Orkambi Video Diaries – Day 11

A day late posting this! Here’s my video update from yesterday, day 11 on Orkambi. Yesterday I was feeling the best I’ve felt since starting Orkambi. Today, though I’m a little less bright-eyed and bushy-tailed, I’m still feeling much better than last week and gearing up for week 3 on Orkambi. Stay tuned…



On Orkambi and Hope

Girl With a Balloon by Banksy

Girl With a Balloon by Banksy

When you live with illness your entire life, you get used to certain realities. With Cystic Fibrosis, the reality is this disease kills you. Thanks to research and medications funded by the Cystic Fibrosis Foundation, the life expectancy has risen substantially in my lifetime, but its still hovers at about 40 years old. Another reality is we don’t often go gently into that good night. Whether it’s respiratory failure, transplant complications, heart failure, or liver failure, end stages of Cystic Fibrosis are hardly forgiving.

Yesterday, my reality maybe changed. At the very least, it got put on hold. Around lunch time, my speciality pharmacy called to say my health insurance approved a prior authorization for the newly FDA-approved CF drug, Orkambi. In other words, my insurance agreed to foot the whopping $300,000 annual bill for the prescription. I nearly fainted at the news.

If you’re not part of the CF community, maybe you haven’t heard of Orkambi. I’m here to tell you, it’s a big deal. When I was born in 1988, they didn’t even know what or where the CF gene was. Now, 27 years later, Orkambi is the second drug to get at the underlying cause of CF. The first drug to treat the underlying cause was Kalydeco. While a breakthrough in CF research, Kalydeco was only successful for a rare mutation affecting about 4% of CF patients. Though Orkambi is not an all-encompassing drug (it does not work for all CF mutations), it does target the mutation found in almost half the CF population. Rather than just treating symptoms of the disease, Orkambi helps the defective CFTR protein function normally for those with two copies of the F508del mutation.

During drug trials, researchers noted a 40% decrease in lung exacerbations. It’s those tricky lung exacerbations that lead to inflammation and infections, decline in lung function, damage to the lungs, and ultimately, death for Cystic Fibrosis patients. A 40% decrease is life changing.

I’ll be starting Okrambi in the next two weeks and as I type this I can’t believe it’s real.

Orkambi is not a cure, regular daily treatments and clinic checkups are still required, and as mentioned, it is not effective for all mutations of CF. But cure or not, it feels like I’m getting a new lease on life. Having kids and actually sticking around to raise them feels less like a daydream. Growing older doesn’t sound as scary because maybe I won’t get sicker with each passing year. Maybe I’ll still be here when they cure this thing.

My life leading up to Orkambi has hardly been all doom and gloom. I have an incredible support base of family and friends. A beautiful and loving boyfriend and an even more beautiful and loving golden retriever. I’ve traveled, been on a few adventures. And on the health front, I’m doing well. My lung functions are up and I can run a full three miles without wheezing. All in all, I really, really like my life. But it’s hard to shake a weight as heavy as chronic illness. Like many people with CF, I struggle with anxiety and at times, depression. Some of my anxiety probably stems from my fear of infection, from my fear of getting worse. Every flu season I hold my breath and hope I can evade it. Every flight I flinch at any passenger sniffle. I worry so much about getting sick, sometimes I actually make myself sick with all the worrying.

With Orkambi and its possible 40% decrease in lung exacerbations, for the first time in a long time, I feel light, unburdened. I feel hopeful.

I’m not the only one feeling hopeful. Yesterday, when I called my parents with my Orkambi news I could practically hear their hearts bursting over the phone – and from 2000 miles away. The fight’s not over. CF is still scary, it’s still a killer, and I still have it. But I think my family is due at least one long sigh of relief.

Before I start planning my bright(er) future, there might be some side effects. Maybe even brutal ones. Some CFers call the first week on Orkambi “hell week” or “the purge.” Excessive cough accompanied by boatloads of mucus, bloody sputum, and at times, vomit, are all common experiences in the first weeks on this pill. And while I’m not particularly looking forward to any of that, I can’t shake this feeling of lightness.

Plus, I’ve been feeling crappy for going on 28 years – what’s a few weeks?


On Cystic Fibrosis, Loneliness, and Writing It Out


image by Rikki-Tikki-Tavi

I go back and forth  a lot when it comes to writing about Cystic Fibrosis (CF) – writing about my Cystic Fibrosis. Some days it feels so coded not just literally in my DNA but in my life experience as a whole that I couldn’t possibly share my thoughts with any one person, let alone an audience of anonymous internet readers. Other days I worry that writing about CF would put me in a box – forever stamped a sick girl who blogs about mucus. And no one wants to read about mucus. But it’s on days like today, as I sit down on my porch with a cup of tea and my MacBook, when I think, well, shit – I guess I’d probably read it.

The truth is, having a chronic disease is lonely. And when you’re living with a rare chronic disease with a high risk of cross-infection making it difficult to meet the few others like you face-to-face, the alienation can be tough to shake. Though I have an amazing support network of friends and family who constantly motivate me to stay healthy and happy, at the end of the day, none of them have CF. And when CF is freaking them out, they get to talk to other people in their exact same situation – other parents, other significant others, other siblings, other friends. They can meet for coffee and hold hands and say “man, this sucks.”

For the most part, my only interaction with someone like me is when I stumble across the occasional local newspaper story about a high school senior raising money for the CF walk. Or worse – the obituary of someone who’s lost their battle. And while any exposure to this orphan disease is more than welcome, Cystic Fibrosis isn’t all just fundraising and dying – at least that’s what I’m hoping.

“Solitude is fine but you need someone to tell that solitude is fine.”
Honoré de Balzac

There are parts of this illness that I don’t want to think about, let alone scare my friends and family by saying them out loud. Maybe this stems from being a middle child – I always want everyone to be OK. But I think it also relates to the media’s portrayal of illness and our culture’s expectations and discomfort surrounding “the sick.” We want to be inspired by their bravery, by their indomitable spirit, by their thirst for the life despite the tragic brevity of their own. It seems the worst thing a sick person can be is angry or depressed.

So, here’s where I come back to the idea of writing about CF. But in the end this won’t be a “CF blog.” A little because of the difficulty and discomfort mentioned above, but mostly because I’ve got more on my mind than just my illness. Remember, there’s a whole lot that goes on between the fundraising and the dying parts. When I’m not thinking about CF, I’m thinking about a million other things I want to write about, things I want to do, places I want to see, snacks I want to eat after work. But I will include CF from time to time in these posts because I can’t shake the feeling that I’m not the only one disconnected by illness. Maybe someone else is perusing the internet like I have countless times, wondering how others with CF are getting by. Wondering if they’re happy, if they’re healthy, if they’re funny, if they’re angry, if they’re scared.

And if they stumble across any of my forthcoming blog posts, they’ll see at least for me, it’s often a combination of all the above.