It is hard to believe that so much activity and information can possibly be packed into a single day...This morning Brock and I kicked off the NACFC with a breakfast meeting with Dr. Beall. I get incredibly nervous when I know I will be meeting with Bob, but after that first hug and bout of tears, it only takes minutes before his demeanor puts me at ease. We took the opportunity to share all the details of our Kalydeco experience with him and gave him the book we made filled with the "true Kalydeco stories" from other patients. Hearing those success stories is extremely rewarding for Bob and if you contributed to the book, Thank you. He truly loved it. I feel like a big hog, because all the "thanks" has been given to me, even though all I did was assemble the stories. When I get back home, I will post some pics of the final product. It was such a privilege to put together so many beautiful stories.
* The most exciting and fantastic news that I have to report from today is the positive buzz regarding the VX-809/VX-770 combo! Tweaking dosages of both compounds has enabled them to reach the statistically significant improvements they were looking for. They were able to rescue approximately 30% CFTR function, which is sort of the magic number to see big clinical improvements ((Kalydeco rescues approximately 50%, for perspective). HELL YA!! I'm super encouraged that this combination will be progressing to phase 3 trials, and could be coming along sooner rather than later. Even if it isn't the "magic bullet" that Kalydeco is for gating mutations, this combo could go a long way toward improving/stabilizing lung function and increasing quality of life. Isn't that what it is all about?
* The recently listed trial on clinicaltrials.gov taking place in Denver for heterozygotes IS the individualized "trial of 1" or "trial in a box" design that Peter Mueller of Vertex proposed earlier this year! http://iom.edu/~/media/Files/Activity%20Files/Research/GenomicBasedResearch/2012-MAR-21/3%20-%20Peter%20Mueller.pdf It is a small initial trial, but this is really exciting news for heterozygotes, patients with rare CF mutations, or anyone who wants to see individualized genetic modifier treatments reach patients FASTER. This will enable patients with any suspected surface CFTR activity to trial Kalydeco and decide whether the therapy should be continued based on their own body's response. Again, HELL YA!!
* The overall feel I got today regarding Ataluren, is that this drug hasn't lived up to expectations. There was some discussion about the possibility of combining a drug to increase translational read-through of the CFTR protein (like Ataluren), with a potentiator like Kalydeco to boost results... But, it was mentioned several times that they already have discovered compounds that work BETTER than Ataluren ever did at improving protein read-through. The biggest problem with Ataluren is that it binds to the same site on the cell's ribosome as some inhaled antibiotics like Tobi and other aminoglycosides. Basically, Tobi and Ataluren need to work on exactly the same spot in the cell...if Tobi is there, Ataluren can't bind and do its job. Inhaling Tobi almost completely nullifies the therapeutic effect. Damn. The Ataluren presentation was very vague and I still don't have any answers about what their steps are moving forward (I even mustered the balls to stand up to the mic and ask the presenter a question about their plans for further trials or to combine with another compound and wasn't given a conclusive answer.). Again, I'm not certain about the future of Ataluren, but my overall feeling is pretty negative about this drug reaching the market to treat Class 1 mutations on its own. The primary trial endpoints were not met, and the antibiotic interaction problem may be too large to overcome. Again, the information given was quite vague, and I just want to reiterate that this blog is my interpretation of what was presented. It was never directly stated that Ataluren won't move forward. In their words, "we are working with regulatory bodies in the U.S. and Europe to determine the best path forward." What does THAT mean??
* Lastly, I want to mention that the first Plenary session today was amazing. There was a SHORT celebration of the success of Kalydeco, followed by details of their plans to move forward to treat the rest of the CF pop. They are impatient and understand the urgency to keep progressing. They now understand, in dizzying detail, the dysfunction of F508 and have a road map for correcting it. As mentioned earlier, there is positive buzz about the VX-809/VX-770 combo, but the "second generation" of combos already being tested in the lab have the potential to rescue 70-80% CFTR function--far exceeding the effect of Kalydeco for gating mutations. To achieve these results, the second generation drugs must address the multiple dysfunctions that this mutation presents: 1) folding, 2) stabilization, and 3) gating. I said it last year and I'll say it again: THEY HAVE IT FIGURED OUT. IT IS A MATTER OF TIME AND MONEY. I'm more optimistic than ever after a full day of lecture. I have so much more to say, but it is after midnight and I have another full day tomorrow. I plan to write summaries of the individual talks I attended today, which focused mainly on F508 correction and early CF lung disease. Brock and I also attended a fancy dinner this evening, where my Kalydeco tattoo was finally uncovered to be appreciated and photographed by many : ) The speakers at this dinner gave moving presentations that didn't leave a dry eye in the house. I am experiencing emotional overload. I may have finally exhausted myself to the point of sleep.
Thursday, October 11, 2012
Sunday, September 30, 2012
Brain Pain
I will never ever forget the fear that comes along with dealing with CF, but it has been over 7 months since Brady has been on Kalydeco now and I can feel myself adapting to a new normal. The fear is losing its grip and I've even found myself away from home WITHOUT hand sanitizer a time or two (gasp!). Before February 10th, when Brady took his first dose, I simply could not conceptualize what my post-Kalydeco world would look or feel like. Getting him that medicine was my singular focus and goal. Well, he got his medicine and time has marched on. Since I'm one of those people that like to have control of everything, this "after" time has been really refreshing for me. For once in my life, I feel like instead of making lists and plans...I am just enjoying the ride. There have been a few surprises for me along the way--some fantastic, others not so great.
#1--I thought that once Brady got Kalydeco, everything would be great and I could stop worrying about CYSTIC FIBROSIS. The reality is that I am tortured by the fact that others who could benefit from Kalydeco can't get it because of the many roadblocks to access. This feeling mixes with the guilt of being one of the first families to benefit and rips my heart to shreds. Overall, I feel extremely disillusioned with the world because people that I care about will get sicker or need a transplant because they don't have the $300K/year to pay for this drug. Or maybe their Dr. just isn't on board with writing the Rx because he isn't educated enough about the potential. It hurts my heart and my brain. This feeling is only amplified as I see how wonderful Kalydeco is working for Brady.
#2--I thought that once Brady got Kalydeco, I could stop worrying about him all the time and everything would be just peachy. What I've found instead, is that every parent worries about their children all the time no matter what. Now my worry has shifted to what some might consider more "normal" parent concerns. I spend a lot more time worried about Brady getting kidnapped (and hoping the kidnapper will at least remember to take the Kalydeco!), or hit by a car when he is playing outside. I worry about him picking the wrong person to date or experimenting with drugs...With CF on the sidelines, my mind has been more free to just wander. The realization I've come to is that there is ALWAYS going to be something to worry about. I can't control the world (as much as I want to!). All I can do is love my family and friends every day and cherish the health and happiness that we have in this moment.
#3--I have found tremendous JOB SATISFACTION. Let me just reiterate that I am unemployed (convenient, huh?). I don't work for the CF Foundation. I don't work for Vertex. I wondered what the hell I would do after Brady got Kalydeco and I didn't have to dedicate every second of my life to fighting for this medicine. I'm so lucky that my husband has an excellent job that has allowed me to stay home with Brady from birth to present. I've been able to teach Brady so many things in our time together and I've found myself a job as a caretaker/cheerleader/mentor/translator/fundraiser for the CF community. I remember the first few months after Brady's diagnosis as the darkest time of my life. Sharing research information with families makes me so happy because scientists are on the right track and there is GOOD NEWS. It is incredibly empowering and wonderful. I don't have any conflicts of interest. My only concern is helping CF patients and families get what they need.
#4--I thought that once Brady got Kalydeco, life would be calm and serene. Man I am an idiot. I've been so "worked up" since Brady started taking the drug that my insomnia is worse than ever! Since I've been subsiding largely on coffee and champagne for the last several weeks, I'm sure I'm working on an ulcer flare-up also. Watching Brady's transformation has been so thrilling. Every new test result sends me right back over the moon (like his most recent sweat test: 17mmol/L!! I walked around yelling 17?!?! for days). My heart is all wrapped up in all these other CF families and I constantly have YOU/YOUR child's mutations swimming around in my mind. I'm working on my little notebook to take to the NACFC with all the questions I want to ask, and all the specific mutations I want more information on. Basically, I've worked myself into a frenzy. And because I attended the Conference last year, I know exactly how awesome it is going to be! The thought of sitting down to dinner with "CF Celebrities" makes my palms sweat! Not to mention that I have a huge knot in my stomach wondering what new data will be presented because I know how much it means to CF patients and families. I know how much is riding on the results of that research. Will an effective combo be ready to market in 2016? Or will it take longer?
I got a haircut today, bought new luggage, and am trying to figure out what I'm going to wear?! It's crunch time! I seriously hope I don't just drop dead of a heart attack from all the excitement!
#1--I thought that once Brady got Kalydeco, everything would be great and I could stop worrying about CYSTIC FIBROSIS. The reality is that I am tortured by the fact that others who could benefit from Kalydeco can't get it because of the many roadblocks to access. This feeling mixes with the guilt of being one of the first families to benefit and rips my heart to shreds. Overall, I feel extremely disillusioned with the world because people that I care about will get sicker or need a transplant because they don't have the $300K/year to pay for this drug. Or maybe their Dr. just isn't on board with writing the Rx because he isn't educated enough about the potential. It hurts my heart and my brain. This feeling is only amplified as I see how wonderful Kalydeco is working for Brady.
