The Phase 3 results for the Kalydeco/VX-809 combo are in, and the final step is submitting everything for FDA approval! For the approximate 50% of the CF population that is homozygous F508del (or those that have 2 copies of that mutation), this is HUGE news. Heck, it's pretty big news for the rest of us as well.
It's going to be a while before the heterozygous F508del studies reach this point, and possibly longer yet before VX-661 gets there, but this is exciting news! It's not a cure, and we still need to look for a cure. But this drug has the potential to dramatically improve life expectancy and quality of life in CF patients who can get access to the drug.
Personally, I'm remaining cautiously optimistic. There are a lot of good things with Kalydeco, and I'm excited to read the phase 3 study results in-depth to see the positives that came out of it. At the same time, I've also read some of the negatives that are happening, and I really want to know what the long-term effects are going to be. I'm fully aware that we won't know the long-term effects for a while, but it's still something I can't help but think about.
In any case, it's an exciting day for the CF community, and I'm hopeful that the FDA will expedite the approval process so we can get this into the hands of patients as soon as possible!
Tuesday, June 24, 2014
Friday, June 13, 2014
So About That New Vest...
I got our call back Wednesday afternoon, and it was shipped out yesterday! Talk about fast service! Between the 2 insurance plans, everything will be 100% covered, so we'll have no out of pocket payments. Insurance was waiting for the final paperwork and whatnot to give approval, so hopefully everything continues to go through smoothly.
The next step is to wait for the call from the respiratory therapist who is a rep for Respirtech so we can set up an appointment for them to come to the house and train us on the machine. Since we're already using a vest and are familiar with how it works for airway clearance, we had the option to waive the training, but I told the rep that John and I would still like to go through with it so the RT can show us the ins and outs of the new machine. I'm sure I could easily figure out how to use it by reading the instructions and whatnot, but it's just going to be easier for the RT to show us what to do since my reading time is limited these days.
We're hoping that we can get an appointment set up for the end of next week. I'm also planning to order the Ikea cart that I linked in a previous post. I'm going to bite the bullet and pay the $15 in shipping, because if the fuel cost estimates are accurate on mapquest, it's going to cost almost that much for me to drive round trip to the nearest store. The extra dollar or 2 that I would be saving by driving will go toward saving my sanity because I won't have to worry about driving on the highways around Philly, which can be pure hell.
I'm also keeping my fingers crossed that the novelty of a new vest will stick around for a bit, because Judith's been fighting me during treatment times... again. I know full moon last night has not helped with anything around here, but even once that has passed each month she's still a nightmare. At least she's been eating a little more, so I guess that compensates for the constant whining I hear for the entire half hour she's on her vest. After an hour each day of listening to that awful whining, it's no wonder I'm ready to pull my hair out some nights, and just feel like I want to fast forward to bed time.
The next step is to wait for the call from the respiratory therapist who is a rep for Respirtech so we can set up an appointment for them to come to the house and train us on the machine. Since we're already using a vest and are familiar with how it works for airway clearance, we had the option to waive the training, but I told the rep that John and I would still like to go through with it so the RT can show us the ins and outs of the new machine. I'm sure I could easily figure out how to use it by reading the instructions and whatnot, but it's just going to be easier for the RT to show us what to do since my reading time is limited these days.
We're hoping that we can get an appointment set up for the end of next week. I'm also planning to order the Ikea cart that I linked in a previous post. I'm going to bite the bullet and pay the $15 in shipping, because if the fuel cost estimates are accurate on mapquest, it's going to cost almost that much for me to drive round trip to the nearest store. The extra dollar or 2 that I would be saving by driving will go toward saving my sanity because I won't have to worry about driving on the highways around Philly, which can be pure hell.
I'm also keeping my fingers crossed that the novelty of a new vest will stick around for a bit, because Judith's been fighting me during treatment times... again. I know full moon last night has not helped with anything around here, but even once that has passed each month she's still a nightmare. At least she's been eating a little more, so I guess that compensates for the constant whining I hear for the entire half hour she's on her vest. After an hour each day of listening to that awful whining, it's no wonder I'm ready to pull my hair out some nights, and just feel like I want to fast forward to bed time.
