Orkambi—the new Cystic Fibrosis drug from Vertex Pharmaceuticals—just received FDA approval. I was hoping that it would, because it’s a fine drug. That’s my review of it: it’s fine. It does some things very well—my lung function is currently hovering around 77%, which matches my adulthood high from 2009—while other things—my digestion, diabetes and sinuses—haven’t responded as positively. If you’d like, I wrote a whole bunch of words about my experience here, here and here. Most of that still applies, though I’ve since switched digestive enzymes and added a long acting insulin to my routine, both of which have been positive changes. With my new enzymes, I’m able to get away with around 240,000 units of lipase per meal, which is down from the all-time high I hit during the study—300,000, a number that’s probably unsafe–but still above my pre-study number of 200,000 units. Continue reading
Tag Archives: Kalydeco
Thoughts on Vertex, Spin Machines and Destabilization
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While you read, enjoy this picture of my lungs.
Yesterday, UNC School of Medicine published a research article in Science Translation Medicine saying that they found evidence that Vertex Pharmaceuticals experimental potentiator drug—the one that is supposed to move the defective CFTR protein—is destabilizing the CFTR protein after the corrector drug fixes it.
My Vertex Study Experience or “Going to the Bathroom for Fun and Profit”
Not nearly as cool looking as the KISS Alive II gatefold.
In my life, I’ve seen lots of advancements in CF treatment. These have all been medicines/techniques/what-have-yous don’t treat the underlying cause of the disease, but mitigate the symptoms. None of it is easy or fun, but it does provide a concrete answer to the age old question of what I would do for a Klondike bar: I would take 8 pills and 8 units of insulin.
Focus!
You can use this chart if you’d like to play along at home.
I recently participated in a study to gauge to efficacy of Kalydeco—a new drug that treats the underlying cause of CF instead of just the symptoms—in patients with two copies of the Delta F508 mutation. I kept a diary for the first month of that study and I wrote a piece about my experience, but I figured that piece would have more context when the results of the study actually come out. In the meantime, here’s a journal entry from the day I went to have my qualifying eye test. Continue reading