#2--I thought that once Brady got Kalydeco, I could stop worrying about him all the time and everything would be just peachy. What I've found instead, is that every parent worries about their children all the time no matter what. Now my worry has shifted to what some might consider more "normal" parent concerns. I spend a lot more time worried about Brady getting kidnapped (and hoping the kidnapper will at least remember to take the Kalydeco!), or hit by a car when he is playing outside. I worry about him picking the wrong person to date or experimenting with drugs...With CF on the sidelines, my mind has been more free to just wander. The realization I've come to is that there is ALWAYS going to be something to worry about. I can't control the world (as much as I want to!). All I can do is love my family and friends every day and cherish the health and happiness that we have in this moment.
 |
| Enjoying our fall Farmer's Market! |
#3--I have found tremendous JOB SATISFACTION. Let me just reiterate that I am unemployed (convenient, huh?). I don't work for the CF Foundation. I don't work for Vertex. I wondered what the hell I would do after Brady got Kalydeco and I didn't have to dedicate every second of my life to fighting for this medicine. I'm so lucky that my husband has an excellent job that has allowed me to stay home with Brady from birth to present. I've been able to teach Brady so many things in our time together and I've found myself a job as a caretaker/cheerleader/mentor/translator/fundraiser for the CF community. I remember the first few months after Brady's diagnosis as the darkest time of my life. Sharing research information with families makes me so happy because scientists are on the right track and there is GOOD NEWS. It is incredibly empowering and wonderful. I don't have any conflicts of interest. My only concern is helping CF patients and families get what they need.
#4--I thought that once Brady got Kalydeco, life would be calm and serene. Man I am an idiot. I've been so "worked up" since Brady started taking the drug that my insomnia is worse than ever! Since I've been subsiding largely on coffee and champagne for the last several weeks, I'm sure I'm working on an ulcer flare-up also. Watching Brady's transformation has been so thrilling. Every new test result sends me right back over the moon (like his most recent sweat test: 17mmol/L!! I walked around yelling 17?!?! for days). My heart is all wrapped up in all these other CF families and I constantly have YOU/YOUR child's mutations swimming around in my mind. I'm working on my little notebook to take to the NACFC with all the questions I want to ask, and all the specific mutations I want more information on. Basically, I've worked myself into a frenzy. And because I attended the Conference last year, I know exactly how awesome it is going to be! The thought of sitting down to dinner with "CF Celebrities" makes my palms sweat! Not to mention that I have a huge knot in my stomach wondering what new data will be presented because I know how much it means to CF patients and families. I know how much is riding on the results of that research. Will an effective combo be ready to market in 2016? Or will it take longer?
 |
| Is "Brain Pain" a real condition?? |
I got a haircut today, bought new luggage, and am trying to figure out what I'm going to wear?! It's crunch time! I seriously hope I don't just drop dead of a heart attack from all the excitement!
Wednesday, September 19, 2012
NACFC Prep
Last year I attended the NACFC in Anaheim and blogged about my experiences there. I've been reviewing what I learned and working on my plan of attack to get the answers I'm looking for this year. Here is my first NACFC 2011 post if anyone else is interested in reviewing: http://luckycfmom.blogspot.com/2011/11/prelude-to-kiss.html
As I prepare to attend the NACFC next month in Orlando, I'm thinking mostly about:
1) Combo Trials--I will be looking for details on how both VX/770-VX/809 and VX/770-VX661 are progressing. I am also interested in seeing if they will be presenting a new 3 drug combination to start down the development pipeline. Last year, it was revolutionary news that they had discovered 2 misfolding problems that needed correction for DF508. In the lab, 3 drug combinations (2 correctors to treat each misfolding site + Kalydeco to open the CFTR channel once it reached the surface) worked REALLY WELL. A combination like this would be a few years behind the combos currently in trials, but has the potential to achieve the magnitude of change for DF508 that Kalydeco has shown for gating and conductance mutations. http://luckycfmom.blogspot.com/2012/02/genetic-origami.html
Also within the realm of combo trials, I am very curious how they plan to proceed in trials with heterozygotes. Is there new "transcomplementation" data being presented this year? http://luckycfmom.blogspot.com/2012/03/calling-all-heterozygotes.html What about the "trial of 1" concept proposed earlier this year by Peter Mueller of Vertex? http://iom.edu/~/media/Files/Activity%20Files/Research/GenomicBasedResearch/2012-MAR-21/3%20-%20Peter%20Mueller.pdf
Is this recently announced clinical trial a "trial in a box" as described by Mueller? http://clinicaltrials.gov/ct2/show/NCT01685801?term=vx-770&rank=19
2) Kalydeco questions like: Will they be moving forward to see Kalydeco marketed to COPD patients? http://www.sciguru.com/newsitem/14332/cystic-fibrosis-drug-kalydeco-may-be-useful-treating-copd-smokers If they open up the market to a large population like this, it could certainly affect the price of Kalydeco.
What the hell is going on in Europe and other parts of the world in terms of access...or rather, lack of access?!?! Everyone who read my last blog entry knows exactly how I feel about making Kalydeco available to those who need it.
I want to take the opportunity to discuss the many off-label prescriptions (because of both age and mutation) have successfully been filled and are showing positive results. Last year, everyone swore that we would be unable to get Kalydeco to Brady at age 4 1/2 and they were WRONG about that! Being at the NACFC is going to enable me to sit down to coffee with Bob and give him the real scoop on what is really happening in our CF world. Last year he called me his, "link" to patients and parents. I honestly think that the CF community is more empowered and educated than ever before because we've come together to share like a family (and we have the internet!). We are able to learn about the latest research from the comfort of our own homes. I love to hear how many parents are educating their child's DOCTOR about the latest in CF research and newest information on their child's particular genetic code. We have DEMANDED that our voices are heard, even when our own CF specialists might be hesitant. I would love to hear more detail on the timeline they see to opening up the FDA approved label to include other mutations and/or children under 6.
I have lots of other crazy question like: Could a pregnant woman take Kalydeco? Could meconium ileus, pancreatic insufficiency, and other complications be prevented if the mother of a fetus with G551D(or other mutation positively affected by Kalydeco) began taking the drug during pregnancy and then the infant began therapy at birth? Can you imagine?? What about how Kalydeco affects other organs besides the lungs? I've seen Brady's serious sinus issues disappear and that was not a reported or tested clinical trial parameter. Theoretically, Kalydeco should be able to prevent deterioration of other organs such as the liver and pancreas. Are clinics seeing this in practice?
I realize that there are no real answers to some of these questions, but I'm still pretty curious to sit down with some of these researchers and CFF execs and hash out the possibilities.
3) Ataluren
Please. What is the hold up with information regarding Ataluren?? Are they still planning on marketing Ataluren? I'd like to hear more detail about the side effects they recorded in the clinical trials. I'm also very curious to hear if they have tested an Ataluren + Kalydeco combination. This was a concept mentioned as a possiblity at last year's Conference. If non-toxic in combination, Kalydeco might be able to provide those important sweat chloride changes that they didn't see with Ataluren alone.
These CFTR modifiers are game-changers, and I hope I can report some amazing, awesome news this year! My husband Brock is attending the Conference with me this time, and we are a great team! We plan to split up during some sessions so we don't miss any important talks taking place during the same time slot. He is so excited to shake hands with Dr. Beall and the Vertex team. Last year, I was afraid that my heart might explode with gratitude as I sat side by side with these brilliant researchers...and that was BEFORE BRADY EVEN GOT KALYDECO! This year, the "thank you's" will mean even more!! Stay tuned...these blog updates are about to get really GOOD! Only 21 more days til Conference! If YOU have a burning question about CFTR modifiers that I didn't mention, leave me a comment and I'll add it to the list!
As I prepare to attend the NACFC next month in Orlando, I'm thinking mostly about:
1) Combo Trials--I will be looking for details on how both VX/770-VX/809 and VX/770-VX661 are progressing. I am also interested in seeing if they will be presenting a new 3 drug combination to start down the development pipeline. Last year, it was revolutionary news that they had discovered 2 misfolding problems that needed correction for DF508. In the lab, 3 drug combinations (2 correctors to treat each misfolding site + Kalydeco to open the CFTR channel once it reached the surface) worked REALLY WELL. A combination like this would be a few years behind the combos currently in trials, but has the potential to achieve the magnitude of change for DF508 that Kalydeco has shown for gating and conductance mutations. http://luckycfmom.blogspot.com/2012/02/genetic-origami.html
Also within the realm of combo trials, I am very curious how they plan to proceed in trials with heterozygotes. Is there new "transcomplementation" data being presented this year? http://luckycfmom.blogspot.com/2012/03/calling-all-heterozygotes.html What about the "trial of 1" concept proposed earlier this year by Peter Mueller of Vertex? http://iom.edu/~/media/Files/Activity%20Files/Research/GenomicBasedResearch/2012-MAR-21/3%20-%20Peter%20Mueller.pdf
Is this recently announced clinical trial a "trial in a box" as described by Mueller? http://clinicaltrials.gov/ct2/show/NCT01685801?term=vx-770&rank=19
2) Kalydeco questions like: Will they be moving forward to see Kalydeco marketed to COPD patients? http://www.sciguru.com/newsitem/14332/cystic-fibrosis-drug-kalydeco-may-be-useful-treating-copd-smokers If they open up the market to a large population like this, it could certainly affect the price of Kalydeco.
What the hell is going on in Europe and other parts of the world in terms of access...or rather, lack of access?!?! Everyone who read my last blog entry knows exactly how I feel about making Kalydeco available to those who need it.
I want to take the opportunity to discuss the many off-label prescriptions (because of both age and mutation) have successfully been filled and are showing positive results. Last year, everyone swore that we would be unable to get Kalydeco to Brady at age 4 1/2 and they were WRONG about that! Being at the NACFC is going to enable me to sit down to coffee with Bob and give him the real scoop on what is really happening in our CF world. Last year he called me his, "link" to patients and parents. I honestly think that the CF community is more empowered and educated than ever before because we've come together to share like a family (and we have the internet!). We are able to learn about the latest research from the comfort of our own homes. I love to hear how many parents are educating their child's DOCTOR about the latest in CF research and newest information on their child's particular genetic code. We have DEMANDED that our voices are heard, even when our own CF specialists might be hesitant. I would love to hear more detail on the timeline they see to opening up the FDA approved label to include other mutations and/or children under 6.
