Clinical trials are great…unless they leave you dizzy, sobbing, paranoid, and not sure if you are hallucinating.
This is not the topic I want to be writing about. I should be polishing off and posting my marathon race report, or attempting to convey our life-changing experience at ALS.net’s gala last Saturday night (posts coming soon!), but I can’t seem to focus on either of those assignments until I clear the submarine that almost sunk my battleship last week. And just like the game of Battleship, this attack wasn’t even on the radar…
DP and I learned a lot from the training (and met many compassionate, committed researchers and made a ton of new friends!), but here were our 3 main takeaways relevant to this Battleship story:
- You need to know that the primary goal of the trial is to advance ALS research. It seems like cruel irony, but people with ALS typically only have 2-3 years from their diagnosis date to enroll in a clinical trial. If you’re still alive after that point, you’re usually excluded from trials because it’s assumed you have a slower progressing form of ALS…in other words, the researchers can’t tell if their medicine is working or it’s just YOU.
- You need to be an informed consumer. Understand the basic science behind the trial, what phase it’s in, and what the drug is supposed to do. Understand that this may be the only shot you have at a trial to save/extend your life, and even then, you might get the placebo.
- You can drop out of the clinical trial at any time. If you do though, the resources and potential scientific advancement is lost. But it’s YOUR life and the only one you have…
Enter Tirasemtiv (the Submarine)
The training was perfectly timed for me because the day after I got home, I started a clinical trial for tirasemtiv* – a drug to strengthen the lung muscles, since respiratory failure is the actual cause of death for most people with ALS. Early trial findings also showed increased strength in the arms and legs (to which I was like, “yay, bring on the marathon PRs!”). This is a Phase 3 trial so if it all goes well, is likely the last hurdle before FDA approval.
[*For the record, this would be the second drug approved in the US for the treatment of ALS. The first, riluzole, statistically extends life a whopping 2-3 months.]
According to my neurologist and Patients Like Me, some people in the Phase 2 trial experienced negative side effects – vertigo, nausea, lightheadedness, confusion, headaches, and stomach cramps/GI issues. As a result, Phase 3 is designed to test for the side effects first. Everyone gets 250mg of the drug for the first two weeks. If you can tolerate the side effects, you are double-blind randomly assigned to take 250mg, 375mg, 500mg, or the placebo daily for a year with periodic appointments to test strength and vitals. In other words, 75% chance of getting the medicine…
Okay, sweet. Let’s do this thing!
I felt fine on Monday when I started the drug.
By Tuesday, I felt like I’d taken too much NyQuil or Advil PM but was still trying to stay awake. I had some mild side effects when I started other medications too, so I wasn’t really alarmed.
By Wednesday, I asked my acupuncturist to forgo the treatment she’d planned to do on my too-tight hamstrings in favor of an anti-vertigo treatment. I also cancelled a trike/run date with Ashley (the REAL sign I was feeling bad!). I convinced DP to pick up takeout instead of cooking dinner so I could stay curled up on the couch to minimize the world spinning.
By Thursday, I woke up even more foggy, groggy, dizzy and sluggish. But my friend Angela had secured for me literally THE LAST appointment available in the tri-state area at the DC Social Security office during Medicare open enrollment. DP’s open enrollment deadline at work was the next day too. In short, it was the single best shot I had to untangle the health insurance mess that has plagued us since June*…and position us to be able to order my power wheelchair sometime in 2016.
[*Side note: I cannot be the first person in America to have applied for Medicare while on a working spouse’s health insurance. Why is this so hard??]
So I had to go. And because I hadn’t anticipated the severity of the tirasemtiv side effects, I was stuck driving myself to/from the appointment, alone. Nothing bad happened – no honking, weaving, or close calls to signal that I was actually driving badly – but the vertigo was so bad that it was all I could do to concentrate on the road required a lot more concentration*.
[DP and I had a loooooong conversation about this paragraph. In retrospect, it was a mistake for me to drive. I have promised him that I will give up driving when it is no longer safe. I am still a good driver, but not on this medication. I’m sorry, DP!]
I arrived at the Social Security office early for my appointment and saw one of those handicapped automatic door openers beside the entrance. Whew, I thought, such a relief to be at an ADA-compliant federal agency. After all, it was Social Security whose existence is to serve the needs of elderly and disabled people.
But as I got through the automatic door with my walker, I realized there was second door to get into the office – this one with NO automatic door opener and NO windows for anyone on the inside to see that I needed help. There was nothing I could do but oh-so-carefully pull the door open while backing up, and then gingerly wedge the walker (and myself) through the doorway to avoid falling.
