What will VX-809 do for me?

jmiller · Dec 9, 2009

Hi Alexander,

(Disclaimer: I am a meteorologist, not a doctor <img src="i/expressions/face-icon-small-smile.gif" border="0">)

VX-809 and VX-770 address different problems within the cell. VX-770 aims to restore the chloride transport across the cell membrane and VX-809 gets the protein up to the cell surface. So if you have two DF508s, you may see improvement with VX-770 alone and even more improvement (in theory) with both drugs together. VX-770 is closer to the market than VX-809 at this point (in Phase 3 trials) so we will get to try it out first- but there are plans to evaluate the drugs in combination very soon. Luckily, both drugs have orphan status and should be pushed to the market as quickly as possible (barring any problems during the trials)

As far as permanant damage goes, I have heard reports that during some of the initial VX-770 trials, doctors were very encouraged to see individual's numbers climb to values they never thought they would see again. I am confident that we have resilient lungs and that if the underlying problem was fixed our bodies can do a lot more than we give them credit for.

I guess we will just have to wait and see!!!

jmiller · Dec 9, 2009

Hi Alexander,

(Disclaimer: I am a meteorologist, not a doctor <img src="i/expressions/face-icon-small-smile.gif" border="0">)

VX-809 and VX-770 address different problems within the cell. VX-770 aims to restore the chloride transport across the cell membrane and VX-809 gets the protein up to the cell surface. So if you have two DF508s, you may see improvement with VX-770 alone and even more improvement (in theory) with both drugs together. VX-770 is closer to the market than VX-809 at this point (in Phase 3 trials) so we will get to try it out first- but there are plans to evaluate the drugs in combination very soon. Luckily, both drugs have orphan status and should be pushed to the market as quickly as possible (barring any problems during the trials)

As far as permanant damage goes, I have heard reports that during some of the initial VX-770 trials, doctors were very encouraged to see individual's numbers climb to values they never thought they would see again. I am confident that we have resilient lungs and that if the underlying problem was fixed our bodies can do a lot more than we give them credit for.

I guess we will just have to wait and see!!!

jmiller · Dec 9, 2009

Hi Alexander,

(Disclaimer: I am a meteorologist, not a doctor <img src="i/expressions/face-icon-small-smile.gif" border="0">)

VX-809 and VX-770 address different problems within the cell. VX-770 aims to restore the chloride transport across the cell membrane and VX-809 gets the protein up to the cell surface. So if you have two DF508s, you may see improvement with VX-770 alone and even more improvement (in theory) with both drugs together. VX-770 is closer to the market than VX-809 at this point (in Phase 3 trials) so we will get to try it out first- but there are plans to evaluate the drugs in combination very soon. Luckily, both drugs have orphan status and should be pushed to the market as quickly as possible (barring any problems during the trials)

As far as permanant damage goes, I have heard reports that during some of the initial VX-770 trials, doctors were very encouraged to see individual's numbers climb to values they never thought they would see again. I am confident that we have resilient lungs and that if the underlying problem was fixed our bodies can do a lot more than we give them credit for.

I guess we will just have to wait and see!!!

jmiller · Dec 9, 2009

Hi Alexander,

(Disclaimer: I am a meteorologist, not a doctor <img src="i/expressions/face-icon-small-smile.gif" border="0">)

VX-809 and VX-770 address different problems within the cell. VX-770 aims to restore the chloride transport across the cell membrane and VX-809 gets the protein up to the cell surface. So if you have two DF508s, you may see improvement with VX-770 alone and even more improvement (in theory) with both drugs together. VX-770 is closer to the market than VX-809 at this point (in Phase 3 trials) so we will get to try it out first- but there are plans to evaluate the drugs in combination very soon. Luckily, both drugs have orphan status and should be pushed to the market as quickly as possible (barring any problems during the trials)

As far as permanant damage goes, I have heard reports that during some of the initial VX-770 trials, doctors were very encouraged to see individual's numbers climb to values they never thought they would see again. I am confident that we have resilient lungs and that if the underlying problem was fixed our bodies can do a lot more than we give them credit for.

I guess we will just have to wait and see!!!

jmiller · Dec 9, 2009

Hi Alexander,
 
 (Disclaimer: I am a meteorologist, not a doctor <img src="i/expressions/face-icon-small-smile.gif" border="0">)
 
 VX-809 and VX-770 address different problems within the cell. VX-770 aims to restore the chloride transport across the cell membrane and VX-809 gets the protein up to the cell surface. So if you have two DF508s, you may see improvement with VX-770 alone and even more improvement (in theory) with both drugs together. VX-770 is closer to the market than VX-809 at this point (in Phase 3 trials) so we will get to try it out first- but there are plans to evaluate the drugs in combination very soon. Luckily, both drugs have orphan status and should be pushed to the market as quickly as possible (barring any problems during the trials)
 
 As far as permanant damage goes, I have heard reports that during some of the initial VX-770 trials, doctors were very encouraged to see individual's numbers climb to values they never thought they would see again. I am confident that we have resilient lungs and that if the underlying problem was fixed our bodies can do a lot more than we give them credit for.
 