I have lots of other crazy question like: Could a pregnant woman take Kalydeco? Could meconium ileus, pancreatic insufficiency, and other complications be prevented if the mother of a fetus with G551D(or other mutation positively affected by Kalydeco) began taking the drug during pregnancy and then the infant began therapy at birth? Can you imagine?? What about how Kalydeco affects other organs besides the lungs? I've seen Brady's serious sinus issues disappear and that was not a reported or tested clinical trial parameter. Theoretically, Kalydeco should be able to prevent deterioration of other organs such as the liver and pancreas. Are clinics seeing this in practice?
I realize that there are no real answers to some of these questions, but I'm still pretty curious to sit down with some of these researchers and CFF execs and hash out the possibilities.
3) Ataluren
Please. What is the hold up with information regarding Ataluren?? Are they still planning on marketing Ataluren? I'd like to hear more detail about the side effects they recorded in the clinical trials. I'm also very curious to hear if they have tested an Ataluren + Kalydeco combination. This was a concept mentioned as a possiblity at last year's Conference. If non-toxic in combination, Kalydeco might be able to provide those important sweat chloride changes that they didn't see with Ataluren alone.
These CFTR modifiers are game-changers, and I hope I can report some amazing, awesome news this year! My husband Brock is attending the Conference with me this time, and we are a great team! We plan to split up during some sessions so we don't miss any important talks taking place during the same time slot. He is so excited to shake hands with Dr. Beall and the Vertex team. Last year, I was afraid that my heart might explode with gratitude as I sat side by side with these brilliant researchers...and that was BEFORE BRADY EVEN GOT KALYDECO! This year, the "thank you's" will mean even more!! Stay tuned...these blog updates are about to get really GOOD! Only 21 more days til Conference! If YOU have a burning question about CFTR modifiers that I didn't mention, leave me a comment and I'll add it to the list!
Sunday, September 9, 2012
Case Study
Our friends in Europe are still fighting to get Kalydeco. Patient advocacy groups have been rallying to pressure governments to move forward with marketing of the drug, but cost negotiations continue to stall distribution. We were asked to participate in a "case study" for our European friends to present to deciding bodies about the effects of Kalydeco. This is what I said,
"Brady Schroeder was born on July 26th, 2007. He tested positive on a newborn screen for CF
and we proceeded with genetic testing, which confirmed his diagnosis of cystic
fibrosis (G551D, DF508). Like most
children with CF, Brady has required digestive enzymes and special vitamins to
maintain proper growth. He began having
serious upper respiratory issues with sinus polyps at age 3. By age 4, his sinuses were completely
obstructed with CF related nasal polyps, and he had completely lost his sense
of smell. CT scan revealed that the
polyps had grown so large, that they had begun to thin and shift bones in
Brady’s face. Bursts of oral Prednisone
were used every 8-10 weeks to slow the polyp growth as well as 3X daily steroid
rinses for the sinuses. Even with
regular steroid and antibiotic use, the polyps required surgery when our son
was just 4 years old. While this was not
a life-threatening issue, it was a huge quality-of-life issue.
 |
| Surgery 1 of 2: Summer 2011 |
Even though Brady had maintained relatively good lung health
for a child with CF, he still did hours of daily nebulizer and airway clearance
treatments to slow the progression of CF lung disease. Prior to beginning Kalydeco, he did twice
daily Albuterol/Hypertonic Saline treatments as well as once daily
Pulmozyme. He also spent 20 minutes on
his Vest machine twice daily as maintenance.
The regimen of staying healthy with CF is extremely time consuming and
exhausting. It feels like fighting a
battle you know you will never win.
Every clinic visit seems to bring a new complication, a new infection,
and a new medicine. It becomes very hard
to look toward the future when you aren’t even sure how to get through the
current day. When CF is in your family,
the focus becomes keeping that person breathing, growing, and alive. Friendships and vacations tend to fall by the
wayside because an exacerbation or virus too frequently forces a change in
plans. It is impossible to describe the
pain and mental anguish of watching your child struggle to breathe within the
grips of this cowardly disease.
On February 10, 2012 Brady took his first dose of
Kalydeco. The first few days after
beginning the drug, we noticed a remarkable increase in his energy level and
exercise tolerance. Within 3 days, Brady
began breathing through his nose for the first time in over a year. He stopped snoring and breathing through his
mouth at mealtime. Within a week he began
to make non-stop comments about smells in his environment. Brady hasn’t needed a single dose of
Prednisone or antibiotics since beginning Kalydeco, and has also completely
stopped the steroid rinses for his sinuses!
It has been a truly phenomenal turn-around that shocked his
Ear/Nose/Throat specialist. Before
Kalydeco, we were seeing the ENT on a monthly basis. Post Kalydeco, we have only seen him once (to
document the positive effects of Kalydeco!).
Brady’s appetite increased noticeably and his growth has
been absolutely phenomenal since starting Kalydeco. He has grown 3 inches in just 6 months on the
drug and has gained over 3 lbs, putting him into the 90th percentile
for both height and weight-for-age! He
has a decreased need for digestive enzymes, and has fewer stomach aches and CF
digestive issues in general.
 |
| Camping trip, summer 2012. Best summer of our lives! |
Brady had a dramatic drop in sweat chloride with
Kalydeco. When he began on Feb. 10, 2012
his sweat chloride was measured at 105 mmol/L.
After 20 days on the drug, we retested and the number had dropped to 48
mmol/L. After more than 6 months on
Kalydeco, we repeated the sweat test and were amazed to hear that it had
dropped to 17 mmol/L—completely within normal range, indicating properly
functioning CFTR. The implications of getting
a genetic modifying drug like Kalydeco to our son at the early stages of
disease are immense. Because cystic
fibrosis is a degenerative, passage of time generally equates to permanent
organ damage as a result of dysfunctional CFTR.
Because Brady had suffered little to no permanent lung damage before
beginning Kalydeco (as seen in CT scan), he has been able to greatly reduce the
number of maintenance treatments he does on a daily basis. We have completely stopped the twice daily
Hypertonic Saline treatments. We are
currently tapering down from Pulmozyme treatments and spending reduced time in
the Vest also. We will continue to be
cautious and slowly reduce his treatments under the advisement of his CF
specialist, but we can clearly see the day in the near future that our son will
no longer need any nebulizer treatments or airway clearance treatments on a
daily basis. Kalydeco has given our
young son a second chance at long, healthy, “normal” life. He will likely NEVER be hospitalized again as
a result of CF. He will not need a
lifetime of intravenous and inhaled antibiotics and steroids. I will not have
to watch him struggle to breathe. There
is simply no way to accurately describe the enormous positive impact Kalydeco
has had on Brady and our entire family!
Watching a disease like cystic fibrosis destroy your child is any
parent’s worst nightmare. You
desperately wish there was something you could do to help. You would give anything in the world to ease
their suffering—Kalydeco is that miracle.
As someone who has seen the power of this drug first hand, I urge you to
not only consider the price, but also the mountain of clinical and anecdotal
evidence that this drug lives up to its reputation. Unfortunately,
in the CF world, time is not a luxury that we enjoy. I respectfully urge you to approve and begin
marketing Kalydeco to those who need it immediately. I would be happy to provide further detail
and/or documentation of the claims made in this letter. It has been such a life-changing miracle for
us, that my husband and I are flying across the U.S. to attend the NACFC in
Orlando next month. Shaking hands with
the Vertex team that developed Kalydeco is going to be one of the most memorable,
intense moments of my life!
We haven’t seen any negative side effects of Kalydeco. We have regularly tested Brady’s liver
enzymes and they have remained stable and within the normal range. The positive side-effects of Brady beginning
Kalydeco have included: more energy, more restful sleep, restored sense of
smell, complete disappearance of serious nasal polyps, increased appetite, increased rate of growth,
fewer digestive problems, reduced breathing treatment time, reduced airway
clearance time, no further need for antibiotics or steroids, and much more
normally functioning organs. Those who
love Brady have experienced a huge reduction in stress and anxiety. We now have the ability to look toward the
future, and an endless gratitude for modern medicine. It is absolutely critical to get Kalydeco to
those who need it in the timeliest manner possible.
Sincerely,
Rebecca Schroeder, Mother of Brady Schroeder age 5."
I also included the link to the news interview about Brady and Kalydeco: http://www.krem.com/news/health/New-drug-provides-miracle-for-4-year-old-CDA-boy-battling-Cystic-Fibrosis-148978145.html
I think it is pretty clear that I am a Kalydeco "super fan"! I'm already buzzing with excitement for the NACFC next month! Look for some exciting blog updates!
Sunday, July 22, 2012
Tricks of the Trade
Originally, I started this blog to document the experience of getting Kalydeco to Brady. Now that Brady has been taking it for 5+ months and is doing so great, I don't have too many new exciting changes to report (boring is so AWESOME!)...so I'm going to have to expand the content of my blog a bit if I want to keep writing!
