I am a freelance columnist for Friedreich's Ataxia News. I was recently published on my column, My Darling Disability, and I wanted to share it here, too. You can either read it by following this link, or just keep scrolling below.
Note: This column describes the author’s own experiences with Skyclarys (omaveloxolone). Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy.
When I was diagnosed with Friedreich’s ataxia (FA) in 2013, I was incredibly overwhelmed, to say the least. I’d never even heard of FA, let alone planned on waging a lifelong battle against this cruel disease. The worst part was being told there was no actionable hope. There weren’t any treatments then, and none foreseen on the horizon: There wasn’t anything publicly celebrated in the late stages of clinical trials, nothing awaiting approval from the U.S. Food and Drug Administration (FDA).
Being handed such a debilitating diagnosis without any hope to fight it felt isolating, even suffocating. I was forced to digest an incredibly bleak future of heartbreaking medical hardships, disabilities, and eventual death, with no tools to aid my battle or provide any kind of advantage.
Fast forward to 2018, when Reata Pharmaceuticals was recruiting for Phase 2 of a clinical trial that the FA community was optimistic about. In August 2018, I started the 48-week, double-blind, placebo-controlled study of omaveloxolone, knowing that my participation was in the name of science rather than a guaranteed access to treatment.
Even though everyone around me expected that the cure for FA would be a cocktail, we hoped that this drug would be a vital, results-showing, miraculous first ingredient that could at least slow my progression. My village waited with bated breath to see if this drug was “it” — the drug that would finally combat my DNA.
Now that I have been on the drug for nearly five years, it’s hard to put into words my thoughts. Since I don’t have two Kendalls living with FA, one on the drug and one not, I have trouble identifying its true impact on my progression.
Since my participation in the trial began, my FA-related symptoms have progressed and new symptoms have begun. As of February 2019, I’m 100% dependent on a mobility aid. I use my walker for every single move I make, aside from the times I depend on a wheelchair or electric scooter, when the terrain is tricky or taxing. Peripheral neuropathy is a debilitating and daily nuisance. My speech is slow and slurred. My fatigue is crippling.
With all that racing around my mind, it’s challenging to paraphrase my feelings about the recent FDA approval of my medicine, which now has the brand name Skyclarys and is our first FA treatment. I think about the hope this development would’ve given 2013 Kendall. I think about the validation it would’ve given 2018 Kendall — that all of the taxing requirements to participate in a trial halfway across the country were eventually going to prove themselves worth it. And I think about what it means to 2023 Kendall, too.
I celebrate the success with Reata and pat my fellow clinical trial participants on the back, full of gratitude and excitement. I’ll continue taking Skyclarys every morning, praying that this drug will work to slow my symptom progression today and every day.
I can’t stave off the creeping thoughts that twist my mind into a state of worry about how FA’s move into the category of treatable will affect future investments by the scientific community into treatment research. But I’ll continue doing everything in my power to help my community achieve that next ingredient in our curative cocktail. I’ll continue fundraising and advocating for our future, celebrating this battle victory while keeping in mind that we’re still at war, fighting for a cure.
I desperately pray that everyone continues to pursue the next advancement with urgency as we keep taking our Skyclarys every single morning. I remain full of hope that it’s having a positive impact while I patiently wait for the next big announcement.
“I have fought the good fight, I have finished the race, and I have remained faithful. And now the prize awaits me.” — 2 Timothy 4:7-8 (New Living Translation)