Sue has been in the Syfovre research for about 5 years: here’s the timing:
- 7 years ago, my long-time friend Sue became legally blind from GA.
- In 2019, she was accepted into the phase 3 clinical trial for what was then called APL-2 from Apellis Pharmaceuticals. It lasted 24 months/2 years.
- 1-1/2 years ago, she was accepted into the long-term follow-up study that was then mostly called pegcetacoplan. It’s a 36 month3 year study so she’s halfway through it.
If there’s a gap in time, it’s because the trial was shut down for a time because of COVID.
On February 17th, that treatment was approved by the FDA and is not called Syfovre.
She’s written several articles about this, and this is a new one.
Next week she is going to Philadelphia to appear on an ABC-affiliate TV show with one of the researchers from Wills Eye Hospital which is where she was enrolled in the studies. She’s not sure how widely it will be distributed. I’ll keep you posted.
This is her latest article which includes this statement:
“This drug means hope. This drug means no retinal specialist ever again has to tell a patient there is no treatment, no cure [she’s talking about stem cell research here; read her other article in the comments] and they should go home and go quietly blind. No GA patient will need to leave his retina special appointment feeling hopeless. This drug shines a glimmer at the end of the tunnel. We just may get out of this yet.”
What Does Syfovre Mean for You?’ https://maculardegeneration.net/living/thoughts-on-syfovre
Questions and Answers From Her
1. Does she know whether she got the placebo or the treatment in the phase 3 trial?
No. Why not? She’s in the long-term follow-up so she’s still in what’s considered a research study. They don’t reveal the results to individuals till all the date is gathered.
She HAS been getting the treatment (now called Syfovre) every month for almost 1-1/2 year in the long-term follow-up study. In the phase 3 trial, they used her ‘bad’ eye and continue to treat it only. It’s common to do that in research so as not to jeopardize a ‘good’ eye, relatively speaking. While she’s in still in the research, there’s no option to treat the other eye.
2. Can she tell any difference?
The treatment was not shown to improve vision in the clinical trials. She’s had no improvement in visual acuity. She says her visual acuity in both eyes has stayed pretty much the same during these 5 years, but keep in mind that tests of visual acuity are not entirely objective. She admits that they allow her to use her ‘sweet spot’/best area of vision which may have varied over the years. She says she has seen some ‘real world’ decline, but that’s hard to objectify and not uncommon in geographic atrophy. Her visual acuity in her bad eye over the years has ranged between 20-160 to 200; good eye 20-60 to 80. Bottom line is that she is legally blind.
3. If the purpose is to slow down the progression, has it slowed hers?
She doesn’t know…yet. Hopefully at the end of the long-term study (1-12/ more years), they’ll give her the data they gathered from her trial.
4. Does she have any side effects from the injections?
She has them. They’re similar to those of wet AMD because it’s the same concept: ‘intravitreal injection’ means injection into (inter) the vitreous fluid. The injections don’t hurt, she’s not had any bruising/bloody eye, but she does regularly get floaters & air bubbles that resolve in a few hours. She had some episodes where her eye pressure increases, and she only has light perception – lasts only seconds.
Any Other Questions For Her?
I can pass them along. She even said she’d do a Zoom session if there was enough interest.
Her Diabolical Plan
She has a long-range plan. I’ll let her tell you about it. It includes being included in a stem cell trial in the future which would replace damaged RPEs.
If you don’t know the full story of Sue’s journey, I highly recommend that you read it – it’s in the article at the end. Briefly, in July she’ll be 70. She stills work and is extremely active. When I redid her bio as I[‘ve done many times, I came up with this line which might sum it up:
“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.”
You can read or listen to her story here. It’s a 7 minute listen–>
https://mymacularjournal.com/my-diabolical-plan-revisited-march-2023