Recently, two families with newly diagnosed infants with G551D have contacted me with a few questions about CF and Kalydeco. It feels like such an enormous privilege to be able to help those people through such a scary time. Of course, I get to try to explain to them how lucky they are to have been given that G551D, because right now that is like the golden ticket in the CF world. But also, I get to simply help them understand this new CF Club they have unwillingly become a part of. This blog is dedicated to "the newbies" and anyone else who just wants to know some of the tricks of the trade I have learned to make CF life healthier and easier :). These are my top 10 "secrets of the pros" that I wish someone had told me about when Brady was diagnosed:
1) Invacare Mobilaire 50 psi compressor. Insurance might not cover this compressor, but I swear it is worth every penny out-of-pocket. We bought this bad boy when Brady was about 9 months old and it is still going strong today (Brady is almost 5). I love the adjustable pressure feauture, because you can crank it full blast for meds that are not "dose dependent," and adjust the pressure appropriately for other meds that you want to nebulize more slowly to get the maximum dosage out of. It is more powerful (higher psi) than smaller less expensive models and actually blasts medicines into smaller particles, allowing drugs to reach the smallest of airways (*Side note, this is the compressor recommended by Genentech for use with Pulmozyme). http://www.pulmozyme.com/taking/treatment.html. The best thing about this compressor is that it will allow you to nebulize medicines FAST and will cut treatment time for most meds. When you have a baby or toddler getting used to treatments...reducing the time it takes can be HUGE! I give this compressor two big "thumbs up" and if it ever breaks down, we will likely buy another one to replace it! We ordered ours online and I've seen them range in price from about $250-$315 or so. http://www.allegromedical.com/respiratory-therapy-c534/mobilaire-50-psi-compressor-p551975.html
2) Omron Micro Air. Treatments are a part of life with CF. Why not make them as easy as possible? We just recently acquired this handy little device for treatments on-the-go and so far I'm impressed! It takes 2 AA batteries to operate and uses a new "vibrating mesh" technology to break medicines into a fine mist. It is seriously almost SILENT when it is nebulizing a medicine! We used it on our recent camping trip and it was really awesome to watch Brady sit shirtless and do his treatments in the woods! I can't comment on the durability yet, because it is still new. I like having this device around and wish we had gotten it sooner!
3) Philips Avent iQ24 Steam Sterilizer and Germ Guardian
There are lots of different ways to keep your nebulizer equipment clean. This is how we do it. First, wash the neb parts in warm soapy water (I use a clean paper towel to wipe down parts), rinse well, and then place them in the electric steam sterilizer http://www.amazon.com/Philips-AVENT-iQ24-Steam-Sterilizer/dp/B001C3MHF0. It takes 6 minutes to sterilize. Then I move the neb parts to the Germ Guardian, http://www.onestepahead.com/catalog/product.jsp?productId=487759 which uses dry heat to kill germs and as a side-effect, quickly dries the nebulizer parts with hot air. **Always make sure to handle nebulizer parts with freshly washed hands!! After the parts are dry, I toss them in their special tupperware in the cupboard. Voila! Clean and ready to use again!
4) Power Inverter for Car
We recently got a power inverter installed in our vehicle and I wish we had done it a long time ago! Power goes out--no problem! You can still do treatments in the car! Travelling and don't want to stop for an hour and find a place to do treatments? No problem! Do your treatments en route. We bought our inverter and cables online and had a local stereo shop install it for us. We got this inverter: http://www.theinverterstore.com/1250-watt-power-inverter.html You have to order the cables separately. It is also pretty handy to have a power source in your car when you need to plug-in the air pump to inflate your camping mattress or water toys at the lake :).
5) Hand Hygiene
This is the #1 way to prevent the spread of germs and I can't underestimate the value of maintaining really good hand hygiene for your CFer. For us, this means that I pack hand sanitizer in my purse and use it frequently when I'm out and about. I use my own pen rather than the one at the counter. I keep hand sanitizer in the door of my car and use a bit everytime I get back in the car from being in the grocery store etc... In our house, we wash everytime we come in from being outside (playing, running errands, whatever). I installed paper towel dispensers in the kitchen and bathrooms in my house. Hand towels remain damp for long periods of time and get used by everyone. They are bacterial traps and we don't use them. Using a clean paper towel to dry your hands and then throwing it away is a much more effective way to keep germs from getting around. I found a company that makes a less industrial looking paper towel holder and I buy paper towel refills at Costco. http://brickhousedispensers.com/
6) Home Hygiene
I realize that many of the bugs that can affect CFers are ambient in our environment and cannot ever be completely eliminated. With that said, there are many things you can do around the home to reduce the concentration of the most harmful bacteria and reduce the spread of viruses. Brock and I team up for our cleaning routine every single weekend. For us, cleaning is less about tidying up and more about killing! Our weekly routine includes:
*Wipe down all doorknobs, kitchen cupboard handles, lightswitches, cell phones, remote controls, and any other frequently touched items with a disinfecting wipe.
* Vacuum carpeted areas with our UVC vacuum (UVC is a wavelength of light that kills bacteria, viruses, mold spores, and dust mites upon exposure).
* Steam clean our hardwood and ceramic tile floors with the trusty Steam Shark (steam mop). I love cleaning with UVC and steam because no chemicals are involved and there is no irritating smell left behind.
*Clean showers and sinks frequently (any place that remains wet for longer periods of time is going to be a bacterial "hot spot"). It might be worth paying some special attention to your showerhead with a bleach/water soak every few weeks. http://www.boston.com/news/nation/articles/2009/09/15/bacteria_in_showerheads_may_be_harmful_study_finds/ It is extremely important to follow package instructions on cleaning products for appropriate disinfection. Products that are not left on long enough or applied in the appropriate concentration can actually contribute to bacterial resistance.
*We wash Brady's clothes, bedding, and towels in HOT water. Laundry can be another unexpected source of bacteria. It is important to move wet clean laundry to the dryer right away after washing, and dry completely. http://abcnews.go.com/Health/Wellness/washing-machines-loaded-bacteria-dirty-clothes/story?id=10751420
*No shoes in the house. We remove our shoes at the door to avoid bringing germs and bacteria into the home via the bottom of our shoes. We ask our guests to do the same. I keep a small basket of slippers and cozy socks by the front door in the winter so guests can keep their feet nice and warm.
If anyone wants more detail on our cleaning schedule or UVC technology, I wrote a whole blog about it and even made a little video! http://luckycfmom.blogspot.com/2011_04_01_archive.html
7) Smart Exposure
As much as I'd like to, I can't control the outside world. I try to limit Brady's exposure to germs in public places with smart scheduling. For example, I always make Brady's Dr. or CF clinic appts. first thing in the morning, when the waiting room isn't full of sick kids and the exam rooms haven't been recently occupied by patients. In the middle of winter (cold and flu season), I don't take Brady with me to the grocery store during the busiest times of day, when the store is packed with people. We schedule our trips early in the morning or on Friday or Saturday nights (when the rest of the world seems to have other/fun things to do!). Our friends are fully aware of our "sick policy," which states that if you are currently ill...we don't want to hang out. Let me stress that we DO NOT LIVE IN A BUBBLE. I just try to make smart decisions about Brady's exposure and I think it has gone a long way.
8) The Bravery Box
I keep a special box of toys and books to use when Brady has to undergo an unpleasant medical procedure like a blood draw. I tell Brady what I'm offering (a book or little stuffed animal he has been wanting) in exchange for his bravery and holding still during the procedure. Most of the time, he can't wait to get to the lab or wherever so he can get his reward! The rewards don't have to be expensive, just whatever you know your kid will go the extra mile for! The Bravery Box has made our lives so much easier and Brady typically feels really heroic and awesome after he successfully earns a bravery reward!
9) Praise Praise Praise Compliance to Breathing Treatments and Meds.
From the time Brady began breathing treatments at 6 mo. of age, we have tried hard to make treatment time special. We pay loads of attention to Brady during this time--because every kid wants attention from their parents! Brady gets to watch whatever he wants on TV during treatments and I usually sit with him and massage his legs. Figure out what YOUR kid likes best and praise the crap out of them when they do things RIGHT! It is so easy to wait until they start misbehaving or throwing a fit for treatments before you pay attention...but it is more effective to simply praise them when they are being responsible by taking all their meds and doing great! We still give Brady a high-five and tell him how proud we are of him after every single treatment session! Not only has it kept him healthy, it is a great confidence builder because staying compliant to treatments is hard work and he knows it!
10) Don't Forget about Nutrition
Most CF patients visit with a dietician during clinic. We know that our CFers need more fat and calories...but what about actual nutrition?? Doctors in Europe pay closer attention to the nutrition aspect of CF and encourage a "Mediterranean style" diet rich in healthy fats. Here in the U.S., Doctors tend to focus more on quantity vs.quality of calories and food consumed. The problem with this is that the CFers need more than just extra calories and fat--they also need more NUTRITION! CFers struggle to metabolize fat soluble nutrients in particular--vitamins A, D, E, and K (hence the specialty CF vitamins). A diet rich in antioxidants is vital to maintain good health for ANYONE and is particularly important for individuals with CF. Brady isn't always cooperative with his diet, so I give him some antioxidant supplements to help fill in the gaps. I try to focus on healthy sources of fat like those found in avocados, coconut, nuts, and seeds rather than relying on junk food filled with sugar and unhealthy trans fats. We like JuicePlus Chewables (whole food gummies made with fruits and veggies) https://www.juiceplus.com/nsa/content/OtherProducts.soa. I'm also a fan of the supplement NAC (N-acetyl cysteine) . It is a powerful antioxidant that is most effective in the effervescent form sold by Bioadvantex (NAC is an easily oxidized compound. Bioadvantex produces its NAC in an oxygen-free environment to preserve potency). http://www.bioadvantex.com/us/pharmanac.html My husband and I are also big proponents of juicing fresh fruits and veggies. We juice every day! Brady still turns his nose up at our green juice blends...but I think adding concentrated nutrition to the diet through juicing would be beneficial to ANYONE--especially CFers!
I hope people find some useful tips in this entry! These are the things that have made our CF lives easier! Remember that this isn't necessarily the "right" way to do things...it is just what works for us!
