Kalydeco/lumacaftor for DDF508 results are in.

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Aboveallislove

Super Moderator
Vertex is completing the same study with 809 and kalydeco one those with one copy. The FDA required it so it knew how to do labeling. Unfortunately, vertex does not expect it to work well enough for those with one copy. But vertex has another drug that should be in clinic by end of year or early next year which they intend to combine with 661 and kalydeco to help those with only one copy. And they are doing 661 with those wit one copy, but that is not quite inderwAy yet is my understanding.
 
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TreasureGoddess

Member
ok, I don't mean to be dense, but is this one of the studies with kalydeco combined with something else or is this a study of two entirely different med combos?
 
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politicaljules

New member
I heard there was a statistically significant number of patients who experienced liver enzymes increased up to 3 times the normal level.

Has anyone heard what the results were of the sweat chloride testing? Since this drug combo was supposed to correct the CFTR transport of sodium at the cellular level, this would be a better indicator of positive results. I would like to review the actual data to verify the claims of the press release.

Mostly because I am not pleased with only a minimal increase in lung function. It was not even noticeable for the patients taking the drug. I do not think the result means that each year you get a 2% increase on top of what you got the year before, but more long term studies I guess will show that.

Pulmozyme with an increase in lung function of 10% and Hypersal with an increase in lung function of 12% are much more of a miracle cure than what I see with the results of this drug combo. And those drug makers are not charging what Vertex wants to charge.
 
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bkc3

New member
politicaljules said:
I heard there was a statistically significant number of patients who experienced liver enzymes increased up to 3 times the normal level.

Has anyone heard what the results were of the sweat chloride testing? Since this drug combo was supposed to correct the CFTR transport of sodium at the cellular level, this would be a better indicator of positive results. I would like to review the actual data to verify the claims of the press release.

Mostly because I am not pleased with only a minimal increase in lung function. It was not even noticeable for the patients taking the drug. I do not think the result means that each year you get a 2% increase on top of what you got the year before, but more long term studies I guess will show that.

Pulmozyme with an increase in lung function of 10% and Hypersal with an increase in lung function of 12% are much more of a miracle cure than what I see with the results of this drug combo. And those drug makers are not charging what Vertex wants to charge.
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i am not sure how to fully interpret the data. The most important elements as I see them are difficult to measure. Will efficacy of other treatments increase as a result of the vertex drugs mechanism? Will patients live longer healthier lives as a result of reduce exacserbations? Will the results improve after a year or more of use? Will the environment in the lungs change such that some bugs can be erradicated?
an increase that sustains beats a year over year decline even if small. Time will tell. This has been more than 20 years in the making and I cant help but feel that more treatments will follow. Will early start of this regimen prevent colonizations? So much uncertainty, but the thought of the possibility makes me so happy for all of the people that stand to benefit.
 
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Aboveallislove

Super Moderator
My recollection was it was 7 patients out of all the treatment arms with elevated liver enzymes and that 6 of 7 returned to normal on discontinuance and 7th decreased substantially. That likely means checking liver enzymes is key.
Pulmozyme, though, does not have a 10% increase. It is actually very comparable to the results for the combo. http://www.drugs.com/pro/pulmozyme.html. (The FEV improvement shown for Pulmozyme for long term is 5.6/5.8 and is for the relative improvement which is relative for the four arms was 4.0, 4.4, 5.3, 6.4). Pulmozyme reduced need for antibiotics 27/29%, while the combo reduced the need by 45/56%!!!! And reduced hospitalizations by 39-61%. The combo also impacts the whole body, gi track, etc. so will help with weight (as shown). And it is a pill. While the combo is definitely not as good as Kalydeco is for those with g551, in my view the combo is better than Pulmozyme because it has similar FEV improvement (higher in one arm), and better reduction in need for antibiotic. And it is a pill so has minimal treatment burden, as well as improving entire body. It does not substantially decrease SC--Phase 2 I think showed only 10 points, but Pulmozyme doesn't reduce SC either. We aren't there yet, but the combo is one small step to get us there and keep CFers healthy during the process. And it is proof that we can get there.
 
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pipersmom

New member
While I love the thought of fewer exacerbations, I'm not sure the weight gain is from the actual drug. Aren't people taking it required to take it with a meal or snack with a high amount of fat? I was attributing the weight gain more to that, they're probably adding a significant increase of fat/calories to their diet just taking the combo twice daily. I haven't heard of anyone bring able to reduce enzyme use, and have actually seen complaints of increased bowel movements. While it may be a small price to pay, I wouldn't call that an improvement. Maybe someone here has more info?
 
