Can One Email Save Two Lives??

It has come to my attention that two girls in the cystic fibrosis community are in desperate need of our help. In this case, “help" can come in the form of an email from YOU and others in the CF community.

Here's what I know:
A couple of years ago Arkansas State Medicaid denied coverage of Kalydeco for four girls in need of the medication. Through the hard work and dedication of Beth Sufian (lawyer with CF herself) and her law partner, two of the girls were able to receive Kalydeco recently. Unfortunately, that still leaves two of the girls in Arkansas who are in desperate need of the medication. Their health is in decline, and time is limited. Action needs to be taken now!

The doctors of both girls have petitioned Vertex pharmaceuticals for a compassionate use of Kalydeco. The response to these pleas has been disappointing. Vertex’s response to the pleas was that they would meet with Arkansas State Medicaid. A meeting is good, but the medication would be better. A meeting will not stop the rapid decline in the health of these two girls. 

Vertex has the ability, under the law, to provide the girls with Kalydeco as early as tomorrow! The FDA allows for on-label compassionate use of any approved drug of which both of these girls qualify.

One can only imagine what these girls and their families must be thinking. The same family who participated in fundraising efforts that provided the money to accelerate the research for this drug, is now being denied access to it. Does that sound at all fair to you?

What YOU can do:
You can copy and paste the following text into the body of an email and to send it Charles Johnson, VP of Global Medical Affairs at Vertex. His email address is charles_johnson@vrtx.com. Let him know that this is unacceptable. Feel free to use any or all of the email provided or write your own. Please copy ronniesharpe@cox.net and info@cff.org to any email sent. 

***

Mr. Johnson - 

It saddens me to hear that Vertex is refusing to provide Kalydeco to the two girls in Arkansas; despite having the ability to provide it under compassionate use. As a community we have worked very faithfully for many years to raise funds for this very drug; for you. I understand you can’t help everyone who is denied coverage, but it would be nice to see you try. The CF community stands up and helps one another. We work tirelessly to raise these funds with the expectation they will be used to improve the health and lives of those living with cystic fibrosis. My hope is that you will serve this community like we serve each other. Thank you in advance for doing what is always right, not what is always profitable.

Sincerely, 

Your Name

***

Are you tapped into social media? You can also help on Facebook and Twitter!

Facebook:

Vertex’s 2014 second quarter financial results stated Vertex had $1.22 Billion dollars in cash.  The only drug Vertex sells is #Kalydeco. Yet they refuse to provide the drug to two girls in Arkansas who have been denied access through Medicaid, even though they can, according to law. http://ow.ly/A5NCi

#Fighttheinjustice #supplydontdeny

Two girls in Arkansas whose health is declining are being denied coverage by Medicaid for #Kalydeco, a drug which could help them drastically. Legally, #Vertex can provide compassionate use of #Kalydeco while their legal case continues. Yet they are doing nothing to help. Help these girls. http://ow.ly/A5NCi

#Fighttheinjustice #supplydontdeny

It’s important for a company to do the right thing. #Vertex is not in the case of two girls denied coverage for #Kalydeco by Medicaid. Show your support for fellow Cysters and their families. http://ow.ly/A5NCi

#Fighttheinjustice #supplydontdeny

Doing what’s right is always more important than doing what’s profitable. http://ow.ly/A5NCi #Fighttheinjustice #supplydontdeny

Twitter:

@vertexpharma Doing what’s right is always more important than doing what’s profitable. http://ow.ly/A5NCi #Fighttheinjustice #supplydontdeny #Kalydeco

@vertexpharma How can a company with $1.22 billion dollars in cash not provide the compassionate use of #Kalydeco? #cysticfibrosis http://ow.ly/A5NCi

@vertexpharma The Arkansas cysters need #Kalydeco now, not a meeting! http://ow.ly/A5NCi #supplydontdeny #cysticfibrosis

I can't thank you enough for doing this and taking time out of your busy day to help these girls.

You can read more about the girl's case here: http://online.wsj.com/articles/costly-drug-vertex-is-denied-and-medicaid-patients-sue-1405564205

My Thoughts Kalydeco

Ran onto this old post I wrote about my thoughts on Kalydeco. Updated it with some current information. Hope you enjoy.

I am sure you are aware of this.  What does the CF population in general think about this?  Does it help you?