Recently, two families with newly diagnosed infants with G551D have contacted me with a few questions about CF and Kalydeco. It feels like such an enormous privilege to be able to help those people through such a scary time. Of course, I get to try to explain to them how lucky they are to have been given that G551D, because right now that is like the golden ticket in the CF world. But also, I get to simply help them understand this new CF Club they have unwillingly become a part of. This blog is dedicated to "the newbies" and anyone else who just wants to know some of the tricks of the trade I have learned to make CF life healthier and easier :). These are my top 10 "secrets of the pros" that I wish someone had told me about when Brady was diagnosed:
1) Invacare Mobilaire 50 psi compressor. Insurance might not cover this compressor, but I swear it is worth every penny out-of-pocket. We bought this bad boy when Brady was about 9 months old and it is still going strong today (Brady is almost 5). I love the adjustable pressure feauture, because you can crank it full blast for meds that are not "dose dependent," and adjust the pressure appropriately for other meds that you want to nebulize more slowly to get the maximum dosage out of. It is more powerful (higher psi) than smaller less expensive models and actually blasts medicines into smaller particles, allowing drugs to reach the smallest of airways (*Side note, this is the compressor recommended by Genentech for use with Pulmozyme). http://www.pulmozyme.com/taking/treatment.html. The best thing about this compressor is that it will allow you to nebulize medicines FAST and will cut treatment time for most meds. When you have a baby or toddler getting used to treatments...reducing the time it takes can be HUGE! I give this compressor two big "thumbs up" and if it ever breaks down, we will likely buy another one to replace it! We ordered ours online and I've seen them range in price from about $250-$315 or so. http://www.allegromedical.com/respiratory-therapy-c534/mobilaire-50-psi-compressor-p551975.html
2) Omron Micro Air. Treatments are a part of life with CF. Why not make them as easy as possible? We just recently acquired this handy little device for treatments on-the-go and so far I'm impressed! It takes 2 AA batteries to operate and uses a new "vibrating mesh" technology to break medicines into a fine mist. It is seriously almost SILENT when it is nebulizing a medicine! We used it on our recent camping trip and it was really awesome to watch Brady sit shirtless and do his treatments in the woods! I can't comment on the durability yet, because it is still new. I like having this device around and wish we had gotten it sooner!
 |
| http://omronmicroair.com/ |
There are lots of different ways to keep your nebulizer equipment clean. This is how we do it. First, wash the neb parts in warm soapy water (I use a clean paper towel to wipe down parts), rinse well, and then place them in the electric steam sterilizer http://www.amazon.com/Philips-AVENT-iQ24-Steam-Sterilizer/dp/B001C3MHF0. It takes 6 minutes to sterilize. Then I move the neb parts to the Germ Guardian, http://www.onestepahead.com/catalog/product.jsp?productId=487759 which uses dry heat to kill germs and as a side-effect, quickly dries the nebulizer parts with hot air. **Always make sure to handle nebulizer parts with freshly washed hands!! After the parts are dry, I toss them in their special tupperware in the cupboard. Voila! Clean and ready to use again!
4) Power Inverter for Car
We recently got a power inverter installed in our vehicle and I wish we had done it a long time ago! Power goes out--no problem! You can still do treatments in the car! Travelling and don't want to stop for an hour and find a place to do treatments? No problem! Do your treatments en route. We bought our inverter and cables online and had a local stereo shop install it for us. We got this inverter: http://www.theinverterstore.com/1250-watt-power-inverter.html You have to order the cables separately. It is also pretty handy to have a power source in your car when you need to plug-in the air pump to inflate your camping mattress or water toys at the lake :).
 |
| We had our inverter installed under the driver's side seat, so the plug-ins are easily accessible for Brady to do his treatments in the backseat while we are travelling. |
5) Hand Hygiene
This is the #1 way to prevent the spread of germs and I can't underestimate the value of maintaining really good hand hygiene for your CFer. For us, this means that I pack hand sanitizer in my purse and use it frequently when I'm out and about. I use my own pen rather than the one at the counter. I keep hand sanitizer in the door of my car and use a bit everytime I get back in the car from being in the grocery store etc... In our house, we wash everytime we come in from being outside (playing, running errands, whatever). I installed paper towel dispensers in the kitchen and bathrooms in my house. Hand towels remain damp for long periods of time and get used by everyone. They are bacterial traps and we don't use them. Using a clean paper towel to dry your hands and then throwing it away is a much more effective way to keep germs from getting around. I found a company that makes a less industrial looking paper towel holder and I buy paper towel refills at Costco. http://brickhousedispensers.com/
6) Home Hygiene
I realize that many of the bugs that can affect CFers are ambient in our environment and cannot ever be completely eliminated. With that said, there are many things you can do around the home to reduce the concentration of the most harmful bacteria and reduce the spread of viruses. Brock and I team up for our cleaning routine every single weekend. For us, cleaning is less about tidying up and more about killing! Our weekly routine includes:
*Wipe down all doorknobs, kitchen cupboard handles, lightswitches, cell phones, remote controls, and any other frequently touched items with a disinfecting wipe.
* Vacuum carpeted areas with our UVC vacuum (UVC is a wavelength of light that kills bacteria, viruses, mold spores, and dust mites upon exposure).
* Steam clean our hardwood and ceramic tile floors with the trusty Steam Shark (steam mop). I love cleaning with UVC and steam because no chemicals are involved and there is no irritating smell left behind.
*Clean showers and sinks frequently (any place that remains wet for longer periods of time is going to be a bacterial "hot spot"). It might be worth paying some special attention to your showerhead with a bleach/water soak every few weeks. http://www.boston.com/news/nation/articles/2009/09/15/bacteria_in_showerheads_may_be_harmful_study_finds/ It is extremely important to follow package instructions on cleaning products for appropriate disinfection. Products that are not left on long enough or applied in the appropriate concentration can actually contribute to bacterial resistance.
*We wash Brady's clothes, bedding, and towels in HOT water. Laundry can be another unexpected source of bacteria. It is important to move wet clean laundry to the dryer right away after washing, and dry completely. http://abcnews.go.com/Health/Wellness/washing-machines-loaded-bacteria-dirty-clothes/story?id=10751420
*No shoes in the house. We remove our shoes at the door to avoid bringing germs and bacteria into the home via the bottom of our shoes. We ask our guests to do the same. I keep a small basket of slippers and cozy socks by the front door in the winter so guests can keep their feet nice and warm.
If anyone wants more detail on our cleaning schedule or UVC technology, I wrote a whole blog about it and even made a little video! http://luckycfmom.blogspot.com/2011_04_01_archive.html
7) Smart Exposure
As much as I'd like to, I can't control the outside world. I try to limit Brady's exposure to germs in public places with smart scheduling. For example, I always make Brady's Dr. or CF clinic appts. first thing in the morning, when the waiting room isn't full of sick kids and the exam rooms haven't been recently occupied by patients. In the middle of winter (cold and flu season), I don't take Brady with me to the grocery store during the busiest times of day, when the store is packed with people. We schedule our trips early in the morning or on Friday or Saturday nights (when the rest of the world seems to have other/fun things to do!). Our friends are fully aware of our "sick policy," which states that if you are currently ill...we don't want to hang out. Let me stress that we DO NOT LIVE IN A BUBBLE. I just try to make smart decisions about Brady's exposure and I think it has gone a long way.
8) The Bravery Box
I keep a special box of toys and books to use when Brady has to undergo an unpleasant medical procedure like a blood draw. I tell Brady what I'm offering (a book or little stuffed animal he has been wanting) in exchange for his bravery and holding still during the procedure. Most of the time, he can't wait to get to the lab or wherever so he can get his reward! The rewards don't have to be expensive, just whatever you know your kid will go the extra mile for! The Bravery Box has made our lives so much easier and Brady typically feels really heroic and awesome after he successfully earns a bravery reward!
9) Praise Praise Praise Compliance to Breathing Treatments and Meds.
From the time Brady began breathing treatments at 6 mo. of age, we have tried hard to make treatment time special. We pay loads of attention to Brady during this time--because every kid wants attention from their parents! Brady gets to watch whatever he wants on TV during treatments and I usually sit with him and massage his legs. Figure out what YOUR kid likes best and praise the crap out of them when they do things RIGHT! It is so easy to wait until they start misbehaving or throwing a fit for treatments before you pay attention...but it is more effective to simply praise them when they are being responsible by taking all their meds and doing great! We still give Brady a high-five and tell him how proud we are of him after every single treatment session! Not only has it kept him healthy, it is a great confidence builder because staying compliant to treatments is hard work and he knows it!
10) Don't Forget about Nutrition
Most CF patients visit with a dietician during clinic. We know that our CFers need more fat and calories...but what about actual nutrition?? Doctors in Europe pay closer attention to the nutrition aspect of CF and encourage a "Mediterranean style" diet rich in healthy fats. Here in the U.S., Doctors tend to focus more on quantity vs.quality of calories and food consumed. The problem with this is that the CFers need more than just extra calories and fat--they also need more NUTRITION! CFers struggle to metabolize fat soluble nutrients in particular--vitamins A, D, E, and K (hence the specialty CF vitamins). A diet rich in antioxidants is vital to maintain good health for ANYONE and is particularly important for individuals with CF. Brady isn't always cooperative with his diet, so I give him some antioxidant supplements to help fill in the gaps. I try to focus on healthy sources of fat like those found in avocados, coconut, nuts, and seeds rather than relying on junk food filled with sugar and unhealthy trans fats. We like JuicePlus Chewables (whole food gummies made with fruits and veggies) https://www.juiceplus.com/nsa/content/OtherProducts.soa. I'm also a fan of the supplement NAC (N-acetyl cysteine) . It is a powerful antioxidant that is most effective in the effervescent form sold by Bioadvantex (NAC is an easily oxidized compound. Bioadvantex produces its NAC in an oxygen-free environment to preserve potency). http://www.bioadvantex.com/us/pharmanac.html My husband and I are also big proponents of juicing fresh fruits and veggies. We juice every day! Brady still turns his nose up at our green juice blends...but I think adding concentrated nutrition to the diet through juicing would be beneficial to ANYONE--especially CFers!
I hope people find some useful tips in this entry! These are the things that have made our CF lives easier! Remember that this isn't necessarily the "right" way to do things...it is just what works for us!
