We went to Iowa for Visit 1. Visit 1 is the screening process to see if she is still eligible for the pegcetacoplan trial. No medication is given until visit 3. Today, she gave urine and blood, had a 12-lead EKG, and a chest x-ray. Adolescents get the advantage of being able to use recent biopsy and do not need to redo the biopsies required by the trial. We all signed consents to join the trial. We signed away our firstborn. I’m kidding. Kind of. Now we wait to see if she gets called back to proceed to Visit 2, possibly in a month.
This medication is to be given twice a week, via a 30-min subcutaneous infusion pump. This is a 1-year trial, and for the first 6 months, it is placebo-blinded trial, meaning we don’t know if she will get the medication or placebo. The 2nd half of the year, everyone gets the medication. So overall, it’s a good gamble for us. She has such mild problems that likely even if she gets placebo, it won’t be too damaging for her, and then she will for sure get the medication. And there’s a 50% chance she will get the medication the first half of the year. The medication has already been FDA approved for geographic atrophy and has gone through trial for paroxysmal nocturnal hematuria (PNH), so we do have a lot of data that it is safe and feel this is a rather safe gamble. It is our best bet for significant long-term benefit of preventing kidney scarring and preserving her native kidneys.
She is currently the youngest trial participant for C3G. Iowa said their location had the most C3G participants in this world-wide trial. How exciting! How many participants do they have? 7. Suddenly it’s sobering again, how rare this disease is, and let’s face it, each patient is a Guinea pig because so little is known, and so many factors make each patient unique, and only several are on the trial. But wow, seven. From a different perspective, that’s really impressive because our Chicago doctors have so little experience with this condition that Alyssa is the only local patient until I recently heard of another C3G patient at another hospital.
Interestingly, there is another medication iptacopan that is trying to open up a trial for adolescents sometime this year. Iptacopan is an oral medication, so that would be more ideal than injection, but we cannot wait indefinitely for that trial she may not even be eligible for, and possibly risk losing the solid chance we currently have with pegcetacoplan. It’s almost as if we have the illusion of choices. But with rare diseases, beggars can’t be choosers. You can only gamble with what limited options you have.
Is it gambling? Or is everything in God’s hands? If Alyssa gets the medication instead of placebo, does that mean God loves her and answers prayers more for her than for those who got placebo? If she gets placebo or even rejected from the trial, is God still there for her? Does He still have a plan for her? Our faith is on trial too. I am not a believer of the prosperity gospel, for Jesus had loved the poor and broken. My hope and prayer is that through thick and thin, Alyssa would come to know Christ deeply and personally. I hope that she learns how to truly pray. Ok, my prayer is also that she gets on this pegcetacoplan trial and that it helps her with minimal side effects.
One in a Trillion: Alyssa’s medical journey 