5 weeks of Kalydeco

So I have now been on Kalydeco for 5 weeks. For those who don't know me, I do NOT have G551D nor do I have any sort of gating mutation.

That being said, as many of you know, there was a small subset of patients with DDF508 in the Phase III Kalydeco study that had a very large increase in FEV1 with the drug. Genes don't tell the whole story with this drug - as you see with many CF patients, their genes don't predict how healthy/unhealthy they are. Siblings, with the exact same CF mutations, have varying CF outcomes.

Diet, exercise, modifier genes all have an impact on how CFTR functions.

So I wanted to try Kalydeco. Keep in mind, as I started the drug, I was diagnosed with CFRD. Meaning I have had out of control blood sugars that are artificially suppressing my lung function - most likely over the past 1.5 - 2 years I think (I have been having false negative OGTT results).

Upon starting Kalydeco, my FEV1 was approximately 2.3L, which is around 81% (waaaaaaay down from my normal of mid 90s, I know. It's been a rough year for me).

2 weeks in to taking Kalydeco, using my home FEV1 monitor, my FEV1 went up to 2.56. This was prior to taking any diabetes medication or doing any other changes in my diet, exercise or medication routine.

That's an 11% increase in FEV1. And I felt it.

I could laugh without coughing my brains out for the first time in years.

(EDITED TO ADD:I did PFTs on Monday, April 9 at my clinic and then came home to compare to my home meter - the FEV1's were 0.03 apart. So although I didn't have the opportunity to take a look at my FEV1 on the same machine a few weeks ago because I didn't have a clinic appointment, I do believe my home machine correlates pretty closely to my clinic machine.)



Unfortunately I got bad allergies from really really crazy winds that were blowing around that turned me in to an inflammation nightmare. That, coupled with experimenting with my blood sugar log (I wanted to see what would happen with my sugars when I ate a "treat" aka sugary stuff, vs normal stuff vs. healthy stuff) I think really set me back. I blew about 2.32L last Tuesday (at about 4.5 weeks).

Due to diabetes, they did my first urinalysis and it came back that I was super dehydrated (they were checking for me spilling protein in my urine - big sign of uncontrolled sugars) - and of course DUH! Dehydration wasn't helping my lungs either. This was prior to starting insulin as well - no changes in medications until 4/11 where a basal insulin was added.

So bottom line is, I don't have G551D and I had no expectation of having miraculous, mind-blowing FEV1 results with Kalydeco. But I do know that one's genes aren't the only story with CFTR function, so I was bound and determined to get my hands on this drug off label.

So we shall see where my lung journey will go - I am anticipating that my lungs will feel a bit better with some  insulin on board. But any change in FEV1 from here on out can be attributed to better CFRD control, not necessarily Kalydeco. So I a grateful I had a handful of weeks to change nothing about my care other than Kalydeco to see the true changes.

I will continue to keep you all posted.

But there is no doubt in my mind that Kalydeco had some impact......

........and I'm so grateful that my country doesn't have nationalized healthcare or socialized medicine so I could get a hold of the drug off label and my government couldn't restrict me. I'm willing to bet getting a hold of Kaly in Australia, England and Canada for non-G551D patients will be nearly impossible. I already know of more than a dozen here in the States, and I'm sure there are more.

Yes, it's my blog, and I can make a political statement if I want.


"A government big enough to give you everything you want, is also big enough to take it away." 
-Thomas Jefferson, 3rd President of the United States and principle author of the Declaration of Independence