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.

Wowza.

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.

The State of Our Union

Patients, Friends and Family, Doctors, members of the CF community, and my fellow humans:

During my term as author of this blog, I have been honored by the chance not only to share my story with you, but to share yours with the world. The voice of all of us as a single, powerful whole has always been, in my mind, the driving power behind these posts. And so, when I came to you in 2008 to write about my conflicting emotions over being evaluated for lung transplant, when I came back in 2009 to post about my joy and sorrow over being listed and my fear that I might not make it, when I came in 2010 to post about the agony of waiting, and when I came in 2011 to wonder at my life with new lungs, I did it all with the knowledge that I was sharing scenes of a battle that was never only mine to fight. I did it with the knowledge that there would always be someone here who would understand, and that I, in turn, would always strive to be there for others. I did it because I knew -- have known all along -- that this war might be a long one, with lots of rocky battlefields along its way, but that together, we would always claim victory.

So when I wrote about those moments, whether happy or sad, I wrote the words with hope. And when I wrote about the Cystic Fibrosis Foundation and the amazing drugs in our pipeline, I wrote those words with conviction. Because when I wrote about the need for US -- for you and for me and for everyone who can -- to take the lead in making our disease, our communities, and our world into something we can live with, I did so with utter and unwavering faith.

And now, it is my true honor and privilege to stand (sit) in front of you (or in front of your screen, anyway) and send out this message with every last bit of the hope, conviction, and faith that I would like to think has characterized this blog from the very beginning.

Ladies and Gentlemen: THE STATE OF OUR UNION IS STRONG.

The state of our union is strong because even as more of our friends and loved ones continue to lose their lives to this disease every.single.day, we continue to fight, and we know that every angel only makes us that much stronger against this monster.

The state of our union is strong because we are leading the way in drug development for all genetic illnesses -- not just ours, but all of them. We are solving problems and searching for answers that others have deemed too challenging, and we are succeeding.

The state of our union is strong because we don't take no for an answer. Ever. And we never will.

The state of our union is strong because we come together in tragedy and in triumph. We know that neither one ever comes in isolation and we have learned that anything worth doing is going to come with pain. We don't need every second of every minute of every hour of our lives to go exactly as planned; we know that life is worth living just by virtue of being life itself.

The state of our union is strong because we celebrate it.

The state of our union is strong because we never forget who we're doing this for.

The state of our union is strong because we are seeing results. We have a new drug in front of the FDA right now that will probably change the face of this disease for ever. We've done that. But we're not stopping there, and we have new drugs already in testing, new partnership agreements already signed, and new dollars pledged for even better results in the future.

The state of our union is strong because unlike some people and some officials sitting on a certain hill in a certain district-that-shall-not-be-named, we are working for everyone. When we say "no CFer left behind" we mean it. And our officials in the DC/Bethesda area are actually doing what they promise. Yep, the state of our union is pretty unique in that way.

The state of our union is strong because the state of our union is up to us.

And we choose strength.
We choose to fight.
We choose to lead.
We choose not to take no for an answer
We choose to come together.
We choose celebrate and never forget.
We choose to work because we know we matter.
We choose us, and our choice is a cure.

If I could leave you with one final thought on this blog -- if I thought, as we CFers know all too well -- that I might not be here tomorrow and that this was the last sentence I would ever type, it would be this:

That you are all magical, and that I cannot envision what my life (short as it might have been) would have looked like without your help and support. That you have already made a difference. Yes, you. So never doubt that you can do it guys, because I have seen it too many times to stop believing now.

Look, if CF or politics or life itself have taught us anything, it's probably that things can change, and often do, on a dime. So why not put that dime to work for real change and reinvest it into the change you want it to be? And if not that, then why not use your other talents instead, because things can also change in heartbeat, in the blink of an eye, or even, sometimes, in a single breath.

And so it is with us. Each time I look at your emails, your FB posts, or your blogs, I am reminded that our union is stitched together by the common threads that bind us as CFers and as people. No one of us alone, as my dad likes to say, is strong enough to beat cystic fibrosis. No disease alone, however, is strong enough or could ever be strong enough to beat us. If we remember that simple truth, then no battles will ever defeat us, no tears will ever wash away our hope, and the state of our union will ALWAYS be strong.

