Modulating Reality: My Miracle CF Med Was All Hype

Modulating Reality: My Miracle CF Med Was All Hype

I’ve spent most of my life hearing that the cure for cystic fibrosis is just around the corner. Just wait a few more years, they keep saying, and it’s going to be right there. Then a few years passed, and a few more, and the cure seemed just as far away as ever. My first real hope came with the release of the CF modulators — in my case, I qualified for Orkambi (lumacaftor/ivacaftor).

The internet was abuzz. Everyone was saying Orkambi was the first real step toward the cure, a miracle drug that would alleviate CF symptoms to the point they almost weren’t noticeable. I was excited, constantly asking my doctor when I could start. Then I held those pink pills in my hand and they felt weighty, significant. This would be my first step toward becoming better.

The side effects were immediate and brutal. The worst was chest tightness, so bad that I would spend hours awake at night, unable to get comfortable because it felt as though I couldn’t breathe. It would be worth it, I thought, once the Orkambi started to work. But there’s the problem — it never did start to work, at least not in the way I felt I had been promised.

My sweat test levels remained exactly the same. My lung function percentage didn’t change at all, except for a swing upward of a few percentage points when I finally got active. This didn’t change with the Symdeko (tezacaftor/ivacaftor) either: I no longer had horrific side effects, but I didn’t see any obvious benefit minus a mild decrease in lung infections. This was the reality for me — the miracle drugs were anything but.

Putting in more research, I learned this has been the sad reality for many people. New medications are constantly hyped up to be the next-level gamechanger when in reality they’re simply a steppingstone to something bigger. I felt so isolated by success stories, as if even in my cystic fibrosis I was wrong. Through putting out feelers, I learned I wasn’t alone.

There’s a strange feeling when what works so well for others doesn’t work for you. It doesn’t diminish the medications’ overall value, but it can feel isolating. We should be able to be honest with each other; not every discovery is as life-changing for every person as it’s hyped up to be, and that’s OK. Perhaps it’s time we held more realistic expectations for medications and a nuanced discussion of disappointments and setbacks in the medication world.

Having a medication do nothing is bad enough. We shouldn’t set ourselves up for further disappointment.

But what about you? What are your expectations of the modulators, and what was your reality?

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Note: Cystic Fibrosis News Today 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 other 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 Cystic Fibrosis News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to cystic fibrosis.

8 comments

  1. Jane says:

    Hi Elizabeth,
    Living with cystic fibrosis is not fun. We have a one year old that will be starting kalydeco soon. I too live with hope that there soon will be a cure for all cf’ers out there. I wish you the best. I believe that gene therapy, correctors and modulators are improving our future. Thank you for your post and sharing your thoughts.

    • Emily says:

      Hi Rachel,

      Kalydeco was a complete game-changer for me. How exciting that young patients like your son can take it. I started it in early 2013 at the age of 30. It has made the disease more manageable. I definately hoped it would just make it go away, but that didn’t happen. Adding Kalydeco made the lung treatments much more effective, which has given me hope and renewed motivation to continue all the work that is required to manage cystic fibrosis.

  2. Michael says:

    The Symdeko is the same thing as Orkambi plus another drug. If you were having side effects with the Orkambi you would have them with the Symdeko. No one said that anyone would be symptom free. The medication should ease the symptoms and prolong the usefulness of your lungs.It takes time for this and patients aren’t all seeing the results right away, but they will.

  3. Emily says:

    Hi Elizabeth,
    I only ever see the press releases announcing these new treatments, and, although dissappointing, I want the whole truth. I think you are right that these treatments are stepping stones to better ones in the future. CF can be such a relentless slog. I don’t ever know how to accurately explain this to those who are not patients or caregivers. Thank you for sharing your story. It really got me thinking about the promises before Kalyedeco came out and then, eventhough overall successful, all the small dissappointments that came after. It was life-changing, but not the cure I hoped it would be.

  4. Dorothy Vallillee says:

    I have been praying for a cure for CF for nearly 51 years. My son who is alive and is now 51 was diagnosed with CF at age 7 months. It’s been a long journey but thanks to Kalydeco he is healthier now than he has been for a number of years. We are so thankful and still working to find a cure.

  5. James Albright says:

    Hi. I am in the exact same spot. Same issues with Orkambi, little sense that Symdeko is making that much of a difference. I do work to remember that because I take so many medicines it is difficult to know exactly what the impact of any specific medication is. It could be that Symdeko is slowing my inevitable decay, but that is not so much for a $330K medicine! (55-y-o)

  6. Patricia says:

    Daar Elisabeth , we have the same here with out 10 year old, only his sweat test did improve but he had severe side effects and we stopped after 9 months. Hope for better meds in the future

  7. Brian Eddy says:

    I don’t understand the purpose of this article. There should never have been an expectation that the drug would work the same way for everybody, that was a given. No treatment for CF has ever worked to the same degree for everybody. There apparently were benefits – slightly increased lung function and fewer lung infections. For a CF patient, simply slowing the progression is a great thing. And even more confusing, “a steppingstone to something bigger”? That is simply the natural evolution of science/medicine, treatments continue to improve. So I find this article very confusing.

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