Tuesday, June 19, 2012
Eye of the Storm
I can still feel CF swirling around me--like a devastating tornado. I turn on my computer every morning and survey the damage. Almost everyone in my CF circle does NOT have Kalydeco or any type of "miracle drug." They are dealing with shitty old fashioned CF, that is a relentless and degenerative disease. The more time that passes, the more perspective I'm able to gather on what is happening for Brady and our whole family right now. Brady saw his CF specialist last Thursday. It has been two months since our last check-up and 4 months total since he started Kalydeco. I want to continue summarizing his progress. You can check out his April visit here: http://luckycfmom.blogspot.com/2012/04/new-brand-of-cystic-fibrosis.html
Cough: None
Weight: 48 lbs. Same as last visit. I wish he had gained at least a little weight, but I feel pretty good about his growth in general. Brady has a tendency to grow either up our out...but not both at once. Plus, we live in Northern Idaho, and the weather has been bad for a long time. We are finally getting some sunny days and Brady's activity level has gone WAY up. Sometimes I swear that those Kalydeco tabs are laced with a tiny bit of crack cocaine because there has been such a noticable change in his energy level. We also did some experimenting with his enzyme dosing, which is further explained below.
Height: 44 1/2 inches. He grew an inch and a half in the last two months!
Pancreatic Function: At the April clinic visit, we had decided to play around with his enzyme dosage to see if we were giving him the appropriate amount. After a small reduction, Brady began to lose a bit of weight, so we decided to keep him at his original enzyme dosage (3 Zenpep with meals, 2 with snacks). He hasn't been complaining of any stomach aches and his poo has been very "normal." It doesn't appear that Kalydeco is going to do much for Brady in terms of reducing his enzyme needs. His pancreatic insufficiency has always been severe. His pancreas just stopped working one day, when Brady was a little over 2 weeks old. He may still need enzymes, but I can say without a doubt that Kalydeco has offered him many other digestive benefits.
Breathing Treatments and Vest: When Brady began Kalydeco on Feb. 10th, his schedule looked like this:
Baseline Morning: Albuterol and Hypersal via nebulizer followed by 20 minutes on the VEST. Total time spent was about 45 min.
Baseline Evening: Albuterol, Pulmozyme, and Hypersal via nebulizer followed by 20 min on the VEST. Total time spent was about 50 min.
After 4 months on Kalydeco, his treatment schedule is now:
Morning: Twice a week (Monday and Thurs) we do Albuterol and Hypersal in the morning via neb. We are in the process of weaning Brady completely off Hypersal. It is incredible to me that we used to do this treatment 14 times every week and now we are down to 2 times a week. Our plan is to continue twice a week Hypersal for a month, then drop to once a week Hypersal for another month...then DONE! We will continue to use Hypersal if Brady is sick, but we no longer feel like he needs this for maintance. Brady continues to use his VEST every morning, but the time has been reduced from 20 to 15 minutes. So now, there are 5 days every week where we wake up and do NO nebulizer treatments. 15 minutes on the VEST and he is done! It is barely enough time for me to cook his breakfast! It feels like we have so much extra time in the morning!
Evening: Another change that happened at the last clinic visit is that we have started using "The Vortex" (a holding chamber with a mask attachment) device with a regular Albuterol inhaler, rather than nebulizing Albuterol. http://www.pari.com/products/holding_chambers/product/detail/info/smarttouch_pediatric_vortex_masks.html
When Brady wakes up from his afternoon nap, I give him a couple of puffs on the Vortex (just takes a minute). He eats a snack while he wakes up and then does his Pulmozyme treatment via nebulizer followed by 15 minutes on the VEST. Total treatment time in the evening is about 22 min. vs. the 50 min. we used to spend each night.
Other Oral Meds: Holding steady. We haven't dropped any of his other meds.
Sinuses: Kalydeco seems to have completely resolved Brady's previously serious sinus issues.
Baseline: Brady had undergone 1 sinus surgery and did a round of oral Prednisone every 6-8 weeks to manage his polyp growth. He also did steroid rinses for his sinuses 3 times every day using a device called the Nasatouch: http://www.sinusdynamics.com/
Today: Brady's sinuses are clear and he is breathing freely. His sense of smell has obviously returned, as he smells everything around him! "Mom, smell this rock." He NEVER used to comment on smells. He hasn't taken any oral steroids in over 4 months now. We also decreased and then eventually stopped all the steroid rinses also. Today, we do NOTHING for his sinuses. He stopped snoring after 3 days on Kalydeco and we haven't heard it since. Brady's sinus issues had a real negative impact on his quality of life and the side-effects of Prednisone are less than pleasant. To be completely free from this issue feels like such a miracle. It is something we used to battle on a daily basis and now the issue is GONE. We visited Brady's Ear/Nose/Throat Dr. a few weeks ago to document how well he is doing and the Dr. remarked that Brady's sinus tissues actually "look different" than before. By the end of the visit, he was asking about Vertex stock as an investment opportunity. I'd say he was pretty impressed!
Mental Outlook: Before Kalydeco, I felt like I was swirling around in the dangerous winds of that tornado. You feel so out of control and every once in a while you get flung into the side of a brick wall. Going to clinic paralyzed me with fear. What new meds are they going to add? Will I be able to get Brady to actually swallow/inhale/sit still through it? What will his throat culture show? Will his lungs be clear? CF never rests and that means that you can never rest. You have to fight CF CONSTANTLY. It is so damn exhausting and wild rides make me sick to my stomach...
Today, we still have treatments to do and Brady still takes lots of pills, but it is eerily calm right here. I can see the winds raging violently all around me...but it feels like Kalydeco has led us to the eye of the storm. Life feels oddly light-hearted and non-eventful. I have an enormous amount of appreciation for the little chunks of time we have been getting back over the last 4 months. I've replaced some of our morning treatment time with back massages (I've been trained in Massage Therapy and Brady has finally gotten wise to it). In the evenings now, we spent more time playing outside because treatment time is so short. An evolution of "normal" has taken place in our home. Kalydeco has been such a gift for all of us. I calculated that Brady has ingested $105,264 (258 pills @$408/pill) worth of Kalydeco so far. I'm so thankful that our insurance has decided that Brady is "worth it." I feel like my experiences with CF and Kalydeco have allowed me to place my priorities firmly in the correct order and live my life accordingly. And while I greatly appreciate the calm that the eye of the storm provides...I think we would all prefer that the storm would just GO AWAY ALEADY!
Cough: None
Weight: 48 lbs. Same as last visit. I wish he had gained at least a little weight, but I feel pretty good about his growth in general. Brady has a tendency to grow either up our out...but not both at once. Plus, we live in Northern Idaho, and the weather has been bad for a long time. We are finally getting some sunny days and Brady's activity level has gone WAY up. Sometimes I swear that those Kalydeco tabs are laced with a tiny bit of crack cocaine because there has been such a noticable change in his energy level. We also did some experimenting with his enzyme dosing, which is further explained below.
Height: 44 1/2 inches. He grew an inch and a half in the last two months!
Pancreatic Function: At the April clinic visit, we had decided to play around with his enzyme dosage to see if we were giving him the appropriate amount. After a small reduction, Brady began to lose a bit of weight, so we decided to keep him at his original enzyme dosage (3 Zenpep with meals, 2 with snacks). He hasn't been complaining of any stomach aches and his poo has been very "normal." It doesn't appear that Kalydeco is going to do much for Brady in terms of reducing his enzyme needs. His pancreatic insufficiency has always been severe. His pancreas just stopped working one day, when Brady was a little over 2 weeks old. He may still need enzymes, but I can say without a doubt that Kalydeco has offered him many other digestive benefits.
Breathing Treatments and Vest: When Brady began Kalydeco on Feb. 10th, his schedule looked like this:
Baseline Morning: Albuterol and Hypersal via nebulizer followed by 20 minutes on the VEST. Total time spent was about 45 min.
Baseline Evening: Albuterol, Pulmozyme, and Hypersal via nebulizer followed by 20 min on the VEST. Total time spent was about 50 min.
After 4 months on Kalydeco, his treatment schedule is now:
Morning: Twice a week (Monday and Thurs) we do Albuterol and Hypersal in the morning via neb. We are in the process of weaning Brady completely off Hypersal. It is incredible to me that we used to do this treatment 14 times every week and now we are down to 2 times a week. Our plan is to continue twice a week Hypersal for a month, then drop to once a week Hypersal for another month...then DONE! We will continue to use Hypersal if Brady is sick, but we no longer feel like he needs this for maintance. Brady continues to use his VEST every morning, but the time has been reduced from 20 to 15 minutes. So now, there are 5 days every week where we wake up and do NO nebulizer treatments. 15 minutes on the VEST and he is done! It is barely enough time for me to cook his breakfast! It feels like we have so much extra time in the morning!
Evening: Another change that happened at the last clinic visit is that we have started using "The Vortex" (a holding chamber with a mask attachment) device with a regular Albuterol inhaler, rather than nebulizing Albuterol. http://www.pari.com/products/holding_chambers/product/detail/info/smarttouch_pediatric_vortex_masks.html
When Brady wakes up from his afternoon nap, I give him a couple of puffs on the Vortex (just takes a minute). He eats a snack while he wakes up and then does his Pulmozyme treatment via nebulizer followed by 15 minutes on the VEST. Total treatment time in the evening is about 22 min. vs. the 50 min. we used to spend each night.
Other Oral Meds: Holding steady. We haven't dropped any of his other meds.
Sinuses: Kalydeco seems to have completely resolved Brady's previously serious sinus issues.
Baseline: Brady had undergone 1 sinus surgery and did a round of oral Prednisone every 6-8 weeks to manage his polyp growth. He also did steroid rinses for his sinuses 3 times every day using a device called the Nasatouch: http://www.sinusdynamics.com/
Today: Brady's sinuses are clear and he is breathing freely. His sense of smell has obviously returned, as he smells everything around him! "Mom, smell this rock." He NEVER used to comment on smells. He hasn't taken any oral steroids in over 4 months now. We also decreased and then eventually stopped all the steroid rinses also. Today, we do NOTHING for his sinuses. He stopped snoring after 3 days on Kalydeco and we haven't heard it since. Brady's sinus issues had a real negative impact on his quality of life and the side-effects of Prednisone are less than pleasant. To be completely free from this issue feels like such a miracle. It is something we used to battle on a daily basis and now the issue is GONE. We visited Brady's Ear/Nose/Throat Dr. a few weeks ago to document how well he is doing and the Dr. remarked that Brady's sinus tissues actually "look different" than before. By the end of the visit, he was asking about Vertex stock as an investment opportunity. I'd say he was pretty impressed!