With love, respect, and gratitude beyond measure,
Your fellow fighter

Prolonged, But Not Saved

I don't know if any of you out there frequent or even glance at the online news source known as The Huffington Post. It's not my sole or even favorite place to go for news, personally, but I do follow it on Twitter (you can find me over there at @piperbeatty, by the way) and I even drop by the website every so often. And last night, as I was waging yet another (unsuccessful) battle for sleep against the twin terrors of prednisone and prograf, I noticed the following article:

Girl With Disability Allegedly Denied Kidney Transplant

Okay, let me just start out by saying that I have done exactly zero outside research on this particular story. I am not, in any way, shape, or form, posting the above link as a statement of fact or as a condemnation of CHOP (the hospital referenced in the article). I don't have any rights to this story and I don't make any representations as to the article or its content. Everyone straight on those facts? Okay, cool.

All disclaimers aside, what I did do is read the article. And while I was doing so, I found the following statement, attributed to a pediatrician and geneticist, that the young subject of the article should probably

"...use a 'living designated donor' who would understand that 'Amelia's life would be prolonged, but not saved, by the transplant.'"

And that, guys, is where I had to stop reading, look around, and then read the whole piece again from the beginning. Because, quite honestly, I was baffled.

What, exactly, is the difference between "prolonging" a life and "saving" it? I mean, heck, we all die eventually, right? If I throw myself in front of a speeding bullet headed straight toward an innocent child, I haven't really saved that child in the sense that she will never die, have I? What I've really done, really and truly, is prevented the child from dying that day, that minute, that instant, and from that bullet. Assuming that my attempt is successful (I don't know that when I make the decision to leap, after all), then the child will still die somewhere down the road, though I think we can all agree that it would hopefully be a long way down the road and not just around the next bend. But even if it is sooner rather than later, my act of jumping in front of the bullet is still considered "saving her life", right? I mean, if the kid dies in 6 years, no one is going to look back at my action and shake their heads sadly, muttering "well, that's a darn shame. She only prolonged little Suzie's life, when she thought she was saving it!"

On some level, I read the doctor's attributed statement as reasonable, I guess. It seems to be saying that the donor should know in advance that the kidney transplant will not cure the child's disease or other disability -- that she will likely still suffer complications down the road and possibly reject the kidney or damage it, or maybe die of other causes. And I get that, seriously. As someone who was blessed to receive new organs, I know the sad truth that these suckers are scarce (sidenote: go here and sign up if you agree with me and want to fix that!). I understand that not everyone can receive new organs and that doctors have to make extremely hard calls about who can and cannot be placed on the transplant list. All of that makes sense to me, even if it does sometimes strike me as incredibly sad.

But as to informing donors that their gifts are "prolonging" rather than "saving" lives? Well, that, to me, is where things get murky. I'm 100% for information and informed consent, but I also don't know how or why that information is really valid. What sounds more reasonable (and maybe this is more along the lines of what the doctor had in mind in the first place) is to tell donors and recipients alike that transplant is not always (or maybe ever) a total cure. Transplant comes with risks, it comes with side effects, and sometimes it comes with unexpected results. What it never comes with, unfortunately, are guarantees. And as someone with a disease that was not cured by my transplant (I still have CF in my other organs and in my sinuses), I also take it as somewhat of a given that transplant does not have to be perfect to still be a wonderful, joyous, and very much worth it miracle. I have no doubt whatsoever that the doctor quoted in this article would agree with me on that as well.

What scares me is the idea that potential donors out there might think their gift is any less valuable if it simply "prolongs" a life rather than "saves" it. That scares me because I have spoken to transplant patients who have told me that "even just having one deep breath with these new lungs would have been a miracle big enough to make everything worth it." It scares me because I know myself, as a patient, how valuable an organ is to anyone who is lucky enough to receive it, and also to their family and friends. It scares me because the real value of a life is not measured by the number of days it continues, but by the passion and the strength and the goodness with which it unfolds, even if that unfolding ends far too soon. It scares me because some of my favorite stories are short ones, and I want to make sure that those stories are cherished just as much as the longest novel.

