The Foundation Fighting Blindness (FFB) has been my main portal for information about the mechanics of eye disease, potential therapies, clinical trial updates, as well as practical living and coping skills through their website, blog, and Visions conferences for a long time. But I learned at the last major FFB conference that you can’t be in two great places at one time. Now “Visions 2012 Rewind” allows me to listen to some other interesting topics and sessions I missed while at that conference in Minneapolis, helping me with some lingering questions.
My FFB education has taught me a few important points about eye research. First of all, the chase for the targeted treatment of retinal degenerative diseases using drug, gene, or stem cell therapy is leading the way by moving years of laboratory research into actual human trials and treatment. Turns out that in the real world, the eye is an easy study – it’s accessible, it’s contained (what goes in the eye stays in the eye), it’s largely invisible to the immune system, and there are a myriad of tests available, along with high definition and cross-sectional photography, that help to track disease progression and prove trial progress.
Perhaps due to my background as a technician, my current participation in the stem cell clinical trial has led me to my latest obsession – the tests. Because this is only a Phase I/II clinical trial, the endpoints (measurements proving trial success) focus mainly on safety, as they should. But being the human subject, I also want to know what’s going on inside there. Lacking any notable changes in my vision as of yet, I want to know if there are indicators that I can’t measure by simply closing one eye. As I explained in a previous post, my baseline (pre-surgery) central vision was pretty non-existent, so perhaps it would take major internal progress before I’d notice a functional difference, like if it became easier for me to cross the street.
As many of you who have visited retina specialists know, there are numerous forms of visual testing torture – bright lights and big cities. But two tests have caught my attention lately – the Goldmann perimetry test and the multifocal ERG. Perhaps they could indicate that the thick fog I’m always looking through is becoming thinner. I’ve had promising, yet mixed results in the Goldmann test, which has a subjective component – am I really seeing a light move across my blind spot now? One day it was obvious; I can still visualize that light where I hadn’t seen it before. However on another, perhaps more tired day, my guarded and skeptical personality may have been slow to pull the trigger. But subjectivity is completely removed during the multifocal ERG, where all I do is sit there while patterns of light flash before my eye In the meantime, it plots out photoreceptor response based on electrical impulses, so you can daydream away while it does all the work.
I’m not sure if any other organ has such a diverse and technical array of devices for testing and observation. It seems that every time I go to a retina center, there is a new or updated piece of equipment. And with computer software, it’s possible to automatically compare results to a normal eye (even based on age) as well as your own baseline results.
And while contemplating other questions I had about testing Stargardt progression, I came across this recently announced FFB Stargardt study called ProgStar:
“The study has three primary goals: 1) determine the best outcome measures to accelerate evaluation of emerging treatments; 2) better understand disease progression for selecting future clinical trial participants; and 3) identify potential participants for forthcoming clinical trials.”
Their findings may pave the way for Phase III trials and beyond, involving a larger number of people and where the treatment’s effectiveness is at the heart of the matter. Beyond that, I wonder if it will become commonplace to predict the effectiveness of certain treatments based on the patient’s medical history and progression of vision loss. Using a combination of therapies for retinal degenerative diseases appears to be on the radar for researchers …pretty incredible given that most of these diseases currently have no FDA approved treatments. I can understand why the eye is leading the way and has the medical community watching, learning and hoping to apply these methods and conclusions to future medical treatments that extend well beyond the eye.
Disclosure: Maurie Hill is a patient in this Stargardt clinical trial and is also an Advanced Cell Technology (ACT) stockholder. ACT is the trial’s sponsor.