Wednesday, June 11, 2014
Vest Machines
Side note: I really want to reply to comments, but for some reason my account isn't letting me comment on anything, including my own blog. Silly blogger. I need to figure out what's up, so please don't think I'm ignoring you! I appreciate the comments, and am definitely reading them! :)
One thing that I look forward to every few months is the new parent support group for our clinic. We all have kids ranging from newborns to age 5, and I can't begin to say how awesome it is talking to other parents, IN PERSON, who totally get it and you can exchange tips with! I enjoy connecting with other CF patients and CF parents via the internet, but there's something about the face-to-face interactions that can make a difference. It just stinks that the disease carries the risk of cross-contamination from 1 patient to another (hence the 6 ft. rule), because if things were different we'd be able to let our kids play with each other and just talk with someone who knows exactly what they're going through.
Anyway, somehow we got on the topic of vest machines, and while I know there are still plenty of people out there using the SmartVest system, I discovered I was in the minority of the group because everyone else was using the inCourage system. Everyone had nothing but positive things to say about it, which is awesome! I have to admit I was a bit jealous that they were all using the system already, because I've been getting exasperated with Electromed. After some encouragement and more positive feedback, I decided to call and leave a message with our nurse coordinator to talk about switching.
Long story short, she said that there were management changes with Electromed at some point, and she wasn't overly happy with them anymore, either. This had to have happened at some point within the last 2 years, because when we got our current vest Electromed was the company the clinic definitely preferred. I told her my concerns, and she said we would definitely get the ball rolling to get a system from Respirtech! I really think this is going to be a good move for us for many reasons:
1. The last few times I've had to call Electromed to get parts for our SmartVest, they weren't nearly as friendly and helpful as they used to be, and the last time the rep got a little snippy with me over the phone because I must not have been describing our problem adequately enough. Sorry, but I don't know how else to describe what the generator was doing other than telling you it sounded like the air from the generator was hiccuping halfway through the treatment cycle, which it never did before.
2. The SmartVest just doesn't fit Judith properly. In 2 years, we've been in the same size vest, and at first I figured it was just because she was still so small and literally just met the height requirement to fit into the SmartVest. For a while, I've been observing that no matter what I try to do to adjust the fit, something just seemed off with the way it was inflating and fitting on her. Even getting the new bladder and outer shell didn't make a difference. SmartVests only come in 8 sizes, and after talking with one of the other moms on Saturday I felt better knowing we weren't the only ones who had problems with the fit. Respirtech, on the other hand, offers 23 size options for the inCourage system, and that alone made me feel more comfortable about petitioning to get a different unit!
3. The single hose on the SmartVest is certainly a perk, and always will be. However, when that hose gives you problems with the connection, it's frustrating. Lately, the hose either seems to get stuck in the connection point of the bladder, or it pops out relatively quickly depending on how Judith moves. If I don't seal it in hard enough, out it pops, and she gets mad because she can't put it back in herself. If I get a good seal, then it gets stuck and I have a heck of a time getting it out. User error? Possibly. But the other problem is Judith acts like an ox on her vest sometimes, and will pull that dang 23 lb generator along the floor to get to something she decides she wants, and that always ends in the hose popping out as well. Could we have the same problem with the inCourage? It's possible. The big attraction for me, however, is the locking hoses. I'll gladly give up the single hose mechanism for a double hose setup when they'll lock into place, and will be easy for her to pop into the vest herself.
4. The older model SmartVest generator is huge. And loud. And weighs a ton. Ok, not a literal ton, but it's 23 lbs. I know I've complained about this before, and I even expressed my concerns about that to the customer service rep when I called with our generator issue the last time. Since they had to send us a new generator (and we had to send the old one back so they could inspect it), I asked if we could get one of their newer model generators that is much lighter and quieter. I was told that nope, they refuse to send me a new model generator, and said that maybe with a new prescription from our doctor they could switch us. I then asked if they were planning to ever phase out the huge generators and transition everyone over, and she told me no.