Most of all, though, it scares me because it ignores the fact that all donors, all gifts, and all recipients are, at the end of the day, equal. The beauty of organ donation for me lies in the personal connection it represents: one soul reaching across the void or the fear to offer another soul a second chance, because we are, after all, made up of the same stuff. And the best thing about second chances is that they are always a testament to faith and spirit -- even if they don't always last forever.

So probably it is true, after all, that most transplants are more of a "life prolonging" surgery than a "life saving" one. But maybe the deeper truth is that if we could all be so lucky as to prolong the life of another, then we just might end up saving our own.

Little "Miss" Compromise

A lot has happened since I last updated this blog, so please bear with me if this post seems a bit all over the map. It's a common problem in my world, though I can honestly say that I wouldn't have it any other way.

The most exciting news is that a dear friend from my hospital's transplant program (and a fellow CFer) got his brand new lungs just about 48 hours after I wrote the last post about our little "CF/transplant group." For all the dry runs and the drama and the frustration of my time on the list, I truly can't imagine what this guy went through in waiting for over 2 years for lungs, all while attempting to keep up with the first one and then two handsome sons he shares with his gorgeous and incredibly loving wife. They are truly inspiring people and wonderful parents, and I am so excited for them to move forward together as a perfect, happy, and HEALTHY family. To the donor and the family who made this all possible, wherever you are today, thank you from the bottom of our hearts from all of us who know and adore these special people!

As for me, I get to take care of several routine and not-so-routine health matters before the month is out, including transplant clinic, some less-than-awesome digestive "fun", the endocrinologist to follow up on my pathetic Vit. D levels, a bravo test for my reflux, and the dermatologist. For sanity's sake, we'll keep the discussion of most of those to a minimum and just focus on the circus du jour, otherwise known as a complete dermatology check up.

For those of you who have never had the pleasure of this experience, you're missing out. The process basically involves stripping down and getting a complete body once over by someone trained to know the difference between a freckle and a serious problem. In my case, since I'm covered in freckles pretty much from head to toe, this typically feels like a some sort of military hazing activity. Luckily the visit today was less painful (literally) thanks to the presence of hand held mirrors. Points to the doc for thinking up that one.

What was fun, though, was the final "consultation" part of the program, during which I was informed by the staff that people on immunosuppressive drugs are 65 to 250% more likely to develop skin cancer than your average John or Jane Doe. Where exactly I fall within that large range was never actually explained, but since I have pretty fair Irish skin I'm guessing it's in the upper numbers. Skin cancer, I was told, is dangerous and possibly deadly. The take home message was definitely that the sun is a monster of which I should be afraid, be very, very afraid.

Now, I am cautious about the sun by pretty much any standard. I've known about the increased skin cancer rates since before I had my transplant, and I've also known that certain of my other drugs (like antibiotics, for example), make me more sensitive than most people to the sun and more susceptible to sunburn. Also, as someone suffering from chronic and incurable whiteness, I learned at a very young age that I go straight from pale to burned. There is no tan in my world, there is just increased freckling that sometimes blends together in some form of brownness -- and that only seconds before I turn roughly the shade of a lobster in a boiling pot. I was about 5 years old when I discovered SPF 45, and I can honestly say that I've never looked back.

But that, according to the helpful folks at the dermatology department, isn't quite good enough. In fact, their list of suggested precautions went something like this:

-NEVER EVER EVER leave the house (regardless of weather) without wearing SPF 30 or higher on all exposed skin patches. EVER.
-In fact, since sun and UV rays can come through windows, always wear sunscreen indoors as well.
-Not that it matters anyway, though, because no part of your skin should ever be exposed to the sun. Wear hats with sun protection and SPF clothing at all times.
-SPF 15 is useless. We laugh in the face of SPF 15. It is basically grease. It probably attracts the sun. The makers of SPF 15 are probably paid by the cancer lobby.
-The safest place to live is in a dark hole underground. Second place goes to houses and apartments with no windows, provided you never leave. Anything less than that and you're screwed.