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CFParent2

New member
politicaljules said:
I heard there was a statistically significant number of patients who experienced liver enzymes increased up to 3 times the normal level.

Has anyone heard what the results were of the sweat chloride testing? Since this drug combo was supposed to correct the CFTR transport of sodium at the cellular level, this would be a better indicator of positive results. I would like to review the actual data to verify the claims of the press release.

Mostly because I am not pleased with only a minimal increase in lung function. It was not even noticeable for the patients taking the drug. I do not think the result means that each year you get a 2% increase on top of what you got the year before, but more long term studies I guess will show that.

Pulmozyme with an increase in lung function of 10% and Hypersal with an increase in lung function of 12% are much more of a miracle cure than what I see with the results of this drug combo. And those drug makers are not charging what Vertex wants to charge.
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FEV1 although is an important measure does not tell the full story. If looking young was an important measure, Pulmozyne would be comparable to applying make-up. Kayledeco/Lumacator would be like slowing down the ageing process. Yes they both do the same thing, but one addresses the root cause, the other just addresses one of the end results in a temporarily. BTW, saline solution was shown to do about the same thing as Pulmozyme and it is free.
 
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occupyjapan

New member
This is a much more important drug (for those who have mutations it works with) than pulmozyme. This may "only" raise FEV1 by a few points, but it seems to hold it there longterm, rather than with pulmozyme or an antibiotic: a short term increase in FEV1 that, over months or years, starts to drift back down. This may not immediately fix your lung problems, but it will improve them and keep you stable while even more new drugs (661 for example) come on the market.

Pulmozyme breaks up mucous making it easier to cough up. Lumacaftor (when paired with kalydeco) actually corrects the problem we have and makes CFTR proteins operate at up to 25% (as opposed to 40% for G551D patients on Kalydeco); VX661, for what it's worth, is supposed to make CFTR operate at ~60%. 661 is only 18 months behind Lumacaftor. This will keep us stable and drastically increase our quality of life until it comes out. 18 months can mean the difference literally between life and death for us. 661, for us with DF508, is shaping up to be "our Kalydeco" as far as the miracle stories go. Lumacaftor is an extremely important stopgap.

Finally, keep in mind that these numbers are the average. I have one friend who is on the combo right now that has seen a 10% relative improvement in lung function and have heard anecdotal evidence of another who had a 6-8% increase. Both have reported feeling MUCH, MUCH better in general: they have put on weight, can actually exercise, sleep well and don't wake up exhausted, their resting heart rate and oxygen% are both much better (one went from a resting heart rate of ~100 to a resting heart rate of ~70).
 
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Alexander

New member
occupyjapan said:
This is a much more important drug (for those who have mutations it works with) than pulmozyme. This may "only" raise FEV1 by a few points, but it seems to hold it there longterm, rather than with pulmozyme or an antibiotic: a short term increase in FEV1 that, over months or years, starts to drift back down. This may not immediately fix your lung problems, but it will improve them and keep you stable while even more new drugs (661 for example) come on the market.

Pulmozyme breaks up mucous making it easier to cough up. Lumacaftor (when paired with kalydeco) actually corrects the problem we have and makes CFTR proteins operate at up to 25% (as opposed to 40% for G551D patients on Kalydeco); VX661, for what it's worth, is supposed to make CFTR operate at ~60%. 661 is only 18 months behind Lumacaftor. This will keep us stable and drastically increase our quality of life until it comes out. 18 months can mean the difference literally between life and death for us. 661, for us with DF508, is shaping up to be "our Kalydeco" as far as the miracle stories go. Lumacaftor is an extremely important stopgap.

Finally, keep in mind that these numbers are the average. I have one friend who is on the combo right now that has seen a 10% relative improvement in lung function and have heard anecdotal evidence of another who had a 6-8% increase. Both have reported feeling MUCH, MUCH better in general: they have put on weight, can actually exercise, sleep well and don't wake up exhausted, their resting heart rate and oxygen% are both much better (one went from a resting heart rate of ~100 to a resting heart rate of ~70).
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Its stories like these that make me tear up out of joy. Please, keep the reports from the patients coming. I am more than curious to find out more about people's experiences.
Good times are coming for us!
 
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