The inCourage system's weight is probably going to be closer to the weight of the new SmartVest system, according to what people told me. So I'd guess somewhere around 16-17 lbs. That's going to be a huge difference for me. Obviously I'm still planning to get a cart to store everything on, but for transport purposes having a lighter machine is going to be easier on everyone, including my parents.
5. The other moms were telling me about the ease of programming the inCourage system, and I'm excited about that! This vest cycles through things itself instead of you having to program and start/stop things on your own, plus the way it inflates and shakes is supposed to clear the airways better. Judith's been doing so well respiratory-wise, and I want to keep that up while her lung involvement is minimal. If we can keep clearing things efficiently now, I can hope that we can delay or minimize some of the damage the disease causes as it progresses.
We have no idea what our insurances' response is going to be to this process. I can guarantee they're going to deny payment at least the first time. They always do, especially given the costs of the equipment. I was told that because our current system is about 2 years old, and we also switched our secondary since we had the SmartVest (and they didn't have to make any payments toward the purchase), we should have a little more leverage. Our nurse coordinator is going to do everything she can to help us, as is Respirtech. I felt really good after the initial phone call with Respirtech Monday afternoon, and feel very confident that this is a good decision for us. I'm hoping that the process doesn't take too long, and we can get the new system (and trained on it) quickly.
One thing that I look forward to every few months is the new parent support group for our clinic. We all have kids ranging from newborns to age 5, and I can't begin to say how awesome it is talking to other parents, IN PERSON, who totally get it and you can exchange tips with! I enjoy connecting with other CF patients and CF parents via the internet, but there's something about the face-to-face interactions that can make a difference. It just stinks that the disease carries the risk of cross-contamination from 1 patient to another (hence the 6 ft. rule), because if things were different we'd be able to let our kids play with each other and just talk with someone who knows exactly what they're going through.
Anyway, somehow we got on the topic of vest machines, and while I know there are still plenty of people out there using the SmartVest system, I discovered I was in the minority of the group because everyone else was using the inCourage system. Everyone had nothing but positive things to say about it, which is awesome! I have to admit I was a bit jealous that they were all using the system already, because I've been getting exasperated with Electromed. After some encouragement and more positive feedback, I decided to call and leave a message with our nurse coordinator to talk about switching.
Long story short, she said that there were management changes with Electromed at some point, and she wasn't overly happy with them anymore, either. This had to have happened at some point within the last 2 years, because when we got our current vest Electromed was the company the clinic definitely preferred. I told her my concerns, and she said we would definitely get the ball rolling to get a system from Respirtech! I really think this is going to be a good move for us for many reasons:
1. The last few times I've had to call Electromed to get parts for our SmartVest, they weren't nearly as friendly and helpful as they used to be, and the last time the rep got a little snippy with me over the phone because I must not have been describing our problem adequately enough. Sorry, but I don't know how else to describe what the generator was doing other than telling you it sounded like the air from the generator was hiccuping halfway through the treatment cycle, which it never did before.
2. The SmartVest just doesn't fit Judith properly. In 2 years, we've been in the same size vest, and at first I figured it was just because she was still so small and literally just met the height requirement to fit into the SmartVest. For a while, I've been observing that no matter what I try to do to adjust the fit, something just seemed off with the way it was inflating and fitting on her. Even getting the new bladder and outer shell didn't make a difference. SmartVests only come in 8 sizes, and after talking with one of the other moms on Saturday I felt better knowing we weren't the only ones who had problems with the fit. Respirtech, on the other hand, offers 23 size options for the inCourage system, and that alone made me feel more comfortable about petitioning to get a different unit!
3. The single hose on the SmartVest is certainly a perk, and always will be. However, when that hose gives you problems with the connection, it's frustrating. Lately, the hose either seems to get stuck in the connection point of the bladder, or it pops out relatively quickly depending on how Judith moves. If I don't seal it in hard enough, out it pops, and she gets mad because she can't put it back in herself. If I get a good seal, then it gets stuck and I have a heck of a time getting it out. User error? Possibly. But the other problem is Judith acts like an ox on her vest sometimes, and will pull that dang 23 lb generator along the floor to get to something she decides she wants, and that always ends in the hose popping out as well. Could we have the same problem with the inCourage? It's possible. The big attraction for me, however, is the locking hoses. I'll gladly give up the single hose mechanism for a double hose setup when they'll lock into place, and will be easy for her to pop into the vest herself.