Mental Outlook: Before Kalydeco, I felt like I was swirling around in the dangerous winds of that tornado. You feel so out of control and every once in a while you get flung into the side of a brick wall. Going to clinic paralyzed me with fear. What new meds are they going to add? Will I be able to get Brady to actually swallow/inhale/sit still through it? What will his throat culture show? Will his lungs be clear? CF never rests and that means that you can never rest. You have to fight CF CONSTANTLY. It is so damn exhausting and wild rides make me sick to my stomach...
Today, we still have treatments to do and Brady still takes lots of pills, but it is eerily calm right here. I can see the winds raging violently all around me...but it feels like Kalydeco has led us to the eye of the storm. Life feels oddly light-hearted and non-eventful. I have an enormous amount of appreciation for the little chunks of time we have been getting back over the last 4 months. I've replaced some of our morning treatment time with back massages (I've been trained in Massage Therapy and Brady has finally gotten wise to it). In the evenings now, we spent more time playing outside because treatment time is so short. An evolution of "normal" has taken place in our home. Kalydeco has been such a gift for all of us. I calculated that Brady has ingested $105,264 (258 pills @$408/pill) worth of Kalydeco so far. I'm so thankful that our insurance has decided that Brady is "worth it." I feel like my experiences with CF and Kalydeco have allowed me to place my priorities firmly in the correct order and live my life accordingly. And while I greatly appreciate the calm that the eye of the storm provides...I think we would all prefer that the storm would just GO AWAY ALEADY!
Sunday, May 20, 2012
Response Guided Therapy
I haven't written much lately for several reasons:
1) The "On Par for a Cure" golf tournament for the CFF was May 11th, and was all consuming,
2) Nothing has changed with Brady, he is still holding steady at a level I would call "AWESOME," and I assume that people aren't super interested unless I have something important to say.
3) My head has been a mess. I want to preface this whole entry by saying: I am not a CF expert. I am a stay-at-home mom of a 4 yr. old son with CF. I happen to have a background in Chemistry and have learned a lot on my own about CFTR science. This is all my opinion.
Things are going great for Brady on Kalydeco. What could I possibly have to worry about?
In some ways I am extremely happy. I am on top of the world that Brady has Kalydeco and is doing as well as we had hoped, so why am I still laying awake at night? Why do I feel so fortunate, yet so tortured at the same time? I'm not going to dance around it and try to be politically correct, I'm just going to say it--THERE IS A MUCH LARGER POPULATION OF CFers THAT COULD BENEFIT FROM KALYDECO, BUT DON'T HAVE ACCESS TO IT. Any CF patient who successfully transports any quantity of CFTR to their cell surface would respond to treatment with Kalydeco to some extent. Researchers already know that this group includes:
*ALL gating mutations,
*Most Conductance mutations,
*Some splice mutations.
The fact that the group of Kalydeco responders might soon be expanding is fantastic news! I talk about mutation classes and what that means for CFTR transport in this blog: http://luckycfmom.blogspot.com/2011/11/mutation-matters.html Yet, all of us are painfully aware that Kalydeco has only been approved for G551D ages 6+ and it is extremely difficult or next to impossible to get insurance coverage for this super expensive drug "off-label." Furthermore, there is a growing body of evidence showing that in some cases, Kalydeco has been beneficial for patients who shouldn't theoretically show any response to the drug such as:
1)Heterozygous patients with class 1 and 2 mutations
Theoretically, From what we know about these mutations individually, there should be no CFTR on the cell surface and these patients shouldn't show a response to Kalydeco.
In reality, I know of several cases of patients obtaining a script and coverage for Kalydeco "off-label," trying it, and showing reduction in sweat chloride and improvement in lung function. I think some patients are having some CFTR reach the surface when their two CF mutations interact with one another through a process called Transcomplementation: http://luckycfmom.blogspot.com/2012/03/calling-all-heterozygotes.html
2)DDF508
Almost everything you read about the DDF508 response to Kalydeco is that it DOES NOT work for this group. I want to very cautiously put this out there.
Theoretically, there should be no surface CFTR to potentiate. The vast majority of patients showed no significant response.
In reality, a very small subset of DDF508 showed some response to Kalydeco in trials. We are talking about 1 in 112 patients. The scary thing is that they (researchers) have no idea how to identify one of these responders. What makes them different from any other DDF508?? Still, right on the insert in every Kalydeco bottle it states that the drug is "not effective in patients with CF who are homozygous for the F508del mutations in the CFTR gene." Is 1 in 112 patients statistically significant? No. Does that extremely rare DDF508 responder deserve the chance to find out if Kalydeco could help them? Yes.
3)Who else?
The thing is, the biochemical reactions that take place within your body are as unique to you as your fingerprint. We know that there is wide variability in the severity of CF, even between siblings or twins with identical genotype and similar environment. There are other factors at play that influence CFTR. I think Peter Mueller from Vertex was on to something amazing when he discussed idea this a few months ago.
http://iom.edu/~/media/Files/Activity%20Files/Research/GenomicBasedResearch/2012-MAR-21/3%20-%20Peter%20Mueller.pdf Take particular note of slides 31-37. Doesn't that just blow your mind? How awesome would it be to receive your own "Trial in a Box" from Vertex to track your body's response to Kalydeco and make a treatment decision based on the results?! **This is not an official trial, or registered with clinicaltrials.gov. This is an idea that is being talked about by those in the know...Vertex is definitely NOT recruiting for this right now. It is just being discussed at a preliminary level. The cooperation of the FDA and insurance industry would be of the utmost importance.
Disclaimer --I just want to clarify that I am not suggesting that Kalydeco would work in every CF patient. There are many CF patients that don't successfully transport any CFTR to the cell surface, and would not respond to treatment. The point that I want to drive home is that: NO ONE KNOWS FOR SURE IF IT WILL WORK IN AN INDIVIDUAL UNTIL IT IS TESTED IN THAT INDIVIDUAL.
Response Guided Therapy
In an ideal world, when a drug like Kalydeco is discovered and found to be safe, people would be free to decide if they want to try it, and see if they feel better, or their sweat chloride drops. If you show a response, then you would remain on the therapy. If you didn't, they would know that you have no surface CFTR and you would stop and wait for the corrector + potentiator combo moving down the pipeline. I mean, wouldn't this be revolutionary! The problem is that this "trial in a box/trial of 1" design is not something that the FDA would look at as a valid statistical measure for approval. That is why it is extremely important that the CF community continue to support the EXPERRT Act, which allows the FDA to bring in disease specialists for rare conditions like CF to best determine the endpoints for trials, etc... The CF Foundation is truly blazing the trail toward a new generation of individualized, gene based medicine. You can imagine that there are a few "norms" that we are going to have to challenge along the way. Both Vertex and the CFF are legally bound to stay out of any off-label use because they are focued on obtaining wider regulatory approval in the U.S. and of course for our friends in Europe and Australia. Waiting sucks. I'm sure that Vertex would love nothing more than to fill thousands more Kalydeco orders and the CFF would love to see its community live longer and feel better. Unfortunately, they have to play by the rules.
I can't underestimate the importance of seeing the EXPERRT Act pass and become law because it would allow us to slightly change the rules and persue more individualized approaches to treatment. The repercussions for CF patients could be phenomenal. Visit the CFF's advocacy tookit to read about the EXPERRT Act, and quickly and easily email your elected officials asking for their support. We need to educate the FDA about the new ways we are individualizing treatment for CF patients and this will allow us to be heard! Personalize your letter for the greatest impact! http://www.cff.org/GetInvolved/Advocate/AdvocacyToolkit/EXPERRTAct/
Off-label Documentation
Brady is part of the National CF Registry and we have documented in detail the benefits of Kalydeco through both office exams and lab work. Every off-label approval and documented success of Kalydeco will tilt the odds a little more in our favor for additional insurance companies to approve it for others off-label. Remember when Pulmozyme was first approved by the FDA? It was only trialed in kids age 6 and older. Of course, Dr.'s soon saw its benefits and began prescribing to younger patients. At first, the insurance industry pushed back, but after seeing it routinely prescribed and accepted by other insurance companies, everyone eventually just began covering it for younger kids also--OFF-LABEL! Even insurance companies can see that PREVENTING problems in CF is cheaper than FIXING problems once they have already occured and caused potentially permanent damage. I realize that Kalydeco is dangerously high priced. It raises an automatic red flag for insurance companies. Our insurance told us that it happens for anything costing over $10K per month in their system. They automatically required the "letter of medical necessity" from Brady's Dr. and a "pre-authorization." I live with constant fear that our insurance company will change their mind about covering Kalydeco for Brady, so we have been seeing the Dr. frequently, doing lots of lab work, and making sure there is NO DOUBT that it is working wonders for him. I order his 5th bottle on Monday! I can hardly believe it!
Back to why I can't sleep
The image from Breck's thoughtful and well-written blog just after Kalydeco was approved has never left my mind. http://bennettgamel.blogspot.com/2012/01/will-helicopters-return-in-time-for-us.html Everyone waiting...stranded on that rooftop with the floodwaters rising around them. She said it so brilliantly. This is how I feel as I see how Kalydeco has positively affected Brady, and also having the knowledge that it could be helping many more.
