As we reach the one month mark of Blaise’s trial, I have certainly learned a lot more about what to expect with being part of a drug trial. I thought I had an idea of what it would be like, but then it actually starts and I’ve learned so much that I hadn’t even considered before! My hope with these blog posts is to not only update everyone on Blaise’s progress with AT-007 (which is VERY important, don’t get me wrong) but also to explain some of my perspective as the parent of a child in a trial. I assume some people reading this blog will be in my shoes someday, if they are not already, and I think it is important to understand it is not all rainbows and butterflies. There are highs and lows, just like any other chapter in life. However, Carl and I believe this is the best thing we can do for Blaise at this point in time, so here is our experience!
So far in the trial, Blaise tolerated the 1 mg/kg dose very well. With the initial dose, (and every subsequent dose escalation, and a safety check 14-21 days after any dose escalation…), we spent a pretty long day at the hospital for blood draws to check pharmacokinetic levels. Some of you might be wondering….”Kara, what the heck are pharmacokinetic levels?!”. This simply means Blaise gets lab draws 4, 8, 12, and 24 hours after a dose is given so we can see what the drug levels in his body are, and see what his exposure to the drug is. On these “long” days, an IV is placed to result in less pokes throughout the day. Blaise is a pretty little guy, but man, he turns into the HULK when trying to draw blood. I am assuming other rare disease parents can relate! I have noticed during this trial so far that Blaise doesn’t mind the needle poke so much as he gets upset about being restrained during the process. I have quite the body contortion routine going on to try and keep him still. I will say there has been one time so far where my mama heart hurt watching him be so sad during a lab draw, and I questioned if this was the right thing. However, Blaise always recovers so quickly and is smiling as soon as you let him go. And I feel like I have to think about the long game, and the potential impact this can have not only on his life, but the lives of others. So we keep calm and carry on!
Back to the good news…Blaise’s labs looked good to dose escalate so we received the green light to move to 5 mg/kg!! Wahoo!!! Now we insert our first “TRUE LIFE: I’m In a Clinical Trial” episode. Blaise started school for the very first time the Monday after he started the trial. Good news is he absolutely LOVES school and went right to his teacher the first day and waved “bye” to me. Unfortunately, as you might expect when entering the petrie dish that is 10+ two year olds playing together, he brought home some kiddie crud. The night before we were supposed to go up to UNC for his dose escalation/increase, he had a fever and was pretty lethargic. *SIGH*. However, as Dr. Jalazo says- “Blaise is a kid, not a robot” and was so kind and understanding. Not only did she check in with us on how he was feeling, but luckily, she was able to reschedule our long dose escalation visit for the following week, so we were still on track!
I need to take a moment for a shout out….I cannot thank Dr. Elizabeth Jalazo enough. She is Blaise’s geneticist at UNC. I call her Superwoman very often (I am running out of Superwoman gifs), and she really is. She has a very unique background in that she did a genetics fellowship because she is a rare disease mama herself, and wanted to become a geneticist to make things better for rare patients. And boy is she! She lives both sides of this life, as the doctor and as a clinical trial mama. Her perspective cannot be beat and she is such a fighter for her patients. She has been such an advocate for Blaise over these past months and I am so blessed she is in our lives!
Okay- back to the trial. Blaise has been on 5 mg/kg dose for over a week now, and all of his follow up labs indicate that the drug at this higher dose still appears to be safe, which is the most important thing! Now we are halfway to what is believed to be the full dose of 10 mg/kg, which hopefully we will be able to increase to within the next month! More to come on that!