The CF community is in one of two camps concerning this: uber excited or feeling that its bitter-sweet.

This particular drug will only work for those in the community that have the G551D mutation. It's estimated that in America, that number stands at somewhere between 2000 - 3000. So, obviously those patients and their families are over the moon right now. There are also many (me included) that are super excited for them and know that this is a potential step forward for the CF community at large.

Then there is a portion of the community that feel happy for "them" but sad for themselves or their kids. Most of this camp has the most common mutation, DF508 (I have two copies). So far, a drug for our specific mutation hasn't yielded great results. We're all hopeful, but I'm not holding my breath for anything ground-breaking for at least 5-10 years.

(Update: The results weren't a slam dunk, but they weren't terrible either. I may have to go with 3-7 years instead :) )

So in short, it was an amazing day for many of my friends and a huge step forward for all in the CF community. I'm pumped over here!!

I guess my view is that they figured out a delivery system for genetic modification, right?  That seems HUGE to me.  This drug, when taken, will correct the genetic mutation in certain cells, yes?

Are there any other drugs that actually correct gene mutations on the market, CF or otherwise?  Just seems like a huge thing to me.

It's big, but we're still a ways away.

The real issue with CF, and ultimately the effectiveness of these drugs, is that there are over 1600 mutations affecting the cell and CFTR in different ways (thankfully not over 1600 different ways, however). This new drug works for those with G551D, because it actually gets the "door" (CFTR) on their cell wall to open up and exchange sodium, potassium, water, etc to a level within "normal range". The key here is that their CFTR is actually on the cell surface to begin with.

The challenge for those of us with the DF508 mutation, is that our CFTR is "stuck" on the nucleus of our cell. They are currently trying to get the CFTR away from the nucleus and to it's proper place on the cell wall. If they can do that, they will in theory be able give this new drug to us as sort of a one-two punch.

I'm not sure whether or not there are other drugs on the market that are similar. This drug doesn't correct the mutation, it's correcting the result of the mutation.

Looks like there are between 2000-3000 WORLDWIDE that can benefit from this drug, not just America.

They have said lately that it will eventually be able to benefit others outside of the G551D mutation, but even then, the majority of people with CF will not see the benefit.

So in short, we're closer than we were yesterday, but not as far as we'd like to be tomorrow.

Kalydeco: First Week In

Guest post by Angie

WARNING: To those of you that are not my fellow cysters and fibros or caretakers of aforementioned cysters and fibros, this post contains discussion about mucus… wait, if that would bother you why would you be reading a blog about CF? Never mind.

I took my first dose of Kalydeco on a Tuesday night.  I took it and went to bed.  It’s hard to know if Kalydeco was the cause or not, but I had a hacky cough that is usually brought on by drainage in my throat all night.  When I got up in the morning my throat felt really dry and scratchy.  The hacky cough continued throughout the day.  The next day (Thursday) during my morning treatment I noticed that the mucus I was coughing out was thinner and lighter in color.  Could this drug already be making a difference or was it just wishful thinking? In the short time I had to research the effects of Kalydeco, I don’t think I took any action to manage my expectations.  I think I was torn between expecting to see a difference right away and thinking that would not be possible it would be more of a marathon than a sprint.  After all, it’s taken 36 years for my lungs to get to their current state, so they aren’t going to improve overnight, right? 

On Saturday I was contemplating how my first few days on Kalydeco had gone.  I realized that I was coughing a lot less than I normally do.  This was a bit concerning because my family and friends have always been able to locate me by the sound of my cough.  If my cough had decreased, how would anyone find me when we were out shopping?  This is a problem I am more than glad to have!! I also noticed I had more energy.  Don’t get me wrong, I still need my weekend naps, but I think that is just because of my love for napping.

 

It’s funny, but I noticed the most changes during my first week on Kalydeco.  I’ve continued to feel this way, most of the time.  My cough still increases when the weather changes.  Since I live in the Midwest, the weather is known to change anywhere from 20 to 30 degrees in a 24 hour period.  This has always made my chest feel tight and my head feel like it was going to explode.  Since being on Kalydeco, I have noticed my cough increases with these weather changes, but I’m not having sinus headaches.  I have also had more big, old looking globs come out of my sinuses and lungs.  I take that as a good sign that some of the old stuff is breaking up.  The true test for me will be when I have my first PFT since starting Kalydeco.  I have a feeling we will be celebrating once again when that day comes!

