Ork… wait, what?

Let’s say that coffee is your thing. Maybe it’s tea for you. Perhaps a hot chocolate or an actual piece of chocolate. Wine might be your treat of choice, paired with soft cheese and quince paste. Donuts, muffins, caramel slice, strawberries, pistachios, kale? Cronuts? Take a moment to think about what food or beverage makes your heart sing, rights your world, and helps your life feel that little bit easier. Can you picture it? Can you taste it? Can you feel it coursing through your veins and giving you an extra frisson of awesome?

Now, take that feeling, distill it into a molecular formula, package it into a pink pill, slap on a humongous price tag, and call it Orkambi.

Ork… wait, what?

Orkambi. This is the slightly bizarre trade name of the Kalydeco and Lumacaftor drug combination (which I have previously referred to as K+).

I am not a fan of the name.

I am not a fan of the price tag.

I am a fan of the drug.

It’s not a cure for CF. Nowhere close. But it is ground breaking. It’s the first drug combination to treat the underlying cause of CF in people who have two copies of the F508del mutation. Every other treatment we have available targets symptoms. Treating the cause was entirely unprecedented before Vertex created Kalydeco, and now, Orkambi.

If you follow CF-themed groups on social media you will have noticed that Orkambi has just been approved by the Food and Drug Administration (FDA) in the USA. This means it is safe and effective. This is big news. Royal-wedding-sized big news. But with fewer hats. And more medication. People in the USA are already getting their hands on this drug combination and waiting for their lives to change. Meanwhile, the rest of the world is waiting for their own national bodies to approve Orkambi, and in some cases, to fund it. Those of us not in the USA are effectively boiling the kettle to make a cup of tea so we can have a bit of a sit with our feet up while the powers that be sort out the logistics.

Dave and I are two of the one thousand or so people who have actually tried this medication. We have been enrolled in the clinical trial for two years so far and we know for sure that we have been receiving active drug for eighteen months. We are uniquely placed to give others insight into what life is like with Orkambi zooming around the bloodstream.

First day of the trial! I couldn't keep the smile off my face. Even though I spent a mind numbing seven hours at the hospital.

First day of the trial! I couldn’t keep the smile off my face. Even though I spent a mind numbing seven hours at the hospital.

A quick preface before I spill the beans. Dave and I are clinically very different CF patients. He is fifty and I am thirty-two which means he has had eighteen more years to decline. He was also born in what I affectionately describe as the dark ages when CF care was rudimentary at best. As a consequence he did not receive the benefits of knowledge, medication and treatment that were available by the time I graced the world with my presence. CF has also manifested itself differently in us both.

This is a bona fide free coffee from Vertex at a conference I went to. Vertex are the makers of Kalydeco and Orkambi. I drank much coffee on their tab.

This is a bona fide free coffee from Vertex at a conference I went to. Vertex are the makers of Kalydeco and Orkambi. I drank much coffee on their tab.

Janette
Since taking known active ingredient… I have been feeling great! Two weeks after my first dose my lung function increased by ten percent and I have stabilised at this level. I have historically bounced up and down when it comes to lung function but now it has plateaued at an extremely pleasing level and is steady as a rock. My exercise tolerance increased. My quality of life increased. My exacerbations decreased and I lasted three whole years between hospital admissions which, I am happy to boast, is my longest stint IV-free since high school. I am still taking all of my meds and doing all of my treatment but I can afford to be a tad more flexible without it having a negative impact upon my health. Life is simply a little bit easier. I feel as though my CF handicap has lessened. I have much more confidence in my lungs these days. I have much more hope for my future. For me, Orkambi is like my first cup of coffee in the morning. I could get out of bed and participate in my daily grind without coffee. I could. I really could. But let’s face it. I don’t want to. Coffee peps me up. It greases the rails of my life. It doesn’t stop my day being difficult but it sets it up superbly so I can hit the ground running. Likewise, Orkambi gives my body the best chance possible.

Dave
Since taking known active ingredient… he has declined. Not exactly what we were hoping for. Throughout the trial we have had to invest more resources into maintaining his health. Despite this, his experience has been disappointing with a lower quality of life and an increased number of exacerbations. He will finish the trial and will hopefully continue taking Orkambi on an expanded access program until it is approved and funded in Australia (should that option become available). We would rather he continue to have access to the drug just in case, but that’s really the only reason why he’s continuing.

In a nutshell, we have had two very different experiences. Which is exactly why clinical trials enrol more than two people.

This is a last special note to all Australians. It took years for Kalydeco to be approved and accessible. There is concern that Orkambi will be similarly delayed. I encourage you all to the join the WeNeedOrkambi Facebook page so you can stay in the loop regarding important developments in our wait for Orkambi approval. There may well be opportunities for those who are particularly sympathetic to help lobby for faster access and equality in healthcare for people with CF living in Australia.

If you have begun, or hope to begin, taking Orkamabi I encourage you to be cautiously hopeful. My fingers and toes are crossed that your life becomes more manageable and that the hope Orkambi inspires for a more secure future comes to fruition.

***

For those with CF who are extra curious here is a little more detail. We did not put on any weight. It has not improved our gastrointestinal symptoms – we both still need to take as many enzymes as before and I managed to get a bowel obstruction recently which was supremely un-fun. Our liver enzymes did not increase. We did not experience a “purge” that others have experienced. In fact neither of us ever had a moment when we knew for sure it was working as the effects seemed gradual. My first dose of certified active dose did make me feel a little breathless and weird but bronchodilators sorted me out and I have not experienced that again.  We have had few adverse events, if any, and it’s hard to know if they are actually side effects of the trial or unrelated. If anyone requires more detail I am happy to provide it! Please contact me: capturingcf@gmail.com.

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4 thoughts on “Ork… wait, what?

  1. Pingback: O Day | capturing cf

  2. We are starting the screening process on Monday 14th December to see if our 10 yr old is eligible for the trial for 6-11yr olds.
    We pray that she will get on and hopefully she will have a outcome like yours. Usually we are in almost every 8 weeks now. Sometimes we get longer…….8 weeks and 3 days between the last 2 admissions. Is it ok that i am excited over a extra 3 days haha…..

    Like

  3. Pingback: Canapés: unexpected challenges for a person with CF | capturing cf

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