Kalydeco!

June 1st 2015 was a very special day for us! Finally Kalydeco was approved by the Dutch government for insurance coverage and we received our first box.

LEARNING HOW TO SWALLOW PILLS
Two weeks before, we heard June 1st would be the grand date and we quickly had to start teaching J how to swallow pills. We watched a Youtube video on how to best swallow pills and started practicing. We started with cake decorations (tiny pink sugar balls left over from decorating my daughter's birthday cake). He soon graduated to TicTac's. It was hard for me to stay relaxed, but the last thing I wanted him to feel was pressure. He'd only start gagging and wouldn't be able to get anything down. So I looked everywhere but at him, while he was trying and the whole family cheered when he swallowed a TicTac. We were all practicing with him, noticing how we swallowed our supplementts and giving J tips ('my tongue moves like a 'coindozer' shoving the pill to the back of my throat, you could try it too'). From there we started practicing with Skittles. Swallow one and you can eat one (we hardly ever eat candy, so it was a big treat for him!). Not sure of the actual size of a Kalydeco pill, I went to the supermarket and looked for larger sized 'practice pills' in the candy isle. Some bags weren't see-through, and it must have looked awkward, but I had to squeeze, feel and measure the size of the candy. By the end of the two weeks he easily swallowed whatever we gave him, he would have tried swallowing a bowling ball, just to get us to cheer for him.

PRE-KALYDECO TESTS
During those 2 weeks we scheduled appointments for a pre-Kalydeco sweat test, an MRI, a blood test and a fecal elastase test.

The week of the sweat test J was taking 3 Curcumin (1800mg) and 2 Genistein (100mg) and the sweat test came back at 70.

We now have this list of sweat tests:
no CurcGen: 118 mmol/L
2 Curc 1 Gen: 90 mmol/L
3 Curc 2 Gen: 70mmol/L
6 Curc 3 Gen: 59 mmol/L

The MRI of the lungs came back 100% clear!!! No signs of CF whatsoever! That made me very proud! This had been our goal all along. Keep him healthy, until there is medication for him and we did it! We've moved heaven and earth, and it was an extreme amount work, but we did it!

The blood work was all good and the elastase test (pancreatic function) showed typical CF very low elastase in the stool. We tested this, because I'm hoping it'll go up after a year or so on Kalydeco, like has happened with the 2-5 year olds with a G551D mutation.

STARTING KALYDECO
Since end of 2012 J had an irritated cough. A dry loud cough a few times a day. It took a while to understand what was going on, but we traced it back to 'something must be hiding in the sinuses', we even figured out which side. How? Because the water dripping out of the nose after a nasal rinse always came out of the the left nostril. We tried some alternative treatments. An ionic silver spray for the nose (which reduced the number of coughs clearly) an oregano oil lozenge (which turned his throat from red and irritated back to pink and calm), but the cough never disappeared completely. Our hospital (as it turned out, to my utter frustration) only tests throat swabs for Pseudomonas (and nothing else). I asked them if they could test for other bacteria, because clearly something was hiding up in that sinus, but if you don't know what is up there, and what it's sensitive to, how can you decide how to treat it? It turned out to be Staph. I asked for a treatment for Staph in the sinus, and not oral antibiotics because it'll just burden the body and mess up intestinal health and the sinus cavities don't seem to be reached very well by systemic antibiotics. But there doesn't seem to be a topical treatment available. How strange! It would be very nice if there was an inhaled antibiotic that would treat Staph or a nasal spray? (anyone reading this that knows of a treatment, please leave a comment!). Then I read about someone that had tried a drop of Tea Tree oil (essential oil) added to a sinus rinse. We started that just around the time that we started Kalydeco.

We started Kalydeco on June 1st. I expected a 'clean up': at least a few days of wet coughs and a dripping nose. Well... all we heard was one very tiny wet cough on day 2 and that was it. The dry irritated cough took 4 days to disappear completely.
I also expected his bowel movements to get firmer and was on stand-by with stool softeners. But... nothing happened. Bowel movements stayed exactly the same. They have been perfect for a few years now, but still I expected some kind of change, but it didn't happen.

After 3 weeks a big blob (?) of clear mucus came out of the right nostril after a nasal rinse. I was surprised it was clear. I had expected something green or yellow because of the Staph infection, but it was perfectly clear. We still don't know if the Tea Tree oil helped at all or if it was Kalydeco, but he's been clear of the dry cough since June...yay!

REPEAT SWEAT TEST
July 2nd was judgement day. The repeat sweat test. We called the hospital a few days later to hear the results, but they werent in yet.... bummer. Another few days later, we called again... still the results weren't in. Then our doctor called the lab to ask what was going on. The lab technician said: "Well, I was analysing the sweat test and chloride came back so low that I checked the patient's earlier sweat tests and figured this was impossible, so I stopped the analysis". We laughed really hard, but now we were REALLY curious what the number would be... A few days later the doctor called. The sweat test result came back at <12!!!!
Unbelievable!!!
No more salty kisses! I've gotten so used to tasting my lips after a kiss on his forehead and it still surprises me that he doesn't taste salty anymore.

REPEAT LUNG FUNCTION TEST
Even though his lungs were perfectly clear on the MRI, surprisingly J's lung function jumped up by 10%. Since there is no scar tissue, no inflammation, no mucus in the lungs, no obstructions, the only logical explanation I can think of is that the extra chloride has lead to increased elasticity of the lungs.

REPEAT BLOODWORK
The bloodwork after a month on Kalydeco showed slightly elevated ALT levels. I've heard this from a lot of other Kalydeco users and with all of them, levels decreases after a few months.
When J was diagnosed, however, his liver enzymes were also raised and he took Urso for about 2 years. During those years we started CurcGen and since Curcumin is known to support liver health, after a while we thought: "let's see what the liver does without Urso". His liver enzymes stayed well within normal range without Urso, but while on CurcGen. So what happened? Is it because he stopped taking CurcGen or is it because we started Kalydeco? I don't know. We'll be repeating bloodwork for liver enzymes soon, so we'll know more. Still on Kalydeco only.

CURCUMIN GENISTEIN
We stopped CurcGen a few days before starting Kalydeco to see what the liver en sweat chloride would do on Kalydeco only. We'll start again soon.
Because, after reading other people's stories about Kalydeco a thought occurred to me, which I think is very important for future development of CF potentiators and correctors and may also explain why so many patients with non-gating mutations also seem to benefit from CurcGen (especially at low doses).