Next Update: This truly is a wonder drug: I’ve started running (although I use the term “running” loosely)!

Note from Ronnie: If you missed Angie's first blog about discovering that she did in fact carry the G551D mutation after many years of thinking she didn't, click here (you can also find her bio). She also gives an update about her emotions after/during receiving her first bottle of "the hope diamond" here. 

Kalydeco: The Hope Diamond??

Guest post by Angie

Who knew such fanfare could surround such a tiny bottle of pills?  I certainly didn’t, but I do now! After I received the news that I had one G551D mutation, my clinic sent my information along with a prescription into Vertex (the company that makes Kalydeco) so they could begin the process of making sure my insurance would cover the drug.  My clinic told me that it would probably take about two weeks to get Kalydeco because of the authorization process with my insurance and the pharmacy processing time.  I had already waited 36 years, what was another 2 weeks?  Also, this wonderful news just happened to hit the day before we were leaving on a week’s vacation… so let the party begin!

About a week after my clinic sent my information to Vertex (and towards the end of vacation), I received a call from Vertex letting me know my insurance would be covering Kalydeco.  They also told me that my copay would be $250 for a one month supply.  Not really a surprise as I already pay this for a couple of my other drugs.  However, the case manager also told me that with their copay assistance program my copay would only be $15 a month!!  Now, I know everyone was suffering from a little sticker shock after Kalydeco received FDA approval and we heard how much the drug was actually going to cost.  Vertex immediately stated that no one would go without the drug.  It seems they are living up to their end of the bargain.

Once my insurance had given the thumbs up, Vertex sent the script to the pharmacy I had chosen.  There are only a handful of pharmacies that can distribute Kalydeco.  I chose to receive mine from the CF Services pharmacy.  At the beginning of the next week (after arriving home from a fabulous vacation!), I received a call from CFS to verify my shipping address and telling me that I had to sign for the drug when it arrived.  I confirmed the delivery address and was told I would receive another call just to verify the address one more time.  Were they shipping me the Hope diamond?!?!  I received the second verification call and was told my prescription would be shipped that day via overnight delivery so I would have it in my hot little hands the next day!!

I am not an overly emotional person by any means.  Some might say I’m not even a mildly emotional person.  But, the weeks that led up to Kalydeco arriving at my door were very emotional.  It was one of the most wonderful feelings in the world.  The day that I started Kalydeco was also quite a celebration.  We toasted the arrival with some tasty champagne and my husband prepared a wonderful dinner.  I decided that I would take my doses at 9am and 9pm since you are supposed to take it every twelve hours.  You also have to take Kalydeco with fatty food.  That is not a problem for me!! My inaugural dose was taken with double stuff oreos and milk.

 

Coming next week: My first week on Kalydeco!!

Note from Ronnie: If you missed Angie's first blog about discovering that she did in fact carry the G551D mutation after many years of thinking she didn't, click here. The story is quite amazing!!

The Start of My Kalydeco Journey

Guest post by Angie 

On January 31, 2012 the CF community received amazing news.  The FDA approved Kalydeco, the first drug to treat the root cause of CF.  However, Kalydeco is only effective for individuals that have the G551D mutation.  I, like many other DDF508’s, celebrated the wonderful news but was still holding out for the drug that would help the mutations that I had.  That’s right… I said had. 

About a month ago I took my 2 year old son to see a pediatric pulmonologist, because just like his Daddy, he has asthma.  The doctor and I discussed the fact that I had CF and my son’s newborn screen did not come back with any genetic markers for the disease.  He thought this was very strange knowing that my son would automatically be a carrier since I have the disease.  The doctor decided to add a CF mutation panel to the labs he was already ordering for my son. 

Fast forward to one week later and I received an email notification that my son’s test results were available.  I logged on to his EHR (gotta love technology) and saw the CF mutation panel.  I clicked on the result fully expecting to see DF508 since I had been genotyped as a DDF508.  Instead, I got the shock of my life when his test showed G551D.  What?!?!  The only logical conclusion I could come to was that I must have a G551D mutation.  But, that was impossible, right?  I had a cheek swab in the late nineties during an initiative sponsored by the CFF to get everyone’s genotype into the CF portal.  It was after 8pm on a Thursday night, so I couldn’t call any of our physicians.  So, where did I turn?  The CysticLife community.  I posted a rambling question asking if there could be any other logical explanation.  Of course I received lots of wonderful feedback from the CL community.  Everyone drew the same conclusion that I had.

