RALLY THE TROOPS!!!

Hi everyone~
I have some crazy news that I think we all need to be aware of as we go forward in our agreement to take the vertex drugs.  I had become increasingly concerned about CFers getting sick and experiencing more exacerbations as the vertex drugs were being discontinued after Phase II trials.  While the vertex scientists are not all doctors, some of them are and I feel  and felt like they were completely falling away from one of the principal precepts of medical ethics taught to doctors: First, Do no harm.  Yes, the drugs are life-changing in a positive way (a cure even) and, for this reason, are harmful when discontinued.
Because of this I contacted a top 25 Lobbyist in Washington to just see if there was anything he could do to help us atleast get the patients who are volunteering a pass to continue the drugs when the phase II trials end (seeing as there is no glaring safety concern).  While he had never heard of this issue with CF patients, he was very aware with all going on with Vertex and CF.  He graciously contacted the FDA commisioner and addressed our concern....
According to the FDA commisoner:  there is no FDA law keeping these patients from receiving these drugs following Phase II trials!!!!  WHAT???  VERTEX has complete control and authority to allow these patients access to these drugs.  Now, keep in mind, that is not all of us, but can we not agree that our community should stand behind these patients who are agreeing to take these drugs!?
According to the LOBBYIST, he said we must all rally together and agree to NOT SIGN up for trials, unless it is written in the contract that the trial patient is able to remain on the drugs after Phase II ends.  We have the power.  Vertex needs us just like we need them....  They can't get paid unless we agree to be a part of this... it is ok to stand up for ourselves in this way....sometimes the boat needs to be rocked.
Along those same lines, there is a bill going through Congress right now with approval (they think) by October that will allow drugs that are life changing to have a very different course of review and trial period.  It is called the Advancing Breakthrough Therapies for Patients Act.... Basically, this allows companies to expidite clinical trials so that patients can get access to drugs possibly after Phase II trials.  The company will have to conduct phase III trials even while patients have access to the drugs.  GUYS, IF THERE WAS EVER A TIME TO CALL YOUR CONGRESSMAN IT IS NOW.  The ExPert Act is also important, but the other will allow drugs to reach us much more quickly.

And, Finally....  Do you know how powerful our words are??  One of the reasons that Vertex released interim results from the 770/809 Phase II trials was due to the fact patienst were blogging and Vertex could no longer control what information was being released.  From the CFO of Vertex at the Deutesche Bank Securities Annual Healthcare Conference last week:
"The safety finding was very strong and we want to accelerate the program into a Phase III setting. For us to do that we have to talk to regulators both in the US and Europe. We have to talk to trial investigators, patients are now blogging regarding the results and there was a corporate concern here about information getting out of the company and not being in a controlled environment. That is what drove us to the announcement"
It is time to make our voices heard...  They need us as much as we need them.

Where I've been...

To all those who are following my two previous posts with great interest, I am so sorry...life has gotten a little bit complicated.

Ugh.  Where to start.  6 Weeks into my AMAZING opportunity with Kalydeco, I got a not so amazing letter from my insurance saying I would only be able to fill my Kalydeco for a few more months and then at that time there would be a "review"----in other words, "denial :)"  Wow.  So thankful for the chance, but kinda pissy the old insurance company ripped it away.

So, after I picked myself up off of the floor, I went into prevent/defense mode.  My doctor told me to stop taking it immediately :(  So sad... it is also so hard to know something is helping and have it go away.  I have been off of it now for three weeks and my PFTs have fallen back down :(  So, I was staying in the 1.55-1.58 range (on Kalydeco) and it promptly fell to 1.42 within two weeks...  I'm not testing it right now because I have NO DESIRE to get too down about it when I know there is something that can help.  Another lovely that is back is my SUPER SALTY SKIN.  Hubby pointed it out only one week off of Kalydeco...  All the moisture feeling that I had is gone...no moisture in the nose, mouth is dry again, less saliva.....:(  I have never produced a lot of mucus and my cough is not normally very productive, but I can definitely tell I am no longer on the drug because my lungs feel tighter and it is harder to get anything up.

Our plan as of right now is to get a sweat test in the next few weeks (four weeks is the magic number).  At that point, my doc and I will decide what I need to do.  He is CONVINCED that insurance is going to decline it for sure...  We are deciding between restarting it and getting as much data as we can for the next several months (sweat test, pfts etc.) or holding onto it for emergency.  Summer is normally my healthy season and he is wondering if we hold off until fall when I get an exacerbation...

I can't begin to describe how exciting, hopeful and completely defeating this whole exercise has been.  Since Kaly isn't made perfectly for my splice site mutation, it isn't my cure... just isn't.  It is, however, the closest thing to a control that my body has ever had and having it be ripped out of my life is pretty devastating....  The only thing I didn't experience was weight gain....everything else was pretty great.

I am praying that somewhere they will do a trial for those of us with these splice site mutations.  There are three of us (that I know of) who have splice site mutations and have had significant improvements with Kalydeco.... by significant I mean 10% to 15% increase in FEV1.... ugh.....why is it taking so long?????

So, no more blogging about my life with Kalydeco for atleast a few more weeks....  I will, however, blog about diet as a few people have emailed me about it...