Thursday, July 2, 2015

On Vertex and Victories

I woke up this morning just like most days. Bleary-eyed and exhausted after a fun night of fighting transplant insomnia, I half walked, half stumbled my way into the kitchen to grab a cup of water and a few crackers before making my way toward my medical-supply shelf. Blood sugar safely checked and vital signs good, I reached for a handful of pills, gulped them down, and began the morning ritual of planning my next high-calorie breakfast. And, in the meantime, I reached over next to me and picked up my phone.

And just like that, my day got better. 

There's nothing quite like waking up to a slew of texts that the newest CF drug -- the next source of hope, another chance at health, at growing older, at LIFE -- has just been approved by the FDA. Even if the drug can't help you specifically (this current drug is not yet being marketed towards those with my specific mutation combination, nor has it been tested on folks who are post-transplant), just knowing that there's a new option out there for people like me, people I know, and people I call my community is enough to bring tears of joy and gratitude into my eyes. Because I know that somewhere out there in the world, at least some of the 70,000 other beautiful people who "breathe like me" are waking up to the news that their world may be about to change. That they, too, may soon be offered the chance to reap the benefits of this newest little miracle. That, if nothing else, they have a chance now -- a chance to see if this could be their time, their magic, and THEIR moment. These are my brothers and sisters in arms, after all, and who am I not to delight in their success?

But then again, that's not entirely true. I have my selfish motivations as well.

You see, today, July 2nd, 2015, is a HUGE day for the CF community as a whole. This is not because Orkambi itself is a "cure" (it isn't), or because we've finally achieved the goal of having a corrector option for ALL people with CF (we haven't, and we will not rest until we do), and it's not even because this could mean longer, healthier, and fuller lives for the people who CAN benefit from this new drug (okay, that one is totally true). Today is amazing simply because it represents yet another victory of our incredible community over this deadly little monster called cystic fibrosis. This approval, in other words, represents the strength we have in numbers (however small we might seem) and the breathtaking tenacity, courage, and commitment of our team against what sometimes can feel like an unstoppable enemy. It represents US, beautiful people. Because believe it or not, WE made this happen.

Without participants for the study, this could never have happened.

Without fundraisers, walkers, climbers, bikers, and hikers for CF, this could never have happened.

Without the tireless efforts of our determined advocates, who lobbied so hard for access to clinical studies for those with orphan diseases and for evaluation standards by the FDA, this could never have happened. 

Without our community's willingness to stand alongside the Cystic Fibrosis Foundation as it took a HUGE risk in developing new and innovative approaches to rare-disease research (approaches now being reproduced by others for their own success), this could never have happened.

Without our incredible showing at the FDA committee meeting in May, where our patient representatives spoke bravely about the impact Orkambi has had on their lives and a professional blogger for the investment news group TheStreet exclaimed that he had never before had so many actual community members tune in to his live blog of the meeting, this might never have happened. 

Without our refusal to take no for an answer, this could never have happened.

But it did happened. It's happened twice now. And that means, quite simply, that it can happen again. And again. And as many times as it takes, until WE make cystic fibrosis a thing of the past. 

Happy Independence Day, beautiful people. You certainly earned it.


  1. As usual, beautifully stated. This community has accomplished amazing things and uses its past performance as motivation and drive of future performance. I'll keep betting on (investing in) US.

  2. As usual, beautifully stated. This community has accomplished amazing things and uses its past performance as motivation and drive of future performance. I'll keep betting on (investing in) US.

  3. Piper, you bring joy to life. Happy Breathing Day to you, Independence Day, and Loving Life Day!

  4. We share your optimism, Piper! Beautifully said!

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