Basically the advice could be summed up as: go outside as little as possible and when you do wear long sleeves with sun protection built in and SPF 30-45 on any exposed parts. And stay away from windows."

Here's the thing though: I'm not afraid. I know the sun can hurt me and I know I'm not invincible (nothing like CF and a lung transplant to teach you that lesson!) and I know I need to be careful. I know all that. But I also know that a lifetime of defying expectations and refusing to listen to life expectancies or warnings not to ride my horse or play too hard or travel too much or whatever have NEVER ONCE persuaded me that the right way to live is to value caution over life. Not then, not now, and hopefully not ever.

So here's my compromise:
-I do wear sunscreen, and I do wear SPF 30 or higher.
-I do NOT wear clothes with SPF, not because I'm stupid, but because fashion is actually important to me. My sister designs gorgeous clothes and I want to wear them.
-I do NOT always cover up every part of me. I do, however, take basic precautions like wearing sun hats.
-I do try to avoid being outside in the sun for extended periods of time, if I can do so without missing out on an activity that is important to me.
-I do not currently wear sunscreen in the winter, but I will going forward. That seems reasonable. Also, I will remember to wear it even if I'm not planning on being outside, particularly in summer.

Okay, so it's not perfect, but I think it strikes a nice balance between being careful and allowing me to be myself, which includes the part of me who grew up in the mountains and loves the outdoors. And who knows? It might even help a little with my low vitamin D levels, which are of course related to sun intake.

The reason I chose to write about this though is not to get approval of my personal compromise plan. In fact, quite the opposite -- I'm quite positive that by posting this I'm leaving myself vulnerable to actual criticism from some of my doctors and the medical staff at my hospital, at least a few of whom know about this blog and occasionally read it. Trust me when I say I'm not expecting any kudos here, nor do I expect everyone will agree with me. Instead, I chose to write this because I think it's important that we, as patients, know and understand our own power to take well-meaning medical advice, think about it logically, and make our own decisions about how to integrate it (or not) into our own lives. I would actually go so far as to say that I think this is the number one job we have as patients: not to follow instructions blindly, but to have an active sense of our own health needs and emotional priorities and to spend at least some time deciding for ourselves how best to balance the two. Anything less is, to borrow from earlier in this post, missing out.

Look, I know my lungs are a gift, and the last thing I want to do is jeopardize that gift by being stupid in the sun. I have friends who didn't make it to transplant, and others who died after their bodies rejected their lungs. To do anything less than the best I could by this new set is in some ways a slap in the face to all those who die each year without the chance at new life, or to my donor who gave me my life back. I know this, and believe me: it is not a responsibility that I take lightly.

At the same time, though, I know my lungs are my chance to live my life in ways I haven't been able to for a long time. For me this means outdoor activities. It means not allowing my health to dictate everything from my clothing choices to the amount of time I spend at the window. It means never taking a single breath for granted and knowing that sometimes I will have to suck it up and do things I don't want to do for the sake of my body, but not that I need to stop living. To put it another way, wearing SPF clothing might not seem like much of a compromise to make. Nor, for that matter, is giving up eating raw food or alcohol or going outside without a mask or riding the subway or swimming in indoor pools or taking all the drugs or not allowing my dog to sleep with me or not taking Communion at church or not shaking hands or avoiding crowds or taking the blood thinner shots that give me nasty bruises or never touching a public computer or not eating from a communal chip bowl at parties or never sharing food or...well, you get the idea. Every single one of these things, taken individually, is actually a reasonable suggestion. Put them all together, though, and it's easy to see how these rules can start to take over your life. And since a lot of them CAN'T be compromised AT ALL (taking all the drugs, for example) then it stands to reason that where one might negotiate leeway is in some of the others (I shake hands with strangers all the time, and then I use hand sanitizer). I personally don't think this is a stupid risk on my part, but I guess that's open to interpretation.

So now I want to hear from a couple of you, if possible. Email me or comment below or whatever, but share a little about how you deal with the "life"/health dichotomy. When, if ever, do you make compromises? Why or why not? And, hugely, what do you do to ensure that you don't "miss out" on any of the things that are important priorities for YOU?

Love, light, and best wishes for a happy and healthy 2012, beautiful people.