Asking that question is one of my earliest memories. I was never under any delusion on what the outcome would be, rather it was a matter of when. Throughout my childhood the median survival age gradually lifted, always staying a step ahead of me. For most of my adult life, the median survival has been 37.
An odd little number that I never paid much attention to. Ironically enough, 37 marked for me, the beginning of swift and rapid decline with a double lung transplant the only hope left.
Fortunately, right before CF won the war it had waged on my body, a lifeline was offered which I grasped with both hands and my life was saved. Life has a different perspective post-transplant. It’s an enormous gift, one I struggle to find the right words to explain and thank you never seems adequate.
I’m always aware that a transplant is trade and not a cure. I have traded one set of health problems for another. The side effects from the anti-rejection medication are waging their own war on my body. I will always be a person with a chronic illness and that’s okay.
But here’s not what’s not okay. In the last few years there have been significant breakthroughs in the treatment of CF. First came Kalydeco, targeting a specific gene mutation with fantastic results. Kalydeco was followed by Orkambi – which targets the F508 gene. My gene. The most common mutation of CF.
Clinical trials show Orkambi can slow the progression of CF. Lung damage is lessened, both hospitalisations and the need for antibiotics are reduced. More importantly, people with CF taking Orkambi have a better quality of life.
Neither Kalydeco and Orkambi are a cure for CF. But they are a huge leap in that direction. This isn’t a band-aid treatment, these medications are the first to treat the underlying cause of CF – rather than the symptoms.
Which all sounds pretty fabulous right? It’s a game changer.
Except there’s a little hiccup. While the FDA in the United States has approved Orkambi, the Australian government has not.
Yep, you read that right. This life-changing medication still needs to be approved.
As I’m post-transplant, Orkambi isn’t an option for me – but while we wait for Orkambi to be approved, people with CF get sicker, get listed for transplant or die. I can not imagine how frustrating it must be to know there was a medication that could help prolong my life, only to be told I could not access it.
You can find out more about Orkambi and the struggle to get it listed on the PBS here.