Today we got some very interesting news that made my heart skip a beat. As many of you know, we have been trying to make a decision about whether or not to enroll Connor in a drug study at Washington University in St. Louis. The theory is that this drug would help stimulate the gene that "backs up" the gene he is missing to produce the protein the cells in his spine need. The drug was FDA approved for sickle-cell disease, and didn't have any dramatic known side effects besides the fact it sometimes makes people smell fishy. Our dilemma was that it has a horrible, horrible taste and he was going to have to take it with very little to disguise it, every 8 hours for about 3 months. That means waking up in the middle of the night to put something nasty in his mouth, and then trying to get my 2 year-old to go back to sleep peacefully. (Riiiiiiiiight.) And then there were the 8 or so 5-hour (one-way) trips we would have to make to St. Louis in that time period.
In short, it was really a dilemma. On one hand it all sounded like 3 months of horrible days, and on the other you wonder if you are keeping your baby from one of the few possibilities you have that might actually help him a little bit retain some of what he still has. We had prayed and prayed and talked to everyone we knew and still come to no conclusion. We finally decided to see one more doctor next Tuesday and then we would make a final decision.
Then today, I got "the call." I expected it to be the call from St. Louis saying now that Connor was two, a slot was open for him in the study and we needed to get there as soon as possible. I had been expecting that call for a week or two. But what they actually told me was quite different. The therapist there said they received an email from the drug maker that they were closing the study to SMA Type 2 and 3 patients because those patients were having so many adverse reactions.
I hung up the phone and my first reaction was relief--now after two months of worrying about this the decision was taken away from us and we didn't have to even decide at all!! Then I thought about how it was sad this option was taken from us, but hopefully there would be more, and better safe than sorry. And then the "sorry" started to really sink in deep, and I realized just how close we might have been to doing something to our little boy.
Sometimes all this SMA stuff really sucks--a lot. What might really help just happens to be the same thing as what might really hurt. So you can do nothing or you just have to gamble with your child's health. How incredibly grateful we are that we have the Holy Ghost to guide us. We would never take any chance without a firm answer that it was the right step for Connor, and how grateful we are today that no answer ever came.