4. The older model SmartVest generator is huge. And loud. And weighs a ton. Ok, not a literal ton, but it's 23 lbs. I know I've complained about this before, and I even expressed my concerns about that to the customer service rep when I called with our generator issue the last time. Since they had to send us a new generator (and we had to send the old one back so they could inspect it), I asked if we could get one of their newer model generators that is much lighter and quieter. I was told that nope, they refuse to send me a new model generator, and said that maybe with a new prescription from our doctor they could switch us. I then asked if they were planning to ever phase out the huge generators and transition everyone over, and she told me no.
The inCourage system's weight is probably going to be closer to the weight of the new SmartVest system, according to what people told me. So I'd guess somewhere around 16-17 lbs. That's going to be a huge difference for me. Obviously I'm still planning to get a cart to store everything on, but for transport purposes having a lighter machine is going to be easier on everyone, including my parents.
5. The other moms were telling me about the ease of programming the inCourage system, and I'm excited about that! This vest cycles through things itself instead of you having to program and start/stop things on your own, plus the way it inflates and shakes is supposed to clear the airways better. Judith's been doing so well respiratory-wise, and I want to keep that up while her lung involvement is minimal. If we can keep clearing things efficiently now, I can hope that we can delay or minimize some of the damage the disease causes as it progresses.
We have no idea what our insurances' response is going to be to this process. I can guarantee they're going to deny payment at least the first time. They always do, especially given the costs of the equipment. I was told that because our current system is about 2 years old, and we also switched our secondary since we had the SmartVest (and they didn't have to make any payments toward the purchase), we should have a little more leverage. Our nurse coordinator is going to do everything she can to help us, as is Respirtech. I felt really good after the initial phone call with Respirtech Monday afternoon, and feel very confident that this is a good decision for us. I'm hoping that the process doesn't take too long, and we can get the new system (and trained on it) quickly.
Friday, June 6, 2014
Imaginative Play, CF Style
Somewhat recently, Judith has really taken off with her imaginative play. Some of it makes me laugh, because for our house it's a totally normal occurrence, but in other homes it would be a nutritional concern for a typical kid. For example, literally a few minutes ago, Judith was at the coffee table preparing some "food" for her Bitty Baby (appropriately named Bitty). She's standing here, ticking off the ingredients, and says, "Now we add the salt!"
Can you tell that salt is a preferred condiment around here? And she's been getting more of it lately since we've had some warmer and more humid days, so I've been getting back into the habit of making sure she's getting extra doses to replenish what she's losing when she sweats.
Just the other day, I heard her saying something about doing breathing treatments on her doll. I'm going to work on cleaning out the supply caddy we have, and getting things into baskets for better organization, so I may dig out her old, tiny spacer so she can pretend with that.
Now I'm waiting for her to find her old percussors and want to do manual CPT on her toys.
It's amazing hearing all of this, and realizing how much she's really understanding and picking up on even though we might think she's not quite getting it. At the same time, it sucks that she even knows what any of this is.
Can you tell that salt is a preferred condiment around here? And she's been getting more of it lately since we've had some warmer and more humid days, so I've been getting back into the habit of making sure she's getting extra doses to replenish what she's losing when she sweats.
Just the other day, I heard her saying something about doing breathing treatments on her doll. I'm going to work on cleaning out the supply caddy we have, and getting things into baskets for better organization, so I may dig out her old, tiny spacer so she can pretend with that.
Now I'm waiting for her to find her old percussors and want to do manual CPT on her toys.
It's amazing hearing all of this, and realizing how much she's really understanding and picking up on even though we might think she's not quite getting it. At the same time, it sucks that she even knows what any of this is.
Saturday, May 31, 2014
A Final Thought for the Month
As CF Awareness Month comes to a close, I want to offer a final thought that I feel is important for people to remember.
