We had our monthly visit to the CF clinic this morning, and all looks well! We will continue to go monthly until Gavin hits six months, then it will be every two months until he is one. At that point we will have appointments every three months for the rest of his life (unless he gets sick then we will go more often).
Gavin dropped at bit in his weight/height ratio (he's in the 34th, goal is the 50th) but the doctors are not at all concerned. His height grew a lot (just under 25 inches), and his weight not so much (14 pounds) so it throws off the ratio a bit. Infants are difficult to measure for length, especially when some nurses "stretch" him out further than others when they take the measurements while he is lying down. The height numbers are much more accurate once he stands on his own. If he continues to fall in the percentiles we will get concerned, but at this point our pulminologist is really thrilled with his overall health.
The CF team noticed his dimples for the first time today. Often, Gavin is sleeping through most of the visit and today was the first time he was awake for everything...alert, talking and smiling. Dr. Laguna kept getting distracted by him while she was talking to us, he's just too dang cute! :) He was definitely flirting with all the women! Our respiratory therapist, Anne, kept commenting on how beautiful he is. By the way, Anne's family (mom) is from Aitkin, the same small town where our cabin is located...such a small world!
People have been asking me what Phase three really means for the
VX-770 drug. Sorry, sometimes I forget that not everyone deals with all this :) So here is a simplified explanation...
Phase 1: very beginning stages of trials. Studies are often done on animals (ie studying different levels of meds on mice to determine its toxicity) and on people considered to be healthy.
Phase 2: Begin testing on a small group of CF patients (all adults) doing studies that last a few weeks (
this is where many drugs FAIL and the study/drug is discontinued) This determines whether or not there are any benefits to taking this medication.
Phase 3: BIG DEAL TO GET TO THIS STAGE! The drug is studied on a much bigger group of CF patients (this next study will include kids ages 6-11) that will last months/years. At this point they are starting the process of getting the drug registered with the FDA.
Phase 4: If the drug gets to this stage, this is when Gavin will most likely be able to try/use the medication. At this point all CFers will have access to the medication and they will try it out on different mutations to see if there are benefits to those patients as well. This is when the FDA begins registering the drug for approval.
Phase 5: The drug is FDA approved! Many times this is when patients with diseases other than CF try the medication to see if there are benefits with other diseases.
Dr. Laguna (our pulmonologist) told us it really is a
big huge deal for this drug to reach phase three. This is the very first time in CF history that a medication has been able to "correct" the functions of the non-functioning cells. Gavin's cells that regulate his salt/water ratio make everything they are supposed to make, but what he needs is for those cells to actually function...at this point they don't (causing mucus to be extra thick and sticky) and this medication allows his cells to open up a (chloride) channel allowing his cells to function. Only 15% of his cells need to properly function for this medication to provide great benefits.
The best way to tell if the meds are working is to do another
sweat test. A person without CF would have a test result under 30. The sweat tests results of CF patients have shown to go from 100+ to around 35/40 from just a few WEEKS of taking the new medication, which is nearly "normal."
Again, only THREE-FOUR percent of CFers have this mutation that the medication is geared for (about 300-400 people in the US). We are so so so lucky to be in this small group of patients.
Another drug
VX-809 is in the early stages of phase 2. This drug is geared for the mutation Df508 (Gavin's other mutation) which is the most common CF mutation (about 95% of the CF population). Those with Df508 also have cells that create the necessary "stuff" for the cell to function, but unlike G551D (where what's needed is right on the edge of the cell walls) what is needed for the cell to function lies within the center of the cell. The VX-809 drug "pulls" everything to the cell wall, where all it needs is a channel (yep the same kind of channel) to open up allowing their cells to function too. So as you can probably guess, they will also take the VX-770 drug that opens that channel. Does this make sense? Sorry, its complicated for me to understand also and I am doing my best to make it easy to follow without using medical lingo!
Do we know how long it could take for this drug to (hopefully) pass through the third phase? Unfortunetaly, not at this time. But we are told that they are moving as quickly as possible...the FDA is what really slows stuff down (both a good and bad thing!)...especially since this is the first drug of its kind to show such great benefits. If this drug passes it means that many other drugs will get funding and hopefully soon all kinds of CF mutations can be treated in this manner!
The best part of this new medication is that it is taken ORALLY! Pretty much all other meds treating CF at this time are inhaled which takes up a good portion of the day. Gavin already does
one hour of therapy a day and he's not on any antibiotics at this point! So, oral drugs don't take up more than 2 seconds of a day...woohoo for this! If this all works out the way we are praying it will, Gavin will be able to live a relatively NORMAL life!
Ok, enough of all the medical talk. It really is just a small part of our life. Anyone up for another video of Gavin giggling? He is really starting to enjoy his toys (and grabs onto them now!). In this video he is laughing at a monkey that Casey got for him. It's the greatest! It plays music and in each hand of the monkey there is something for Gavin to chew/suck on. It's tail has a wire allowing it to wrap around anything...it keeps us from having to constantly retrieve it off the floor :) Sorry its not the best quality, I took it with our digital camera.
Look at me! I'm doin it all by myself!!!
He definitely gets this expression from his mom...
I might be getting kind of tired...
Nope, nevermind I am still having too much fun!
Sometimes I just kick my legs around...
Hi!
Today the weather finally cooled off. The past two days it has been well into the nineties...too hot too fast! We caved and turned on the a/c. We both worried about Gavin getting overheated which is not a good thing with his CF. His body doesn't reabsorb the salt he sweats out so it is very easy for him to get overheated and dehydrated. So we all hung out in t-shirts and shorts (Gavin usually was just in a onesie) and stuck it out til the heat wave passed. As much as I would love for a warm holiday weekend I hope that it doesn't get that warm while we are up north! But when it's warm it's easy to laze around and nap...
and is great for snuggling :)
Gavin also likes to "stand" more than he likes to sit. Once we get him in a sitting position he often pushes himself up into a standing position...pretty strong for such a little guy! His onesie is a bit big on him and hangs down which is why his little legs look so short :) But look at those chunky little things!
Seriously. He LOVES this new blanket :)
Mom & Gavin on the dock
Louie got locked in the porch, whoops!
