Well, day 2 back in the gym. Feels GREAT. Remember how full of excitement I was a the beginning of the year to hit the Transformation horse? Well, I fell hard off the wagon. I realized the easiest thing to do is simply replicate what has worked best in the past. Pure and simple! Why re-invent the wheel? I know that I need to work out before work, otherwise, it won't happen, and I know that I need to work out in a gym. That's all there is to it. It's so funny; now that I am physically back in a gym, the gear has already begun to click. I like a routine. Structure is good for me.
Anyhow, Ang worked from home today, so we walked over to pick up Ali from pre-school together. Ali lit up live a firecracker when we both walked in! Ali's teacher immediately walked up to us to say how well she is adjusting, and how amazing her vocabulary is! "She is just like, "Here I am!"" was how Mrs. Frazer described Ali's confidence. Nothing makes a parent prouder than when a teacher beams, right? LOVE it. Ali is so excited also because she is going to the same school daddy went to! I took one class during my degree at this community college, so Ali thinks we've gone to the same school now. It is too cute. It works for me; I LOVE having her imprint COLLEGE in her mind at such an early age!
Thanks for the continued questions about the trial! Really great stuff! Let me address some of them for you:
What happens after BMT? Well, BMT unfortunately never really ends. LOL. What I mean by that is that there is follow up care that may be as infrequent as annually if all goes well and no side effects are present. Really, in this case, there is a study to monitor the changes in skin 100 days after transplant, so that is why there is a lid on this study. The patients still need to come back for biopsies every year. The whole initial concept is that the Collagen VII will continue to grow into wounded areas over time, so the more wounds that continue, the more healing with working collagen.
Here's the thing though: the transplant as it exists now does not "Cure" anyone of EB. It simply ameliorates the disease into a functional, treatable condition, more like dominant dystrophic EB. The skin gets better, but there are always a couple of wounds. This brings up another important point. The older the patient, the more likely that they have bacterial infections that have left permanent colonies in the tissue, almost like TB. So, when the body gets stressed or depleted, they colony can flare up or break out. This is most likely why the real problem wounds for many kids NEVER heal... because of the permanent residents in the tissue preventing the growth and healing to occur. THIS is another reason why we need the funding to get to the next generation of BMT as well as systemic gene therapy.
Kidney issues are not uncommon for BMT, but they are more common in EB. Again, prior to U of M studying EB, it was primarily studied by dermatologists, not nephrologists. There seems to be some sort of inflammation that occurs internally at a much greater level with EB, and again, the docs aren't sold that collagen VII only resides in the skin.
Yes, we were able to see dramatic differences in Bella's skin after transplant. See Aug 2 and Aug 5 posts for some good before and after pictures.
Last, the doctors change the protocol roughly every 7 kids. The doctors continue to do everything they can for EJ, McKenzie, and Sam. The one thing all us parents in the study knew from the get go was that there were no promises made, and we all rolled the dice. The thing is, it's not like it was a walk in the park for any of these kids before hand. BMT is so complex, and frankly, still so barbaric. You just pray your child sneaks through, but it's like 10 kids running through a mine field; you KNOW that a certain number of them aren't gonna make it to the other side. I remember one EB parent offering their condolences to me after Bella died, and I honestly wasn't sure who should be consoling who. Their child still had EB, was still suffering from it, and was most likely still going to die from it. At least Bella was in no pain.
The thing that sometimes works at me is that Bella was truly a happy girl most of the time. She didn't have any persistent, nasty infections. Her bath time was still relatively peaceful, as she didn't have a lot of nasty wounds that just wouldn't heal... yet. We figured, she's as healthy as an ox, and at only 1 years old, if we go through this now, she'll have virtually no memory of it, and we'll treat it before it gets worse, which is the only direction the arrow moves with recessive dystrophic EB. Sometimes I wonder, what if we didn't go through with it? Would we still have her? Wouldn't THAT have been better?