CF patients may not “look that sick,” but please keep in mind that a ton of work goes into keeping our children (or themselves for adults and older teens) as healthy as they possibly can be. CF is a progressive disease, and as another CF mom puts it, it’s a “365 days a year, 24 hours a day” thing, and “there’s no remission or vacation from CF.” Family, friends, loved ones, and the CF care team are in it for the patient so they can have a full life, with as many typical experiences as they can. So while we do have a month where advocacy and awareness happen more frequently, remember that for those living with the disease and for the families affected by it, awareness and advocacy also happens throughout the year and doesn't end because the calendar flips to June 1.
If you would like to read more about CF or treatments, therapies, and other aspects of the disease that I didn’t touch on throughout the month, please visit www.cff.org and click on any of the categories at the top of the page or on the home page.
CF patients may not “look that sick,” but please keep in mind that a ton of work goes into keeping our children (or themselves for adults and older teens) as healthy as they possibly can be. CF is a progressive disease, and as another CF mom puts it, it’s a “365 days a year, 24 hours a day” thing, and “there’s no remission or vacation from CF.” Family, friends, loved ones, and the CF care team are in it for the patient so they can have a full life, with as many typical experiences as they can. So while we do have a month where advocacy and awareness happen more frequently, remember that for those living with the disease and for the families affected by it, awareness and advocacy also happens throughout the year and doesn't end because the calendar flips to June 1.
If you would like to read more about CF or treatments, therapies, and other aspects of the disease that I didn’t touch on throughout the month, please visit www.cff.org and click on any of the categories at the top of the page or on the home page.
Thursday, May 29, 2014
No News Is Good News
Nah, don't panic at the title. I just meant that not hearing from me means nothing has really changed. Well, besides the fact that the house is in an elevated state of chaos, but that's to be expected when you're getting things ready to stage.
One of my bigger predicaments at the moment is trying to figure out where I'm going to safely store all of Judith's equipment and meds where they'll be safe and out of sight once the house goes on the market and we have showings/open houses. Many other CF moms have recommended this Ikea cart for storing Vest equipment, nebulizer and parts, and even meds that don't need refrigeration. I absolutely love the concept, but am still debating if I want to shell out $15 in shipping so I don't have to drive super long to get one in-store. That, and I'm not 100% sure that this cart will work with our SmartVest, since most of the people who are using them have Hill-Rom or Respritech vests. In any case, I know I need to get this vest on wheels at some point, because the generator is so darn HEAVY. And if it helps with storage, well, that's a definite bonus right now.
I did manage to clean out my closet over the weekend, and almost 6 hours later that sucker looks immaculate. I haven't gotten rid of a lot of stuff since high school, so some of it was at least 12 years old. 9 trash bags of clothes and about 4 of shoes were donated, and earlier in the week I took a huge bag to the recycling center to donate since I knew the other places wouldn't accept it due to it's condition. When he saw my closet, John actually said to me, "Is that seriously all you wear?!" I think he was shocked, because my closet was literally jammed full of stuff, and now there's barely anything in there. I seriously regret not doing that sooner, because I could've had an awesomely neat closet for the last 7 years!
Next on my list is Judith's room, and I know this is going to be a problem. Toys will be going into storage, and I'm facing reality that it's time to sell all of her smaller clothes. Certain things I'm definitely keeping, like all of her smocked and monogrammed outfits, most of her 24m/2T clothes since I can use them on my puppets if I get back into puppet ministry, and all of her preemie/NB clothes since she can use them as doll clothes (and I frankly have too much of an attachment to them to get rid of them anyway), but the rest will be sorted and either sold when we have a yard sale, or donated. I'm anticipating that Judith is going to be upset by the amount of toys that will need to be stored, but realistically she doesn't play with a lot of them right now anyway, so rotating is going to be good for her... whether she wants to acknowledge that or not!
One of my bigger predicaments at the moment is trying to figure out where I'm going to safely store all of Judith's equipment and meds where they'll be safe and out of sight once the house goes on the market and we have showings/open houses. Many other CF moms have recommended this Ikea cart for storing Vest equipment, nebulizer and parts, and even meds that don't need refrigeration. I absolutely love the concept, but am still debating if I want to shell out $15 in shipping so I don't have to drive super long to get one in-store. That, and I'm not 100% sure that this cart will work with our SmartVest, since most of the people who are using them have Hill-Rom or Respritech vests. In any case, I know I need to get this vest on wheels at some point, because the generator is so darn HEAVY. And if it helps with storage, well, that's a definite bonus right now.
