Trial and Error?

“Is that your son ma’am,” a sheepish, young rent-a-cop asked me as Liam joyfully sprinted through the fountains in Sundance Square in Fort Worth, Texas.

“Yes, isn’t he adorable?” I said, grinning.

“I’m so sorry to have to do this but you aren’t allowed to play in the fountain after 6.”

I blushed having assumed he came to gush about my kid and said, “Oh, I’m so sorry we didn’t know.”

The next day, he would start his trial drug, which meant a long day of testing at the hospital. We had the pulmonologist for general DMD assessment (not related to the trial) late in the afternoon at the same place the day before. Liam and I decided to get a beautiful hotel in downtown Fort Worth, right near the fountains.

Dad said Liam would have loved telling his friends he got arrested.

We went country, when we’re typically a bit more rock-n-roll

We decided to head to the Stockyards, an area of Fort Worth straight out of Urban Cowboy. Please tell me you’ve seen this Debra Winger movie?

Old brick buildings with shops that sell over-priced cowboy boots and hats to tourists, country bars advertising LoneStar from women clad in Daisy Dukes and animal stables filled with horses, cows and, of course, longhorns adorn the cobblestone streets. Twice a day they run stock right down the road. Trust me, we live in Texas, but this isn’t at all what we experience in our daily lives.

We picked the oldest steakhouse in the area – Cattleman’s. The decor – wood panel, brick and pictures of cows – I assume hasn’t changed since the 80s. The servers wear wranglers, boots and country shirts. The biscuits are to die for, the steaks are huge, and the potatoes are smothered in delicious things.

I had some wine and a big ole medium-rare steak. Liam had chicken tenders.

We talked about Fortnite. We gossiped about school. He told me about the new house he’s building in Minecraft with lots of windows.

He told me that he wants to be an artist when he grows up. My fears crept in – what happens when he can’t lift his arms someday. But I swallowed it.

We had to go to sleep eventually

I think of life a bit differently now. I’ve always loved moments in time, but I don’t think I was hyper focused on trying to keep those moments from ending. I didn’t want to go to bed, and I was the responsible one. I wanted to talk to my son all night. But, he passed out in my arms and I shed a few tears on his sweet little dirty blonde hair. How can I love someone this much? We slept hard that night, an unexpected peaceful sleep for me.

A harmful thing people say when you find out your kid has a chronic disease is “no one knows when they’re going to die.” Having a DMD diagnosis is like starting a kitchen timer but on a six-year-old to find a cure soon enough before too much damage is done.

We must make life-altering decisions about our young children – like the one Collin and I had to make whether to put our kid on a trial drug that could delay traditional interventions for a year if we get a placebo. What if we’re wrong?

They are getting so close to a cure people tell me, but what if it’s not close enough to help my son. It’s such a hard thing for me personally to bridge between hope and facts, and that is what keeps me up at night. What if my hope, what if my faith in a miracle isn’t strong enough? What if all the doubts, worries and fears in my head stops our miracle? So many stories hit the news about miracles, but what if ours never comes? (oof that sounds like my anxiety spilling out)

Access to care is complicated

The next morning, I went to our typical place to check in – the self-serve kiosk – and it said in big red letters you can’t check in because we’re in a trial. I thought, I’ll just go up to my research coordinator and discuss it with her since she told me to do that with any problems. Last time admissions made us pay $300 or they wouldn’t let us up to the clinic (for something covered by the trial costs) and she said, next time get me with any problems.

The front desk lady at the clinic said, “you didn’t check in downstairs.”

Me: “Well it said I couldn’t check in at the kiosk and we had a lot of trouble last time at the desk since we are in a research trial. I thought it was easier to just pop up here and talk to our research coordinator. She said if we ever have trouble to just get her.”

Her: “Well I don’t know what she told you, but you have to check in downstairs every time. Didn’t the kiosk say to check in with the front desk?”

Me: “Yes but Kayla, our research coordinator, said if we ever have any problems checking in to come get her since we were charged last time at the desk. So, I’m here. Maybe y’all could work with them so they know how to handle people in trials?”

Her: “That’s not my problem. I can get you that manager’s information if you want. They don’t report to us.”

Dad and Collin intervened and gently pulled me away before I exploded right there. It’s not her fault that process is messed up but having worked for a children’s hospital for 10 years, it’s absolutely her problem and she absolutely should note that concern from a parent and send it to the appropriate person. Collin was his charming self and she checked us in there.

And, it was time to do all the things and get our drugs.

Liam ran through the PT tests quicker than expected, and Dr. Marks was able to pop in and do his piece – just checking him over like doctors do – a bit early. The drugs weren’t ready yet – so we adjusted our plans from taking two doses there (three hours apart) to only one dose there. Kayla is awesome about doing her best to be efficient with our time.

I won’t detail it since it was the same issue as above when we went to Radiology. We had to check in to two different desks, and one of the ladies tried to charge us for the damn trial assessment again. I said I’ll call Kayla, my research coordinator, again as I’m instructed to do.

 The woman goes “I don’t need her on anyone’s help.”  

Me, my jaw set tight: “Yes, but you’re trying to charge us for things covered in a trial, paid for by that trial. So, I’m just going to call the person with information about the trial.”

Her: “I don’t need her help”

Collin intervened again, and I walked away.

We had just been to lab – the process is a simple as possible. The staff is super friendly, and they rushed Liam in, so we could make that very radiology appointment. (apparently in radiology, they must book an appointment for a trial like they would a normal appointment so that led to the confusion – the confusion the check in person didn’t care to clear up. And, noting all these different services have different processes and there’s no effort to streamline them. They just expect parents/patients to keep up with them.)

Radiology got a little uncomfortable when the tech taped Liam down with medical tape so he would stay perfectly still for a bone density scan. It’s not like incredibly tight – it’s more so that he can feel any movement and understand when the tape moves he moves. This scan is like three minutes long – and that’s a long time for a 6-year-old to lie perfectly still. But obviously as a mom you want to say hell no, you aren’t taping my kid to anything. And like usual, Liam took it like a champ and thought it was funny, at first at least. The tech was fantastic – and that’s why I take issue with access to care. The complicated processes to access that awesome tech is stupid ridiculous.

A few hours passed, and we got our drug.

And, now Liam takes 10 pills a day with eight grams of fat with each dose to help him absorb it. He takes it no problem at all, like it’s a normal part of his day. We don’t know if we are getting a placebo. Liam said he thinks we got the wrong medicine, but Collin feels he’s seeing some improvement. Liam tells me he just wants to fix his legs and the medicine isn’t fixing his legs. Those chats are heartbreaking, and I try to not show my devastation. I want to say that a wheelchair isn’t the worst thing. People live happy, full lives with disabilities, even though it’s hard, people do it. I want to say keeping you healthy and alive is what matters most to me and a wheelchair will just help you get around. But, he’s six. He wants to run, jump and play like the other kids. He doesn’t want to have his mom’s help to climb the stairs at the zoo. He doesn’t want to be the only kid who isn’t in trouble for climbing on rocks because he can’t. He wants a miracle, and I don’t know if I can give him that, and that sucks in ways I can’t describe.

I don’t know what I feel other than I just want this year to end and roll into open label, so I know for sure what he’s getting. This not knowing is torture. I’m not saying knowing means anything different, but it helps me make decisions about what I’m seeing in him. And maybe, this drug will be the thing that keeps him on his legs for a few extra years.