Tuesday, September 24, 2013

We'll See

Rees just drove away for a 7AM appointment at the hospital, where he will receive his first fistful of Kalydeco and VX-809. After months of preliminary screening exams and waiting on recovering stability from a tough spring, he will swallow down brilliance. Even if he receives the placebo, in six months he will get the real thing, so the march has begun. He is paid for offering his body to science (as he says), which is always attractive to an eighteen year old!

I don't even know the names of the scientists, doctors, researchers, and whatever other job titles who are involved; but I love them. I hope they are paid well (and this is why we happily support the CF Foundation and aggressively oppose socialized medicine ~ ambitious, talented people should be paid.). I hope they keep working. I hope there is a way to express my ... thanks, love, respect... all of it and more.

We've been crushed with disappointment so many times over the years that I'm OK with smiling, but never allow giddiness. It is a trepidatious hope. The first seven years or so, even though he wasn't healed, we were going to take control and have him be the healthiest CF kid in the world. I cooked right, cleaned right, exercised him right, did his lung chores right. But CF is still CF and he still needed hospitalization after hospitalization. Then I was introduced to the World Wide Web and learned that some kids had never been hospitalized at all (It truly takes its course differently in every body without prediction, even with similar mutations.). Disappointment after disappointment, CF controls. Now, aside from skeletal management (lots of butter, ice cream, and gravy; stay away from hot tubs and dripping, sneezing noses), we live. Everywhere. Because remaining in a filtered home with none of us ever leaving (and bringing home bugs) or knowing other people, even with CF, is not living.

Here's a possibility that a handful of pills can unfold the CFTR protein, and, well ...

We'll see. We'll just have to see.



  1. Very exciting that Rees is getting Kalydeco and VX-809! I hope this makes a huge difference for his health!
    I also just want to respectfully say that here in Australia we do have universal health care and doctors and researchers are most certainly paid, lots of them extremely well.
    I hope things go well for you and that the new medication does exactly what we all hope it does!

  2. I hope you come back to see this, Lauren!
    Thank you so much for taking the time to wish him well AND to tell me something about universal health care. Maybe I listen too much to the worst-case-scenarios? My fear is that, in an attempt to save money, our expensive CF drugs (that don't exactly cure it, so it's years of care) will not be covered. Do you think this happens?
    Affectionately, Allison

    1. Hi Allison, I'm glad you're not offended. We have a system here called the Pharmaceutical Benefits Scheme, which is basically a list of medicines that are subsidized by the government. Currently we are having a struggle getting Kalydeco listed on the PBS, and the decision-makers have deferred making a decision, but we have also just had an election so it's been a busy time. I am certain that it will be listed soon. Kalydeco is very expensive, which is I think the sticking point, but they are trying to negotiate the price with Vertex. Failing that, it could be eligible for the Life Saving Drugs Program, which supplies expensive life-saving drugs to the patients in need of them without being listed on the PBS. Just think of the whole system like a big insurance company with extremely low out of pocket costs to you. We have a very good healthcare system and one of the highest life expectancies in the world, which I think is due to the fact that every citizen can access complete medical treatment without large costs involved. Things like hospital stays, transplants, even giving birth are completely covered by taxes. I hope my explanation makes sense!