My optimism is taking off with new Friedreich’s ataxia clinical trials
When the news about an upcoming pediatric trial for Skyclarys caught my eye
During a recent layover at O’Hare International Airport in Chicago, I learned some unexpected news about an upcoming pediatric clinical trial for Friedreich’s ataxia (FA), the disease my 12-year-old daughter, Amelia, has.
My flight to Chicago had been delayed by weather, which I find curious because the climate is always doing something. It might be sunny or rainy, but I guarantee that on any given day, weather is happening.
Our plane eventually took off, and I landed in Chicago just as my next flight was departing without me. If you’ve been in that situation, you know what happens next: long lines of frustrated people with lots of emotions as travelers realize they’ll miss important life events. As I stood there with two strangers, they asked how I could be so calm. Without thinking, I responded, “I have almost no control over any of this. But my attitude? That’s all me.”
The weather, a lack of available flights, the fatigue as the night wore on, and the inability to get a one-way rental car to drive home were all aspects of that moment that I had little ability to influence. So I took time to dig into a book and listen to soothing music while I held my space in line. I tried hard to keep the situation from controlling how I felt.
Now boarding …
To me, an airport feels like one big metaphor for FA research. It contains excitement about trips, fear about flying, unique passengers in different places, and people feeling both hopeful and overwhelmed. Sometimes others get through the process faster or obtain a seat on a flight while we’re on standby. Conversely, sometimes we’re granted a seat while others are left behind. There will always be delays that can’t be avoided.
I mention all of this because information has been made public about a pediatric trial for Skyclarys (omaveloxolone), the only medication approved by the U.S. Food and Drug Administration for Friedreich’s ataxia. I read over the announcement while my flight was delayed.
A lottery process will be used to select participants who meet the eligibility requirements. Continuing with the airport analogy, some children will get into this trial (and others), which will be critical to moving the process of finding new treatments forward. Others won’t be selected, which might be hard for them and their families to accept.
But the bottom line is that this trial is something we in the FA community have been begging and advocating for, for years. So while I was excited, my enthusiasm was tempered by the fact that participation is limited and the drug might not even work well for everyone — just like a flight, which has limited seats and must face possible inclement weather.
But more planes (trials) are coming. Exciting announcements regarding nomlabofusp (CTI 1601), an investigational drug that seeks to ease FA symptoms, keep popping up in my email feeds. I feel both hope and a sense of urgency. I want life events to be possible not only for our child, but for all of the young children with FA who are waiting for access to treatment.
My hope in all of this process is to continue to do what I can to help move things forward by supporting the Friedreich’s Ataxia Research Alliance (FARA) and our FA family at large. I also want to continue encouraging myself, Amelia, and others in my family to live the best life we can, even when things get frustrating.
I’ll celebrate those members of the FA community who are seated on the plane of their choosing, knowing that our work today will make tomorrow much brighter. We’ll weather all of it, together.
A final note: If you or your child aren’t registered with FARA’s patient registry, please consider taking the time to do so.
Note: Friedreich’s Ataxia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Friedreich’s Ataxia News or its parent company, Bionews, and are intended to spark discussion about issues pertaining to Friedreich’s ataxia.
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