geographic atrophy. This is Syfovre from Apellis.
How do I feel about this? While my contribution was small, it is still kind of cool to have been involved in the making of medical history. I feel like the water boy for the Super Bowl or maybe like the ball boy/girl at Wimbledon. Helping to make it happen gives me pride.
So here I am, two steps in the Diabolical Plan successfully behind me. I manipulated getting to see Regillo, I wanted him as a doctor and took steps to make that happen. Regillo almost guaranteed I would be in a study. Funny, although I had no way to determine the outcome of the study, I never doubted the treatment would be approved. Forgive my arrogance, but without its approval, my plan could not progress, and, as far as I am concerned, it IS happening. If my plan was going to work, the treatment had to work first. I never doubted.
Right now, I am half way through a 3-year study in which they are searching for side effects. For me, this study is a place holder in three ways. First of all, it keeps me in front of the doctors running the show. While I cannot go into just any old study, I could go into another study involving Syfovre. When one of those comes along, I will be bouncing up and down on the sidelines. “Put me in, coach. I want to play!”
The other reasons this study is a place holder are these: medical science is not there yet.
Also, Syfovre is not the best we can do.
Second one first: while the Benz Motorwagen was a great innovation in 1886, it was also not at the front of the pack for long. And yes, that really was the first car. I looked it up.) Just like the Benz Motorwagen was replaced by more efficient cars, there will be other, better treatments for GA. My believe is the ultimate “treatment” will be “replacement parts”. Enter stem cells.
They have now learned how to “reverse engineer” blood and skin cells to become induced pluripotent stem cells. Once you have these, they can be “coaxed” and “massaged” into becoming retinal pigment epithelial cells. Remember RPEs are the “servant cells” that keep the photoreceptors alive. Without RPEs there are no photoreceptors and no sight.
According to a panel member on the March 2nd, 2023, webinar ‘Towards a Stem Cell Cure for Blindness,’ lab-grown RPE cells might be available to the general public in about six years. They are moving fast but they are still in phase 1 trials. In other words medical science is not ready for me yet.
Moving on with my thinking, Syfovre will be a way for me, and perhaps millions of others, to buy time. Syfovre slows the deterioration. Current wisdom is that stem cell therapy and RPE replacement stops deterioration. If Apellis wants to sell their drug, they have to prove it will not keep people from eventually benefitting from the superior treatment. Voila! There is my – yet imaginary – next step in my Diabolical Plan. Put me in, coach!
And just a reminder, replacing RPEs will not restore vision. As I have said before, dead is dead and a number of my photoreceptors are dead. However, replacement RPEs will stop my vision from getting worse. They may also improve it slightly, not much, by resuscitating photoreceptors on their last legs.
What about photoreceptor replacement? After all, I do need those replaced to restore my vision. That technology is much further down the road but still getting closer every day. It will get here in time. After all, I have a Diabolic Plan to complete!
Who is Sue and Why Should You Get to Know Her?
The Facebook Group
First, it weren’t for Sue, the Facebook group wouldn’t exist! We’ve been close friends since college which is now a LONG time ago. In June 2015 she was driving, got something in one eye and closed it, and the car in front of her disappeared! You can read about that and how her journey with AMD began here: In the Beginning. In February 2016 when she was told that she was legally blind, she asked me to create a site for her so that she could process what was going on and to hopefully help others. In May 2016 after months of not being able to easily have conversations with followers, I started the Facebook group. The rest, as they say, is history.
Her Journey
After less than a year of learning how to deal with her visual impairment both physically and emotionally, Sue created a ‘normal for her’ life. What is it like? It’s like her life ‘before’ except she does not drive. What she does is travel domestically (sometimes even by herself) and aboard, works as a psychologist, walks her 2 active Labrador Retrievers, attends multiple exercise classes every week, rides her bike safely, takes photographs (some of which have won awards locally), skis in the Winter and rafts in the Summer, goes to social events with her friends and co-workers. She’s also taught courses in the psychological therapy called DBT (Dialectical Behavioral Therapy) that she uses with her clients and used herself in the beginning of her journey.
What she does NOT do is let her geographic atrophy stop her from doing what she wants to do. As she said herself she is persistent and determined.
For quite a few years, she wrote for this site, but it’s now more an archive. She writes for Health Union’s maculardegeneration.net site where you can read her more recent articles. As she wrote, she was in the phase 3 Apellis Pharmaceuticals clinical trial for what was called APL-2 then, was invited to be in the long-term follow-up study where it was called pegcetacoplan which is called Syfovre since it’s approval by the FDA on February 17th. 2023.
When asked what her visual acuity is, she says it’s 20/63 to 20/80 in one eye & between 20/160 and 20/200 in the other. Yes, she has a blind spot in the middle of each eye but has taught herself how to use her ‘sweet spot’ also called a Preferred Retinal Location.