I did manage to clean out my closet over the weekend, and almost 6 hours later that sucker looks immaculate. I haven't gotten rid of a lot of stuff since high school, so some of it was at least 12 years old. 9 trash bags of clothes and about 4 of shoes were donated, and earlier in the week I took a huge bag to the recycling center to donate since I knew the other places wouldn't accept it due to it's condition. When he saw my closet, John actually said to me, "Is that seriously all you wear?!" I think he was shocked, because my closet was literally jammed full of stuff, and now there's barely anything in there. I seriously regret not doing that sooner, because I could've had an awesomely neat closet for the last 7 years!
Next on my list is Judith's room, and I know this is going to be a problem. Toys will be going into storage, and I'm facing reality that it's time to sell all of her smaller clothes. Certain things I'm definitely keeping, like all of her smocked and monogrammed outfits, most of her 24m/2T clothes since I can use them on my puppets if I get back into puppet ministry, and all of her preemie/NB clothes since she can use them as doll clothes (and I frankly have too much of an attachment to them to get rid of them anyway), but the rest will be sorted and either sold when we have a yard sale, or donated. I'm anticipating that Judith is going to be upset by the amount of toys that will need to be stored, but realistically she doesn't play with a lot of them right now anyway, so rotating is going to be good for her... whether she wants to acknowledge that or not!
Kalydeco, VX-809, & VX-661
In January 2012, the CF community was buzzing over the FDA’s expedited approval of the drug that has brought us as close to a cure as we have been: Kalydeco. For the 4% of the CF population that carries a copy of the G551D mutation, the drug has been a major game changer. While it’s not the cure that we are still waiting for, the promise that it is showing in the improvement in the quality of life in patients who have been on it is remarkable!
Kalydeco, or Ivacaftor if you use the generic name, is developed by Vertex Pharmaceuticals with significant funding/help from the Cystic Fibrosis Foundation, and they are currently working through various phases of the study process for a combination of Kalydeco and VX-809 or Kalydeco and VX-661. The drug(s) help to restore CFTR function to the surface of the cell, which means that the flow of salt and other fluids to the lungs is restored, and mucus in the lungs can be thinned (cff.org). The reason that this is so revolutionary for the disease is because up until this point, treatments only targeted and treated the symptoms. Kalydeco is the first drug to actually target and treat the underlying cause of CF.
Currently, Kalydeco is approved for patients with a copy of G551D who are age 6 and older. Studies are underway for patients with a copy of G551D who are ages 2-6, and it is hoped that approval for that age group will happen soon.
VX-809 and VX-661 are not approved by the FDA yet, as they are still going through various phases of the study process. Phase 3 studies are currently underway for the Kalydeco/VX-809 combination for patients who are homozygous F508del (or those who have 2 copies of that mutation), and Phase 2 studies are underway for heterozygous F508del patients (or those that have 1 copy of that mutation and 1 copy of another mutation). It is expected that approval for the homozygous F508del patients will happen in the next few years, and there is hope that the heterozygous F508del population will not take too much longer to get approval. Last time I checked on VX-661, I think I remember reading that it was heading to Phase 2 trials, and was showing more promise than VX-809. It would target the same populations as VX-809.
The reason VX-809/VX-661 has so many people on edge waiting for the studies to finish and approval to happen is because these drugs target a huge portion of the CF population; approximately 90% or more of the CF population carries at least 1 copy of F508del. Once VX-809/VX-661 are approved for both the homozygous and heterozygous population, a vast majority of CF patients will hopefully see a dramatic improvement, and have a chance at living a longer, healthier life than they would have without these drugs.