Someone motioned me to an empty seat on the other side of the crowded room. While I tried to figure out what I was supposed to be doing, the security guard standing near me asked the entire room, “WHO HERE IS A REDSKINS FAN??” and proceeded to carry on a lively discussion with those who answered. This struck me as so bizarre that it punched through the fog to reach my vertigo-addled brain. Wouldn’t he be more effective standing near the door to help people get inside and figure out where to go? I wondered.
I was called before my appointment time, which would have been nice but Angela, who was coming to help navigate the bureaucracy, hadn’t arrived yet. In my slurred ALS speak, I tried to explain what I needed to do with Medicare to which the Soc Sec woman said, “oh? Is that all? Here’s the form. But your other insurance provider needs to initiate the process.” Wait, huh? FOUR other knowledgeable people (the ALS Association rep, DP’s insurance provider, a Medicare insurance consultant, and the wheelchair company rep) had told me the exact opposite. Fortunately, Angela showed up then and I made the woman re-explain it to her to make sure I’d understood her correctly in my current state of delirium.
Throw in a bunch of other random “hits” (to stick with the Battleship game analogy) like a bomb threat that snarled traffic downtown, the flash of a speed camera I saw in my rearview mirror, a black squirrel who sprinted through the legs of my walker chasing a grey squirrel, our beloved-but-rambunctious INDOOR cat Tango getting outside where I couldn’t catch her…and, well, you can see why I ended up sobbing, paranoid, and convinced I was hallucinating by the time DP got home from work at 9pm.
In short, a bunch of the small boats of my sanity got torpedoed.
Some of this cannot be blamed on tirasemtiv, but my ability to cope with what happened can.
Despite it all, including the 11pm pizza delivery and 1am online health insurance enrollment, we made our 7:30am flight to Boston on Friday morning to attend ALS.net’s Leadership Summit. By 10am, I was fully submersed in 6 hours of scientific lectures, panel discussions on clinical trial design, and an awards ceremony.
But the physical side effects had caught up to me. Now compounded by lack of sleep, I faced the still-worsening vertigo, drowsiness, slurred speech, and – for the first time – an inability to overpower my foot drop. I did a 26.2-mile marathon in my trike two weeks before and now I was too unsteady even to walk across the room by myself with my walker.
I emailed the tirasemtiv clinical trial coordinator and asked her what I should do. She called me a couple of hours later and I said something along the lines of “how do I know how much is too much? I don’t want to drop out of the trial, but I feel awful.”
“It’s up to you,” she told me – a fair, true, but completely unhelpful answer.
I was in bed in our hotel by 6pm. I slept but woke up for takeout DP brought just as the Paris terror attacks were unfolding. I wondered again if I was hallucinating. Again, I drifted off to sleep.
At 4am, I woke up with the clearest single thought I’d had all week, I’ve had enough.
Googling more about the side effects of tirasemtiv, I found this article on ALS-TDI’s website with findings from the Phase 2b trial. “Tirasemtiv may help reduce the decline of breathing capacity and muscle strength of some people with ALS,” the headline read. “But tolerability of the drug remains a considerable obstacle going forward.”
Apparently, more than 100 patients out of 711 dropped out in the first week of the study due to an inability to tolerate 250mg of tirasemtiv – the same dose I was now taking. If I stayed in the trial, I’d have a 50% chance of being randomly selected to get 1.5x or 2x that dosage. Moreover, 3x the number of participants dropped out of the clinical trial compared to those on placebo. And that trial was only 3 months – my trial was a year.
Hit. Hit. Hit. Hit. Hit. Hit. Sink.
It’s over, I thought. Thank goodness it isn’t even a close call.
It took a few days, but I’m back to feeling my ALS normal.
I’m grateful to the NEALS research ambassador training in Clearwater for teaching us so much about clinical trials and HOW to be an informed patient. I haven’t been scared off from clinical trials – I’m eager to try again before my eligibility window closes. But I know now that I need to do more research on the front end.
I encourage other people with ALS to enroll in the tirasemtiv trial. I pray that it works for you. I pray that it works, period. I also would strongly encourage you to apply to become a NEALS research ambassador. We need more ALS patient participation to advance the science and cure this damn thing. But nothing is more important than your battleship. It’s YOUR health and YOUR life and it’s the only one you’ll ever have.
(And I will never be able to look at pharmaceutical commercials with their long list of side effects the same way again. Ever.)
Wow. What an overwhelming week. And applying for medicare, grrr. And that entrance and that security guard, grrrrrr!!
Much love to you, woman.
I 100% support you in your decision to go off the trial. I am really proud of you for making the best decision for YOU, not anyone else =).
Holy moly, that's a lot to handle.. Glad you're back to feeling normal again <3
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