“I have told you these things, so that you can have peace because of me. In this
world you will have trouble. But be encouraged! I have won the battle over the
world.”
John 16:33 NIRV
John 16:33 NIRV
The Dark Before the Dawn - The Story Before this Story
A 6-year odyssey of struggle, sorrow, hopes raised and dashed came before and led to my discovery of stem cell treatment. Here is that story.
In May 2008, my Mom alerted me to a news story about gene therapy for a form of RP, a single variant of a group of conditions called LCA. I contacted one of the researchers and learned that the first step was to have my blood tested to identify the exact genetic mutation causing my RP. At the time, there was only one lab in the US doing this testing. Getting results would take a year or more, and individuals were pretty much at their mercy.
It actually took about 2 & 1/2 years and quite a lot of persistence to finally learn that I had the rare variant of LCA that the gene therapy could treat. Then followed 2 more years of making sure my name and test results were in both the files and minds of the researchers when they finally received long delayed approval from the FDA for their next clinical trial. I pursued these researchers because their results vastly exceeded those of any other gene therapy trial, and unlike anyone else, they would be treating both eyes.
In December 2012, with excitement and high hopes, I was screened for their phase 3 gene therapy trial. When I arrived for the screening I learned that, unlike their phase 1 and 2 trials, the FDA had set a low vision limit... a person could see too poorly to qualify. That's what happened to me. My scores on their primary measure fell outside the qualifying range and I was disqualified. ...And yes, that felt like being thrown off a cliff. Many tears and dark moments followed! My family and I were wounded and stunned.
About a week later, the researchers indicated they would seek a compassionate waiver from the FDA to treat me. This would have to be done separately from their clinical trial, so I would need to wait until all the people for the clinical trial were found and enrolled. ...Looooooooong story that went on about a year and a half, with pleas made by phone and in person, and assurances that went unkept.
Meantime, my LCA was doing its degenerative thing. Every few weeks I could see that more of my vision disappeared. The area in which I had any perception at all was getting ittier and bittier with nothing I could do, and the rest of my visual field was going black. With ever decreasing vision I was more frequently banging into things, hitting my face on things I didn't see, feeling isolated and awkward, knowing help was possible but not being able to access it.. On one hand I had my family to love and my life to live, and on the other I tried not to become a frantic mess, but pressed my case whenever I could.
My family, close and extended, and many dedicated friends were a wonderful, loving, and prayerful support through all of this. Their words and deeds held onto me and held me together. They provided rescue, assurance, words from the Lord, scraping me off the pavement, many hugs, hope, and reasons to go on.
Finally I was told that a new batch of gene therapy was being produced. This is an intricate and lengthy process. A previous batch that had taken months to produce ultimately failed, but a new batch was underway. If the yield from this batch was sufficient, they would seek the compassionate waiver to treat me.
In June 2014 after a bunch of unreturned phone calls to check on the production outcome, I received a shocking email. It said that limited gene therapy vector was successfully produced. The researchers did not currently have resources to offer compassionate care. But if they did down the road, selection criteria would be applied to choose who would be treated, and my age and the advanced level of my retinal degeneration would likely mean I would not be chosen.
KABOOM!!!
That was the end!!!
...And then, it was the beginning.
Because in all situations... God is good!
He is trustworthy and I trust Him even when I don't understand it all.
May knowing about the dark before the dawn encourage you and bring you HOPE!
P.S. I forgive the researchers and bless their good work. They're doing cool stuff!
In May 2008, my Mom alerted me to a news story about gene therapy for a form of RP, a single variant of a group of conditions called LCA. I contacted one of the researchers and learned that the first step was to have my blood tested to identify the exact genetic mutation causing my RP. At the time, there was only one lab in the US doing this testing. Getting results would take a year or more, and individuals were pretty much at their mercy.
It actually took about 2 & 1/2 years and quite a lot of persistence to finally learn that I had the rare variant of LCA that the gene therapy could treat. Then followed 2 more years of making sure my name and test results were in both the files and minds of the researchers when they finally received long delayed approval from the FDA for their next clinical trial. I pursued these researchers because their results vastly exceeded those of any other gene therapy trial, and unlike anyone else, they would be treating both eyes.
In December 2012, with excitement and high hopes, I was screened for their phase 3 gene therapy trial. When I arrived for the screening I learned that, unlike their phase 1 and 2 trials, the FDA had set a low vision limit... a person could see too poorly to qualify. That's what happened to me. My scores on their primary measure fell outside the qualifying range and I was disqualified. ...And yes, that felt like being thrown off a cliff. Many tears and dark moments followed! My family and I were wounded and stunned.
About a week later, the researchers indicated they would seek a compassionate waiver from the FDA to treat me. This would have to be done separately from their clinical trial, so I would need to wait until all the people for the clinical trial were found and enrolled. ...Looooooooong story that went on about a year and a half, with pleas made by phone and in person, and assurances that went unkept.
Meantime, my LCA was doing its degenerative thing. Every few weeks I could see that more of my vision disappeared. The area in which I had any perception at all was getting ittier and bittier with nothing I could do, and the rest of my visual field was going black. With ever decreasing vision I was more frequently banging into things, hitting my face on things I didn't see, feeling isolated and awkward, knowing help was possible but not being able to access it.. On one hand I had my family to love and my life to live, and on the other I tried not to become a frantic mess, but pressed my case whenever I could.
My family, close and extended, and many dedicated friends were a wonderful, loving, and prayerful support through all of this. Their words and deeds held onto me and held me together. They provided rescue, assurance, words from the Lord, scraping me off the pavement, many hugs, hope, and reasons to go on.
Finally I was told that a new batch of gene therapy was being produced. This is an intricate and lengthy process. A previous batch that had taken months to produce ultimately failed, but a new batch was underway. If the yield from this batch was sufficient, they would seek the compassionate waiver to treat me.
In June 2014 after a bunch of unreturned phone calls to check on the production outcome, I received a shocking email. It said that limited gene therapy vector was successfully produced. The researchers did not currently have resources to offer compassionate care. But if they did down the road, selection criteria would be applied to choose who would be treated, and my age and the advanced level of my retinal degeneration would likely mean I would not be chosen.
KABOOM!!!
That was the end!!!
...And then, it was the beginning.
Because in all situations... God is good!
He is trustworthy and I trust Him even when I don't understand it all.
May knowing about the dark before the dawn encourage you and bring you HOPE!
P.S. I forgive the researchers and bless their good work. They're doing cool stuff!