I contacted my clinic the next day and they ordered a CF mutation panel for me.  I had my blood drawn and the waiting began.  I was still trying not to get my hopes up, but I think it was too late.  I received the phone call on a Thursday morning.  I had just sat down at my desk at work and my cell rang.  I recognized the number of my CF clinic.  I answered and the nurse said “I have some wonderful news.”  That was all I needed to hear.  I started blubbering.  I have the G551D mutation!!! I could not believe it.  I cannot even describe what it felt like to hear that news.  I can tell you it ranked right up there with the first moment I looked into the eyes of both my babies.  This news actually allowed me to picture holding my grandbabies.  I started the ball rolling that day to have Kalydeco delivered to my door and begin my new lease on life! 

Thank you for taking the time to read my story.  I can hardly believe it when I read it myself, but I am living it every day and it is wonderful!  I will be posting weekly updates about my progress with Kalydeco.  Please feel free to ask any questions or leave comments through this blog.  I look forward to sharing my journey with you!  

Angie's BIO:
I am a 36 year old married, working mother of two beautiful children. I was diagnosed with CF when I was 7 months old. When I was 6 years old my mom decided to become a respiratory therapist. I grew up with my own private RT!!  I was first hospitalized at age 8 and so began the annual hospitalizations. In my twenties I sometimes averaged two hospitalizations a year, but its pretty much just once a year now. I went through the usual rebellion from my late teens to early twenties. Luckily, I have been able to maintain stable lung functions for over 10 years. My FVC averages around 80-90% pred, and my FEV1 is usually around 63% pred.  

Note from Ronnie: I am so thrilled that Angie has agreed to give weekly updates of her progress on Kalydeco!! Her story is amazing, and I know I for one, am really looking forward to hearing about where this story leads. I can only hope that it's not only to improved lung function, but LIFE function as well!!

Kalydeco - Can it benefit others??

A couple weeks back, I was able to listen to Dr. Beall (President of the CFF) present about all of the progress that has been made over the years with specific focus on the new drug, Kalydeco. As most of us know, Kalydeco was approved a few months back for those among us who are rocking the G551D mutation (about 4% of the population). He's a good speaker and I can see how he has spear-headed the fundraising machine known as the CFF. He's passionate, charismatic, engaging and of course, informative.

He gave a very good illustration of what Kalydeco does for the peeps it helps, and why it doesn't work for others. Basically, he had all of us in the room (the cell) pretend we were sodium and chloride and the door to the room represented the protein which opens and closes to allow a natural exchange of different properties from within the cell to the lining of the airways. Those with the G551D mutation have a door that is stuck shut and Kalydeco has shown that it is able to open that door.

I have heard all of this before, but what I haven't heard was what came out of his mouth next. To paraphrase, he said that although Kalydeco is only for about 4% of the population now, they hope that it eventually it will be effective on between 16 and 20 percent of the population. So how could this be? Well that's because G551D is a gating mutation, or "stuck door" mutation, and there are other gating mutations out there. If a CFer's protein is on the correct place on the cell surface and the only issue that the door is stuck, then Kalydeco may work for them. (For those who are wondering - DF508 not only causes a stuck door, but the door isn't even located where it should be, on the surface of the cell). They are currently running further tests to see just how many others may benefit from this new drug.

Which leads me to my last and final point, Dr. Beall wasn't just blowing smoke. In fact, I exchanged an email with a momma in the community whose daughter is currently taking Kalydeco and does not have the G551D mutation. So is it helping her? It seems so! Her FEV1 is up 11% which is her highest in three years, and her sweat test came back almost 30 points lower. I don't know about you, but that excites me to no end. Not only of course for this young gal (who happens to be one of my favorites), but also for others who may benefit from Kalydeco.

I know it's not available to everyone who may benefit, and that sucks, but just know that they are working their butt of to see who will be able to benefit from the drug. In the mean time, do everything you can do each and every day to put yourself in the best position for the day that your "wonder drug" comes out.