Tuesday, January 31, 2012

For The Rest of Us

I'm sure by now most of you have heard the news.

For those who don't yet know, however, today was a watershed day for the CF community. For a long time now, CF research has been aimed at what Dr. Beall, the President and CEO of The Cystic Fibrosis Foundation, likes to describe as "long shots on goal." Now I'm no sports junkie by any stretch, but apparently this means taking a chance and focusing your energy on getting things done, even if it requires a little faith and courage to get you there. And today, with a little assist from the FDA (like that, guys?), it seems the CFF and the entire CF/medical/research world scored a huge goal, touchdown, home run, or whatever the heck you call it when you strike a MAJOR point against a seemingly undefeatable opponent. Because as of today, January 31st, 2012, the FDA in the United States has officially approved the use of Kalydeco (aka VX-770) for the treatment of cystic fibrosis in patients with the G551D mutation.


If you don't know what Kalydeco is, let me explain briefly. This is a drug -- the first of its kind -- that actually targets and corrects the underlying defect that causes CF. Simply put from a non-scientist, it opens up a chloride channel that is typically blocked in CF patients with a specific mutation. Granted, not all CFers have this mutation. In fact, it represents only about 4% of our population. Which means, given that there are about 70,000 cases of CF worldwide, that this drug ONLY offers the opportunity to SAVE THE LIVES of about 2,800 people. It may work on others as well, but as of now it will ONLY allow 2,800 beautiful souls to grow up, live life, marry a loved one, start a family, have a career. It will ONLY save about 2,800 families and communities worldwide the pain of losing a child or a young adult. And this approval ONLY means that about 2,800 pairs of eyes, and the eyes of all their families, friends, and loved ones wept with joy this morning when the FDA made its announcement. Only that.

As for me, I don't have the G551D mutation. I know people who do, wonderful people whose lives have in some cases already been completely changed by this new development, but I also know a lot of people, like myself, who don't. It remains to be seen whether this drug alone or in combination with other medications might work even part of the miracle it promises for that 4% on other members of the CF community. Right now that question, like so many others in CF, remains unanswered. And while this drug represents the raw hope and truth that the CFF has now PROVEN that this disease can and WILL be conquered someday, it is fair to say that this morning's sweet announcement also carried with it the bitter reality that some of us, without question, will still die waiting for our chance.

So what does this mean for the rest of us, then? Because honestly folks, I've been there. When I was first referred for pre-transplant testing in 2008, this new class of drugs was really just a glimmer on the CF horizon. I remember so clearly the thoughts I had back then: that maybe if I could move somewhere with one of the study trials my lung function would miraculously make the leap and allow me to participate; that maybe if I could just hold out one more year we'd make it to a miracle and I could wiggle my way in on compassionate care or extended access; that maybe if we could just speed it up, even by a month or two, this would all be MY chance and I could stop watching from the sidelines and start living this wonderful new reality that seemed so darn close I could have reached out and touched it if I'd only been given the opportunity.

Yeah, I remember that.

Obviously, you guys know the way that story played out. I got tested, was eventually listed, and became one of the lucky ones fortunate enough to walk the precipice all the way to a double-lung transplant, at which point my life was saved by a completely different type of miracle. I was, and am, beyond blessed in this reality, but I have to admit that it didn't always feel that way to me in the moment. The simple truth is that it's hard when breakthroughs that represent so much hope and that deserve such intense celebration don't quite mean for you what they might mean for others. It's hard, as much as it is wonderful, to watch as other people receive a "cure" for your disease -- just as I can only imagine how hard it is for the parents or spouses of my friends who didn't make it to transplant to read this blog. In some ways, the bitter truth is that nothing hurts more than the miracle that comes too late.

I can honestly say for myself, though, that the only way out from the sidelines was to throw myself back into the game, and this time to try and be a stronger player than ever. What I realized through my transplant is that there are a multitude of ways to save a life, and it is always a team effort. There's nothing quite like being on a waiting list for lungs with some of your favorite people in the world, or knowing that in order for you to live another beautiful soul will have to leave this world, to make you think twice about what it means to take care of each other -- to have an assist when deep down you're also waiting for your own "long shot" at the goal, at glory, or even at life. And oddly enough, I know now that had I gotten my wish and had that chance at a miracle shot, I would most likely never have been healthy enough to fight as hard as I did for the goal we just made.

Which is why, when I heard about Kalydeco's approval, I cried too. I cried for my friends who have this mutation and what it might mean for their lives and their health. I cried for the many, many workers and volunteers and researchers whose efforts have made this possible. I cried, of course, for the families who will benefit, and especially for the younger generation who might never need to experience any of the flat-out bullshit that comes along with this disease. I cried for my fellow lung transplantees and the lungs or the lives that might have been saved. And I cried for all the amazing CFers and families out there who assisted in this shot, who are heroes -- every one of them -- in their own right, and who are still waiting for the moment when they, too, will get the chance to score. I cried, as it were, for all of my teammates, past and present and future, and for the knowledge that this goal represents a huge score (and a promise of victory) for each and every one of us.

