Monday, May 21, 2012


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.

Wednesday, May 9, 2012

Where I've been...

To all those who are following my two previous posts with great interest, I am so 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 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...

Monday, March 19, 2012

Getting a script for Kalydeco off-label

I just called Vertex to speak with them about doctor's prescribing Kalydeco off-label.
Here is what I learned~

Any doctor, any where can write a script RIGHT NOW for Kalydeco for any patient with CF regardless of mutation.  They (vertex) do not require proof of mutation...that is only important if you have G551D and vertex can help get financial assistance for the drug.  The form doc s sign is to help G551d get insurance coverage, that is all!!!!

Vertex is bound by legal obligation to only help (financially) patients who are G551D.  There is actually very little they can say to a CF patient who is not G551D because of FDA REGULATIONS because the approval of the drug only covered G551D. They cannot tell you if it might be beneficial (even if it works great in vitro on your mutation) because it is against the LAW and they will get in trouble.

However, I was informed that if your doc thinks it is an important drug to try and might be helpful, they are absoltuely allowed to prescribe it off-label.  At that point, your pharmacy will do what it can to get the drug covered by insurance.
If your clinic has a problem with that, have them call this number at vertex:

877-752-5933 Option 2

Kalydeco for Class I and Class II

Hi Guys~

I have been keeping this info to myself because I didn't want to have a lot of "pressure" trying this new drug....and I really wanted to be able to keep a level head about improvements or no improvements....because, believe me, I really want to see improvements.
I am Df508/621+1G->T  

This second one used to be called a splice site mutation, but apparently vertex decided to make it sound much more ominous and call it NO SYNTHESIS/Premature stop codon mutation 
(Class I).

In November, I was put on IVs for an exacerbation and reached a wonderful low of 37% FEV1. They kept me on IV anti-fungals through the new year when I then caught the croup and plummeted.  Normally, my fev1 has hovered in the low 50's (for many years), but these past few months I just really had a hard time.  The croup led to another incarceration with the discovery of an old friend coming back to visit....m. abscessus. lovely.  

When I left the hospital I was blowing in the 1.4 liter range on my home meter and that promptly plummeted to 1.27, 1.24, 1.29 after a week and a half or so at home.  I tell all of this just so you see my trend.  The consensus was to treat the myco (I've always self treated that with diet and silver and it never came back after culturing for 13 years....until now @6 years later).  We decided to try the kalydeco to see if it might offer any help in my immune response.

The few days beofre I started I was taking my fev1 all throughout the days and they would not budge.  I stayed in the 1.30, 1.31, 1.32 range.....beofre treatment, after treatment, etc.

I started on February 24 in the morning. 
 February 24, 2012 FEV1...1.31
March 2, 2012 FEV1..........1.48 (before morning treatment...holy!)
March 7, 2012 FEV1...........1.57 (beofre morning treatment...holy!)
March 14, 2012 FEV1..........1.55 (but my peak flow was the highest I have done...40 points higher than last week)

I will say that that first week I measured every single day and it fluctuated but never went back down below 1.31, it just slowly crept up...Of course, since I don't have a gating mutation I have pretty much accepted that I might not have all of the "other" great things...weight gain, etc.  This is all new territory.

First DAY:  My stomach started gurgling within 3 hours (my stomach is normally very quiet).  My mom heard it sitting across the room.  By late afternoon, I begin to feel moisture in my nose (exactly like I had just done a nasal rinse)  By 7 hours, I experienced a HORRIBLE stomach ache which lasted exactly 1 hour and was so painful I couldn't think. Within 1.5 hours, the stomach ache was gone and I was fine.  A few hours later at dinner I started having a wet cough, which surprised my friends and my husband.  I then proceeded to cough up 2 huge mouthfuls of crap...again, not my normal at all.

Day 2:  Felt lousy and coughed up some very old crap in my bedtime treatment session (two plugs that were crazy dark)

Day 3- Day 4: Nothing different. feeling a bit run down.

Day 5 : I woke up with hubby thinks he saw it on day 3 and 4, but 5 was definitely a turning day for me.  I notice a slight increase in smells and maybe taste.

Day 6:  1 hour after starting the drug, my right lung which is my problem lung, just started to "release"  It felt less tight and airy...

Day 8: I feel horrible.  I feel like plugs are pulling away and are somehow getting lodged..junky...awful. My doc tells me he believes abscessus is making this a little trickier.  He says to quit taking daily pfts and just do weekly and focus on moving out the plugs.  That night I coughed up three of the darkest most horrific looking plugs I have seen ever come out of me.All of the second week I felt better, but not like I was getting tons better and was quite shocked when my FEV1 was up again.  It was decided I was having a significant response from the drug by my CF team. 

Friday the 16th will be my 21st day.  I have to say I still am having random lung pains in random places and have had asthma-y symptoms, but there is no denying that my numbers are up and I feel better (but not great).  

I don't want to give anyone false hope.  Keep in mind, I stick to a very strick anti-inflammatory diet which I KNOW gives me an advantage with response to a lot of different meds.  I think I wanted to have days of bringing old plugs up, but was reminded by my doc my bronchiectasis are terrible looking, but are completely void of mucus...I have very little plugging which is a praise God for the anti-inflammatory diet.  So, who knows how many more plugs I will really move...

As far as getting the drug, we really believed we were going to have to fight to try it...we were prepared to do anything to get a one month supply.  And then, I got a call from CF Services that insurance had paid for it!!!  PRAISE the LORD!  But, they were sorry to say I had a $50. co-pay.....unbelieveable.