Then they fly away into the distance. Everyone is staring at me asking what the note says. When they will be back? What the hell?? Why did they send help specifically to you? Do you know them? I read everyone the note and they digest the news of several more years of waiting. In the meantime, I can't help but feel relieved as I feed my starving child what he needs to survive. BUT--If I look just a bit further, I'll see another child suffering and feel another Mother's heart breaking. It feels so extremely gluttonous to have access to Kalydeco while others don't. It has been such a lifechanging miracle for us. It just makes me sick to know that we live in a world where money determines whether people live or die. Because it doesn't matter how well the drug might work in you. What matters right now seems to be technicalities like: what mutation you have, how old you are, what country you live in, what type of insurance coverage you have, etc... NOOOOOOOOOOOO!!! It just doesn't feel right. I don't know how I'll ever rest.
I hope everyone writes in to their representatives in support of the EXPERRT ACT! I want to see the day when the "trial in a box" is more than an idea...it is a reality! So what do I do now? Since my family has the nourishment it needs, we can work to build shelters for the others, care for them in their weakest hours, and relentlessly keep the rescue fire burning, in the hopes of attracting a less idiotic helicopter pilot. I may not be able to share my nourishment, but I can share my energy and love. In Breck's version, my family flies away on the helicopter to safety, leaving the rest behind. In my version, and in my heart, I will never leave the CF Community. The rescue fire will never go out on my watch.
1) The "On Par for a Cure" golf tournament for the CFF was May 11th, and was all consuming,
2) Nothing has changed with Brady, he is still holding steady at a level I would call "AWESOME," and I assume that people aren't super interested unless I have something important to say.
3) My head has been a mess. I want to preface this whole entry by saying: I am not a CF expert. I am a stay-at-home mom of a 4 yr. old son with CF. I happen to have a background in Chemistry and have learned a lot on my own about CFTR science. This is all my opinion.
Things are going great for Brady on Kalydeco. What could I possibly have to worry about?
In some ways I am extremely happy. I am on top of the world that Brady has Kalydeco and is doing as well as we had hoped, so why am I still laying awake at night? Why do I feel so fortunate, yet so tortured at the same time? I'm not going to dance around it and try to be politically correct, I'm just going to say it--THERE IS A MUCH LARGER POPULATION OF CFers THAT COULD BENEFIT FROM KALYDECO, BUT DON'T HAVE ACCESS TO IT. Any CF patient who successfully transports any quantity of CFTR to their cell surface would respond to treatment with Kalydeco to some extent. Researchers already know that this group includes:
*ALL gating mutations,
*Most Conductance mutations,
*Some splice mutations.
The fact that the group of Kalydeco responders might soon be expanding is fantastic news! I talk about mutation classes and what that means for CFTR transport in this blog: http://luckycfmom.blogspot.com/2011/11/mutation-matters.html Yet, all of us are painfully aware that Kalydeco has only been approved for G551D ages 6+ and it is extremely difficult or next to impossible to get insurance coverage for this super expensive drug "off-label." Furthermore, there is a growing body of evidence showing that in some cases, Kalydeco has been beneficial for patients who shouldn't theoretically show any response to the drug such as:
1)Heterozygous patients with class 1 and 2 mutations
Theoretically, From what we know about these mutations individually, there should be no CFTR on the cell surface and these patients shouldn't show a response to Kalydeco.
In reality, I know of several cases of patients obtaining a script and coverage for Kalydeco "off-label," trying it, and showing reduction in sweat chloride and improvement in lung function. I think some patients are having some CFTR reach the surface when their two CF mutations interact with one another through a process called Transcomplementation: http://luckycfmom.blogspot.com/2012/03/calling-all-heterozygotes.html
2)DDF508
Almost everything you read about the DDF508 response to Kalydeco is that it DOES NOT work for this group. I want to very cautiously put this out there.
Theoretically, there should be no surface CFTR to potentiate. The vast majority of patients showed no significant response.
In reality, a very small subset of DDF508 showed some response to Kalydeco in trials. We are talking about 1 in 112 patients. The scary thing is that they (researchers) have no idea how to identify one of these responders. What makes them different from any other DDF508?? Still, right on the insert in every Kalydeco bottle it states that the drug is "not effective in patients with CF who are homozygous for the F508del mutations in the CFTR gene." Is 1 in 112 patients statistically significant? No. Does that extremely rare DDF508 responder deserve the chance to find out if Kalydeco could help them? Yes.
3)Who else?
The thing is, the biochemical reactions that take place within your body are as unique to you as your fingerprint. We know that there is wide variability in the severity of CF, even between siblings or twins with identical genotype and similar environment. There are other factors at play that influence CFTR. I think Peter Mueller from Vertex was on to something amazing when he discussed idea this a few months ago.
http://iom.edu/~/media/Files/Activity%20Files/Research/GenomicBasedResearch/2012-MAR-21/3%20-%20Peter%20Mueller.pdf Take particular note of slides 31-37. Doesn't that just blow your mind? How awesome would it be to receive your own "Trial in a Box" from Vertex to track your body's response to Kalydeco and make a treatment decision based on the results?! **This is not an official trial, or registered with clinicaltrials.gov. This is an idea that is being talked about by those in the know...Vertex is definitely NOT recruiting for this right now. It is just being discussed at a preliminary level. The cooperation of the FDA and insurance industry would be of the utmost importance.
Disclaimer --I just want to clarify that I am not suggesting that Kalydeco would work in every CF patient. There are many CF patients that don't successfully transport any CFTR to the cell surface, and would not respond to treatment. The point that I want to drive home is that: NO ONE KNOWS FOR SURE IF IT WILL WORK IN AN INDIVIDUAL UNTIL IT IS TESTED IN THAT INDIVIDUAL.
Response Guided Therapy
In an ideal world, when a drug like Kalydeco is discovered and found to be safe, people would be free to decide if they want to try it, and see if they feel better, or their sweat chloride drops. If you show a response, then you would remain on the therapy. If you didn't, they would know that you have no surface CFTR and you would stop and wait for the corrector + potentiator combo moving down the pipeline. I mean, wouldn't this be revolutionary! The problem is that this "trial in a box/trial of 1" design is not something that the FDA would look at as a valid statistical measure for approval. That is why it is extremely important that the CF community continue to support the EXPERRT Act, which allows the FDA to bring in disease specialists for rare conditions like CF to best determine the endpoints for trials, etc... The CF Foundation is truly blazing the trail toward a new generation of individualized, gene based medicine. You can imagine that there are a few "norms" that we are going to have to challenge along the way. Both Vertex and the CFF are legally bound to stay out of any off-label use because they are focued on obtaining wider regulatory approval in the U.S. and of course for our friends in Europe and Australia. Waiting sucks. I'm sure that Vertex would love nothing more than to fill thousands more Kalydeco orders and the CFF would love to see its community live longer and feel better. Unfortunately, they have to play by the rules.
I can't underestimate the importance of seeing the EXPERRT Act pass and become law because it would allow us to slightly change the rules and persue more individualized approaches to treatment. The repercussions for CF patients could be phenomenal. Visit the CFF's advocacy tookit to read about the EXPERRT Act, and quickly and easily email your elected officials asking for their support. We need to educate the FDA about the new ways we are individualizing treatment for CF patients and this will allow us to be heard! Personalize your letter for the greatest impact! http://www.cff.org/GetInvolved/Advocate/AdvocacyToolkit/EXPERRTAct/
Off-label Documentation
Brady is part of the National CF Registry and we have documented in detail the benefits of Kalydeco through both office exams and lab work. Every off-label approval and documented success of Kalydeco will tilt the odds a little more in our favor for additional insurance companies to approve it for others off-label. Remember when Pulmozyme was first approved by the FDA? It was only trialed in kids age 6 and older. Of course, Dr.'s soon saw its benefits and began prescribing to younger patients. At first, the insurance industry pushed back, but after seeing it routinely prescribed and accepted by other insurance companies, everyone eventually just began covering it for younger kids also--OFF-LABEL! Even insurance companies can see that PREVENTING problems in CF is cheaper than FIXING problems once they have already occured and caused potentially permanent damage. I realize that Kalydeco is dangerously high priced. It raises an automatic red flag for insurance companies. Our insurance told us that it happens for anything costing over $10K per month in their system. They automatically required the "letter of medical necessity" from Brady's Dr. and a "pre-authorization." I live with constant fear that our insurance company will change their mind about covering Kalydeco for Brady, so we have been seeing the Dr. frequently, doing lots of lab work, and making sure there is NO DOUBT that it is working wonders for him. I order his 5th bottle on Monday! I can hardly believe it!
Back to why I can't sleep
The image from Breck's thoughtful and well-written blog just after Kalydeco was approved has never left my mind. http://bennettgamel.blogspot.com/2012/01/will-helicopters-return-in-time-for-us.html Everyone waiting...stranded on that rooftop with the floodwaters rising around them. She said it so brilliantly. This is how I feel as I see how Kalydeco has positively affected Brady, and also having the knowledge that it could be helping many more.
Scene Setting
The whole CF community is on a deserted island in the middle of the ocean. We have run out of food and we are all starving and ill, especially our children. We see a helicopter in the distance and all scream with hope and delight! Help arrives directly over my head and drops down a message for me,
"Here is the food you will need to sustain your family for the next 4 years. I am going back to the mainland to build a boat. I will sail here and rescue everyone in a few years. There is one condition--you cannot share any of this mountain of nourishment with anyone else on the island. You must only feed your own family. We have a videocamera on the island and if you are caught sharing, all your food will be taken back."
I hope everyone writes in to their representatives in support of the EXPERRT ACT! I want to see the day when the "trial in a box" is more than an idea...it is a reality! So what do I do now? Since my family has the nourishment it needs, we can work to build shelters for the others, care for them in their weakest hours, and relentlessly keep the rescue fire burning, in the hopes of attracting a less idiotic helicopter pilot. I may not be able to share my nourishment, but I can share my energy and love. In Breck's version, my family flies away on the helicopter to safety, leaving the rest behind. In my version, and in my heart, I will never leave the CF Community. The rescue fire will never go out on my watch.
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