From Hospital to Beach and Back

By December 26, 2014 November 27th, 2018 Tracy's Blog
Double down, go for broke, swing for the fences… we’re doing it for Charley who does it every day! Case in point: the double loin chop he attacked with gusto last night. I was going to write this blog entry about Charley’s recent trip to Baltimore for the HT-100 clinical trial visit. But more than a week has gone by, and now I’m (thankfully) sitting on a beach watching waves crash onto the sand. My present surroundings make last Friday’s 8-hour medical marathon feel blissfully distant. I’m sure I could recount most of the details of Charley’s interminable day at the hospital: two blood draws, one cardiac MRI with IV contrast, two hours of physical therapy evaluation, six minutes of walking up and down a hallway to see how many meters he could go, one 15-year-old best friend sitting there for 8 hours straight keeping Charley company. But considering the glorious sunshine and invigorating sea air, I’m not really in the mood to dwell on that story today. In fact, I may even get off easy this time because Ethan made a video of the trip on his iPhone so hopefully he will let me post that when it is ready. I’m sure that video will tell the story of Charley’s clinical trial visit way better than I ever could in words.

So let me tell you about Florida. We are here to visit Charley’s grandma who has Alzheimer’s disease. In between visits we are fitting in beach and pool time, fun with older cousins, and some great restaurants. One cousin, a college senior, just landed a coveted job at IBM for when he graduates. That of course sparked a lively conversation about the future amongst all the kids. Charley wants to “be in finance” and live in New York City with a house in the Berkshires.

To get through those conversations without passing out from sadness, I wall off a part of my consciousness. The part of me that knows Charley has Duchenne and that his projected lifespan is 20 years. The part of me that knows so many teenagers with Duchenne who are immobile, who have breathing tubes, who are in and out of the hospital every other week. The part of me that knows Charley’s job might very well not be working at IBM or a finance firm but rather getting through each day with as many pain-free moments as possible. A garage door in my brain zips down and separates all that from the moment, and I can laugh and daydream with my kids about the future.

I know that garage door would not work so well if I were facing a true dead end with no path forward. If we had no chance whatsoever of changing the prognosis, I would likely have to leave the room for a good hard cry every time Charley talked about what college he wants to go to (“one with parties every other weekend, because partying every weekend would just be too crazy and I also care about academics.”) But last week’s hospital visit reminded me that our path does not lead to a dead end. In fact it forks a few different ways, with possibilities in a number of directions. I’m bringing you back to last week’s hospital visit now, but bear with me – it’s for a good reason!

Our clinical trial coordinator was literally running around the hospital all day from patient to patient — she is coordinating four different clinical trials for Duchenne at that site. Charley is participating in a trial to test HT-100, a drug that could delay or prevent the buildup of fibrosis (scar tissue) in the muscles. In another clinical trial Pfizer is testing a myostatin inhibitor, a drug that takes a different approach to delaying Duchenne’s relentless progression. I ran into an acquaintance whose son was experiencing his first day in that trial. The two other clinical trials at Kennedy Krieger/Johns Hopkins are testing “exon skipping” medicines that induce the body to produce dystrophin, the essential muscle-building protein that kids with Duchenne are missing.

Watching the clinical trials coordinator race around from child to child helping out with the tests and administering the medicines was truly a marvel. When Charley was diagnosed with Duchenne ten years ago, there was not one clinical trial for a promising treatment. Today, it is not crazy at all to think that one day soon, all kids with Duchenne could be taking at least one — and hopefully even a combination — of new medicines. It is entirely possible that we can keep Duchenne at bay so Charley and thousands of kids like him can go to college to party every other weekend, get real world jobs, find love, and have kids of their own who will sit around a table and day dream about the future.

I hope your New Year’s celebration is filled with laughter and love!