 I guess we will just have to wait and see!!!

crickit715 · Dec 9, 2009

i thought the vx770 was mainly for the g551d mutation??? i could be wrong but there was a previous post kind of on the same topic dated 11.28.09. ++

crickit715 · Dec 9, 2009

i thought the vx770 was mainly for the g551d mutation??? i could be wrong but there was a previous post kind of on the same topic dated 11.28.09. ++

crickit715 · Dec 9, 2009

i thought the vx770 was mainly for the g551d mutation??? i could be wrong but there was a previous post kind of on the same topic dated 11.28.09. ++

crickit715 · Dec 9, 2009

i thought the vx770 was mainly for the g551d mutation??? i could be wrong but there was a previous post kind of on the same topic dated 11.28.09. ++

crickit715 · Dec 9, 2009

i thought the vx770 was mainly for the g551d mutation??? i could be wrong but there was a previous post kind of on the same topic dated 11.28.09. ++

NYCLawGirl · Dec 9, 2009

770 was developed for the g551D mutation b/c that's what it corrects -- in G551D the protein gets to the surface but can't be transported. In DF508 the protein never makes it to the surface of the cell (or at least not all of it). So I think what JMiller is saying is that using both drugs in tandem (the 809 to get the protein to the surface of the cell and the 770 to transport it) might have the best results for DF508'ers. They haven't tested this yet, but it's definitely a theory that has a lot of doctors excited!

These drugs can't get rid of scarring that is already in the lungs, so they can't reverse permanent damage. On the other hand, not all drops in PFTs are due to scarring. So it doesn't surprise me that patients climbed even higher than expected -- when the mucus thins out and the constant infections go away, the body is going to heal itself as best it can. And all that means reduced inflammation, more open airways, etc. Even if it can't get rid of the scarring itself, that doesn't mean your baseline won't climb at least a little.

NYCLawGirl · Dec 9, 2009

770 was developed for the g551D mutation b/c that's what it corrects -- in G551D the protein gets to the surface but can't be transported. In DF508 the protein never makes it to the surface of the cell (or at least not all of it). So I think what JMiller is saying is that using both drugs in tandem (the 809 to get the protein to the surface of the cell and the 770 to transport it) might have the best results for DF508'ers. They haven't tested this yet, but it's definitely a theory that has a lot of doctors excited!

These drugs can't get rid of scarring that is already in the lungs, so they can't reverse permanent damage. On the other hand, not all drops in PFTs are due to scarring. So it doesn't surprise me that patients climbed even higher than expected -- when the mucus thins out and the constant infections go away, the body is going to heal itself as best it can. And all that means reduced inflammation, more open airways, etc. Even if it can't get rid of the scarring itself, that doesn't mean your baseline won't climb at least a little.

NYCLawGirl · Dec 9, 2009

770 was developed for the g551D mutation b/c that's what it corrects -- in G551D the protein gets to the surface but can't be transported. In DF508 the protein never makes it to the surface of the cell (or at least not all of it). So I think what JMiller is saying is that using both drugs in tandem (the 809 to get the protein to the surface of the cell and the 770 to transport it) might have the best results for DF508'ers. They haven't tested this yet, but it's definitely a theory that has a lot of doctors excited!

These drugs can't get rid of scarring that is already in the lungs, so they can't reverse permanent damage. On the other hand, not all drops in PFTs are due to scarring. So it doesn't surprise me that patients climbed even higher than expected -- when the mucus thins out and the constant infections go away, the body is going to heal itself as best it can. And all that means reduced inflammation, more open airways, etc. Even if it can't get rid of the scarring itself, that doesn't mean your baseline won't climb at least a little.

NYCLawGirl · Dec 9, 2009

770 was developed for the g551D mutation b/c that's what it corrects -- in G551D the protein gets to the surface but can't be transported. In DF508 the protein never makes it to the surface of the cell (or at least not all of it). So I think what JMiller is saying is that using both drugs in tandem (the 809 to get the protein to the surface of the cell and the 770 to transport it) might have the best results for DF508'ers. They haven't tested this yet, but it's definitely a theory that has a lot of doctors excited!

These drugs can't get rid of scarring that is already in the lungs, so they can't reverse permanent damage. On the other hand, not all drops in PFTs are due to scarring. So it doesn't surprise me that patients climbed even higher than expected -- when the mucus thins out and the constant infections go away, the body is going to heal itself as best it can. And all that means reduced inflammation, more open airways, etc. Even if it can't get rid of the scarring itself, that doesn't mean your baseline won't climb at least a little.

NYCLawGirl · Dec 9, 2009

770 was developed for the g551D mutation b/c that's what it corrects -- in G551D the protein gets to the surface but can't be transported. In DF508 the protein never makes it to the surface of the cell (or at least not all of it). So I think what JMiller is saying is that using both drugs in tandem (the 809 to get the protein to the surface of the cell and the 770 to transport it) might have the best results for DF508'ers. They haven't tested this yet, but it's definitely a theory that has a lot of doctors excited!
 
 These drugs can't get rid of scarring that is already in the lungs, so they can't reverse permanent damage. On the other hand, not all drops in PFTs are due to scarring. So it doesn't surprise me that patients climbed even higher than expected -- when the mucus thins out and the constant infections go away, the body is going to heal itself as best it can. And all that means reduced inflammation, more open airways, etc. Even if it can't get rid of the scarring itself, that doesn't mean your baseline won't climb at least a little.

marcijo · Dec 9, 2009