In the first paragraph, I mentioned that the CFF provided a lot of support in various ways through funding, which includes a $75 million investment for Kalydeco alone (cff.org). This is why so many of us reach out and ask for donations and support for the Great Strides walks! Because CF is an orphan disease, government funding is practically, if not totally, nonexistent. Our fundraising efforts as families, friends, and others who care about the disease, is what is funding these studies for our loved ones. It’s why we’re moving closer to a cure! $.90 of every $1 raised goes back to the CFF, which in turn goes into the research for treatments like Kalydeco.
The drug doesn’t come without a hefty price tag: 1 year of Kalydeco can cost approximately $300,000. I’m hearing reports from other CF parents in the US that they are not having to pay a lot out of pocket for the drug and their copays aren’t too bad, but the extremely high costs is a reason why other countries around the world have not yet approved Kalydeco for their patients. In Australia, for example, they are still fighting the government to approve Kalydeco for patients with a copy of G551D. My fear is that once VX-809/VX-661 gain FDA approval, patients in countries that are struggling to get Kalydeco are going to be going through additional battles to get the new combination drugs approved, even though a huge portion of the CF population would benefit.
Source: http://www.cff.org/treatments/Therapies/Kalydeco/
Kalydeco, or Ivacaftor if you use the generic name, is developed by Vertex Pharmaceuticals with significant funding/help from the Cystic Fibrosis Foundation, and they are currently working through various phases of the study process for a combination of Kalydeco and VX-809 or Kalydeco and VX-661. The drug(s) help to restore CFTR function to the surface of the cell, which means that the flow of salt and other fluids to the lungs is restored, and mucus in the lungs can be thinned (cff.org). The reason that this is so revolutionary for the disease is because up until this point, treatments only targeted and treated the symptoms. Kalydeco is the first drug to actually target and treat the underlying cause of CF.
Currently, Kalydeco is approved for patients with a copy of G551D who are age 6 and older. Studies are underway for patients with a copy of G551D who are ages 2-6, and it is hoped that approval for that age group will happen soon.
VX-809 and VX-661 are not approved by the FDA yet, as they are still going through various phases of the study process. Phase 3 studies are currently underway for the Kalydeco/VX-809 combination for patients who are homozygous F508del (or those who have 2 copies of that mutation), and Phase 2 studies are underway for heterozygous F508del patients (or those that have 1 copy of that mutation and 1 copy of another mutation). It is expected that approval for the homozygous F508del patients will happen in the next few years, and there is hope that the heterozygous F508del population will not take too much longer to get approval. Last time I checked on VX-661, I think I remember reading that it was heading to Phase 2 trials, and was showing more promise than VX-809. It would target the same populations as VX-809.
The reason VX-809/VX-661 has so many people on edge waiting for the studies to finish and approval to happen is because these drugs target a huge portion of the CF population; approximately 90% or more of the CF population carries at least 1 copy of F508del. Once VX-809/VX-661 are approved for both the homozygous and heterozygous population, a vast majority of CF patients will hopefully see a dramatic improvement, and have a chance at living a longer, healthier life than they would have without these drugs.
In the first paragraph, I mentioned that the CFF provided a lot of support in various ways through funding, which includes a $75 million investment for Kalydeco alone (cff.org). This is why so many of us reach out and ask for donations and support for the Great Strides walks! Because CF is an orphan disease, government funding is practically, if not totally, nonexistent. Our fundraising efforts as families, friends, and others who care about the disease, is what is funding these studies for our loved ones. It’s why we’re moving closer to a cure! $.90 of every $1 raised goes back to the CFF, which in turn goes into the research for treatments like Kalydeco.
The drug doesn’t come without a hefty price tag: 1 year of Kalydeco can cost approximately $300,000. I’m hearing reports from other CF parents in the US that they are not having to pay a lot out of pocket for the drug and their copays aren’t too bad, but the extremely high costs is a reason why other countries around the world have not yet approved Kalydeco for their patients. In Australia, for example, they are still fighting the government to approve Kalydeco for patients with a copy of G551D. My fear is that once VX-809/VX-661 gain FDA approval, patients in countries that are struggling to get Kalydeco are going to be going through additional battles to get the new combination drugs approved, even though a huge portion of the CF population would benefit.
Source: http://www.cff.org/treatments/Therapies/Kalydeco/
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