Are the Potential Treatments for FA in 2022 Worth Celebrating?

Matthew Lafleur avatar

by Matthew Lafleur |

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This year is an interesting time for those with Friedreich’s ataxia (FA). There are plenty of reasons to be hopeful for a treatment for FA, more than ever before. Finally, the first treatment seems on the horizon for this damned degenerative disorder.

However, it’s important that the FA community be realistic in these exciting times. Although a treatment seems likely to happen, by no means is one guaranteed. Also, it’s important to distinguish between a treatment and a cure. A treatment may stop or slow disease progression, but likely won’t reverse the damage already done to FA patients’ bodies.

I’ll never forget a post on social media from an FA patient during a discussion about potential treatments: “Don’t put your wheelchairs on eBay yet.”

I laughed when I read this. Although many FAers, including me, hope that one day we’ll no longer need a wheelchair, setting informed expectations is wise and less disappointing than wishful thinking.

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Even with these tapered expectations, the FA community has the right to celebrate. Though FA has no approved therapies, a glance at the treatment pipeline by the Friedreich’s Ataxia Research Alliance (FARA) shows 14 potential treatments as they undergo preclinical and clinical trials and trickle ever closer to approval and availability to patients. Fourteen! Compared with the zero currently approved therapies for FA, this number is staggering.

Three proposed treatments on the pipeline are especially worth noting:

  1. Omaveloxolone by Reata Pharmaceuticals was the first potential treatment to show significant effectiveness in treating FA patients, following the results of Phase 1 and 2 clinical trials. No other treatments have made it this far down the pipeline. It is currently being evaluated by the U.S. Food and Drug Administration and may receive fast-track approval soon.
  2. As announced on Rare Disease Day in February, a new investigational drug that seems promising and exciting just received the green light to begin clinical trials. This drug, DT-216, was explained in a webinar to the FA community thanks to FARA and the drug’s creator, Design Therapeutics. A recording of the webinar can be seen here. See the latest Friedreich’s Ataxia News article on DT-216 here.
  3. Gene therapy for FA patients may become more of a reality as Lexeo Therapeutics will begin testing its LX2006 later this year. LX2006 will target cardiac function, which is weakened in most people with FA. Read the latest Friedreich’s Ataxia News article on LX2006 here.

Those are only three promising developments of the 14 currently listed on the treatment pipeline. Considering the lack of any available treatments in this disorder’s lifetime, we in the FA community have the right to celebrate and do a little happy dance at the sudden surge of possible treatments. Our happy dance may be a little awkward in our wheelchairs, but who cares?

It’s true FA remains untreatable and we still have to see if and when these investigational treatments get approved. And even if any of these potential treatments are approved in 2022, they won’t be the grand miracle FA patients are hoping for.

So I won’t put my wheelchair on eBay just yet. But it feels like we are finally reaching the end of the first lap in a race, even if we’re not close to the finish line: a cure for FA.

The end of a long and grueling first lap isn’t a miracle cure, but it is a little victory. And that’s worth celebrating.

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.


Karina Jeronimides avatar

Karina Jeronimides

Some of us are dying. I have been hoping and looking at the "promising" horizon for 30 years. I am lucky to be still alive, but I am so pissed. And not hopeful that something will come soon enough. If by some miracle it does, I am certain that it will not be powerful enough to save my brother, who also has FA, but is more affected. Too many decades of hoping, and disappointments, and too much loss. FA is too complex. When the body attack itself [like in cancer] science has been rather ineffective.


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