I started a double-blind placebo-controlled 48-week clinical trial in September 2018. After the 48-weeks, I enrolled in the open-label extension. With this, I am definitely getting the study drug and must travel to Atlanta every 6 months for an appointment with the trial coordinator and doctor.
At these appointments, I update consent forms, fill out questionnaires, and report any adverse effects, injuries, or new diagnoses. Then I do bloodwork, a vitals check, and a neurological exam with the doctor. This is a whole slew of tasks that measure the deterioration of my coordination, dexterity, speed, and strength. In other words, he assesses how my Friedreich's ataxia symptoms have progressed in 6 months. Then I get the next 6-month supply of study drugs and fly home.
I've been taking 3 little white pills every morning for nearly 4 years now, and the big question everyone always asks is, "is the drug working?" to which I answer, "I don't know."
Since starting the trial, I have progressed across the board. I am now 100% dependent on mobility devices, my speech is slower and more slurred, I am weaker, my fatigue is exponentially exaggerated, and my upper body dexterity is worse. I don't know if my symptoms are better, worse, or the same as they would have been if I wasn't on this drug. There is no way of knowing. I don't know if the FDA will approve this drug. I don't know if this drug will help the FA community.
Here's what I DO know: -This drug is not enough. The cure for FA will be a cocktail of treatments, and this drug is just one small possible ingredient. We have a long way to go. -"Because He lives, I can face tomorrow. Because He lives, all fear is gone. Because I know He holds the future, and life is worth the living just because He lives." While my future is unknown, I know that my tomorrow will hold FA. But I can face tomorrow because I am not facing it alone! My way-maker, miracle-worker, promise keeper, light in the darkness GOD is holding my hand and walking with me.