Congratulations, teammates.


  1. Thank you for sharing. It is hard as a momma of a CF kiddo whose cannot benefit from this drug to wait. Especially when I feel his life slipping away and I am starting to hope. I am praying it comes fast enough for him, but in reality I know it may not. I love your sports analogy as I am a coach. It is kind of like having to play your best friends in a championship game and you don't win. You are happy for them, but you wish it was you.

  2. Another mom to a 2 year old With DDF508. Every time my facebook feed lit up with the declaration of Kalydeco Day, I cired, for all of the reasons you've listed- but I couldn't have said it so well. Thank you for having the words.

  3. Piper, now you have me crying too! Crying for the those with the G551D mutation, crying for those who couldn't be helped in time, crying for those of us who might still be able to receive that cure, and yeah, crying tears of guilt because I haven't had to fight that fight for decades (because of a late diagnosis) yet might, just might, still have a selfish glimmer of hope for myself.

    But yeah. It is a whopper of a milestone, not only for the CF community, but for the medical research community as a whole. So many precedents have been set, from the focused efforts of the CFF, to the amazing fundraising, to the clinical testing, even changing laws so patients on aid programs could participate, to the milestone of the first drug with FDA approval for a group of patients with a specific genetic makeup. Wow. Amazing.

    With emotional thanks to all who helped make this happen. ~Juliet Page

  4. I like a lot of cfers have been crying happy tears for those that this breakthrough will help! I am so happy. I know this will never help me not because I have Df508 but because the damage has already been done to my lungs. Now I wait for another bittersweet miracle of a donor for second chance at life. Although this new break through will never help me I take pride in knowing that my life with CF has been one that has been studied and in a round about way because of us older CF soldiers, have in return help the little cf soldiers born today!! With my eyes full of tears I want to congratulate the CF community!!! I almost feel as if I should say Happy Birthday :)

  5. Thanks for your post. I'm not a mother of a CFer, but a grammy, who loves that little girl with all my being. I cried too when I read that news...praying that some combinations or new development will help Ella, who is also a DDF508.

  6. Thanks Piper for putting it so well. For those that can benefit - this is a medical miracle of epic proportion. It will change the course of their lives in ways we can never fathom. For those like my son, it is the opening of door, eyes, minds, and hearts to so many like him and you that still wait for the day that CF can stand for cure found too. Never let the hope diminish/leave because miracles and joy are all around us - each and every day!! God bless you Piper for all you are and the inspiration just living your life provides to others. I will never forget your courageous letter to "CF" you read in Richmond at the patient ed day. If you get a chance - can you send that to me, I would love to read it to my son.

  7. Beautifully said! Kalydeco won't benefit me personally, either, but I rejoice at any signs of progress against this disease!

  8. So eloquent, as always. As someone who has watched this entire drama (tragedy? comedy? farce?) of a disease for my entire life, from the sidelines instead of as an actor, I can completely understand. Understand both the joy and the disappointment. However, you're absolutely right--the joy in this news isn't necessarily the number of patients that the drug will directly affect. It's in a successful fight and a new discovery that might spread to the rest of our patients. Thanks, Piper, for talking about the elephant in the corner.

  9. The drug is an amazingly promising thing. Please let me appologize in advance for my frustrations. There is alot to be thankful for with this amazing discovery. Everything Except for the greedy deep pockets, and I am sorry to not revel in the splendor of it all. I cry because I cannot afford the drugs we have now! The new drug will cost parents OVER a quarter of a million dollars for every year you want to keep their kid alive with this drug. $294,000 price tag for the privilege of breathing. Sure, that sounds fair. To Warren Buffett! Lets do the math. $75 million to develop the drug. Approx 1600 people initially will benefit. First year profits $470,000,000. Half a billion dollars in the first year alone! Exactly who is getting this fair treatment? My health insurance sure wont put up with that long before they drop or run up to our insurance limits. www.bizjournals.com/boston/blog/bottom_line/2012/01/vertexs-cf-drug-294000-may-be-fair.html?page=all

  10. Piper, it's been many months since we've seen an entry from you. Definitely worried - will we hear from you soon??

  11. I will never ignore your brave mail to "CF" you study in Richmond at the affected person ed day. If you get a opportunity - can you deliver that to me, I would really like to study it to my son.

  12. This is a drug -- the first of its kind -- that actually targets and corrects the underlying defect that causes CF.