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Saturday, April 14, 2012

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 


  1. Woah, impressive results! I do have G551D and have been on Kalydeco for 1.5 weeks. Thus far, I definitely haven't hit the "feeling awesome" part of the drug. I'm still coughing up a lot of crud and feeling generally not so great. The huge thing I have noticed, though, is that coughing it up has become WAY easier!

  2. A couple questions out of curiosity~ were your starting fev1 and the one reflecting the 11% increase taken on the same meter? Because that is truly impressive and I am so happy for you. I am also wondering if you had a sweat test done prior to starting Kalydeco and if you'll have a repeat test done~ because even if treating your CFRD muddies the waters when it comes to further improvements in your pulmonary health, it certainly wouldn't affect changes in your CFTR function. I am really curious about this aspect of it.

    I notice you say you saw these improvements without making any changes to your diet, exercise or meds and I am SURE that helps. Working with the mechanism of action of the med and helping to clear out whatever it's breaking up just makes more sense than dropping stuff like I see a lot of other references to.

    I hope you continue to do well and insurance keeps covering it.

  3. Hi Harriett. I did PFTs on Monday, April 9 at my clinic and then came home to compare to my home meter - the FEV1s were 0.03 apart. So although you are correct, 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. But you certainly make a very valid point and I will continue to update my FEV1.

    I did not do a sweat test prior - unfortunately I felt lucky to just get an Rx and my doc wasn't too keen on doing one. I didn't want to push hard for it because I had already pushed quite a bit just to get Kaly. You are 100% correct about changes in CFTR function - fortunately others are doing this during their off label Kaly trials so we will see what they come up with.

    I definitely appreciate what you are you saying about treatments - for me, they are essential.

    Anyway, I am excited for my results but I definitely understand they were anything but scientific. I did the best I could, but there is no question that not using the same meter as you correctly pointed out and not doing a pre and post sweat test makes my findings even more subjective. Luckily for me, I get to make my own decisions about Kaly. Ha :)So I only have to prove it works to myself.

    I hope others keep sharing their experiences. Thanks again for your questions and I will continue to post!

  4. As a lefty socialist from the UK I am going to resist the urge to respond in a Michael Moore style and just hope that everyone wjho can benefit has access to Kalydeco. (Kalydeco is currently under review by the NHS while we wait for the European license to come through next month) ;-)

    Anyway, thanks for the update.

  5. Your meter seems to be quite good (which one is it, btw? I'm looking for one...) A difference of only .03L in readings, especially for adult capacity lungs- is pretty insignificant in my opinion. If you ever did pursue getting a sweat test in future, it would be interesting to compare against ANY past ones you've had, since it's highly unlikely your sweat chloride level has changed all that much since you had it done way back when.

    Wishing the very best. I know how hard you work to maintain your health and I hope this continues to be, at the very least, another tool to help enable everything else you are doing to work better. :)

  6. @Woody, great news! all patients with G551D that make under $150k in the US can get access to Kaly per assistance from the manufacturer and the CF Foundation.

    So not only will everyone have access to the drug, but - those who don't have G551D have access where we are seeing clinical benefit.

    I'm willing to bet that those without G551D will have access to the drug in the UK.

  7. CHOP in Philadelphia is now doing experiments with Kaly in other non-gating mutations. ;)

  8. Thanks for the update! Sounds like Kalydeco is making a difference for you and that's great. We all know that FEV1 can flucuate and it is just one part of the big picture. If you are feeling better, that's all that matters. :) Wishing you all the best!

  9. Any chance you could tell us your mutations? I am trying to get off-label and know a few people with my son's mutation that have benefited from Kaly.

  10. My original post about Kaly has my mutations

  11. I don't know much about Di507 but I'm looking for evidence of Kalydeco helping a splice mutation in order to write an appeal for insurance's disapproval of Kalydeco for my son's DF508 and 621+1G>T. Did you write any type of an appeal at any point? Do you have any thoughts on where to find evidence it may work for us?

  12. Hi there,
    So how DID you get hold of enough Kalydeco to do this experiment on yourself? My two daughters have a gating mutation and I have been trying to get them into a trial....but there are no participating cf centrum in the Netherlands... Did you pay it yourself?
    Cheers, chantal

  13. @ Chantal, yes my insurance paid for Kalydeco 100%. Many people bash the US healthcare system of insurance, but this is a great example of it working well. I did not pay for it myself - my insurance covered it 100%