MiahsMommy
New member
Hi,, I was reading some of the post and from what I read is that DDF508 patients will not benefit from the new meds that's going thru trail? Is this correct?
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DDF508
- Thread starter MiahsMommy
- Start date
MiahsMommy
New member
Hi,, I was reading some of the post and from what I read is that DDF508 patients will not benefit from the new meds that's going thru trail? Is this correct?
MiahsMommy
New member
Thanks for the response. This gives me a sigh of relief and hope <img src="i/expressions/face-icon-small-smile.gif" border="0">
MiahsMommy
New member
Thanks for the response. This gives me a sigh of relief and hope <img src="i/expressions/face-icon-small-smile.gif" border="0">
S
SarahProcter
Guest
I think "expected" is perhaps a little strong; it is certainly hoped.
S
SarahProcter
Guest
I think "expected" is perhaps a little strong; it is certainly hoped.
Kalydeco (VX-770) is working wonderfully on those with the G551D mutation, not delta508. VX-809 for delta508 isn't proving to be as dramatic and they are working on a 770/809 combo to see if they'll achieve better results. There is another drug in the pipeline for delta508 (VX-661)...this tells me they're still looking for "the" treatment for delta508.
Kalydeco (VX-770) is working wonderfully on those with the G551D mutation, not delta508. VX-809 for delta508 isn't proving to be as dramatic and they are working on a 770/809 combo to see if they'll achieve better results. There is another drug in the pipeline for delta508 (VX-661)...this tells me they're still looking for "the" treatment for delta508.
I don't pretend to be an expert on this subject.
The G551D mutation sits on the top of the gene. Kalydeco (VX770) has the ability to go direct to that mutation and "activate"it. Is doing this with good results. The Delta F508 mutation lays at the bottom of (lower on) the gene and Kalydeco cannot access it in its present location. Still under study is VX809. It is expected/hoped that VX809 will locate the Delta F508 mutation and move/relocate it to the top of the gene (like G551D). At that time Kalydeco would be introduced to the Delta F508 and "activate" it as it would activate G551D.
You can go to CFF.ORG/drugs in pipeline for info on this subject. You will find that the CFF is currently researching many avenues. Most of wich we don't know anything about.
There is even a study for people like me (OLD PEOPLE with CF and limited lung damage). The theory is that we have an natural immunity that protects our lungs from "CF damage. If this "immunity" can be identified and duplicated, it may be produced to protect other CF patients.
Bill
The G551D mutation sits on the top of the gene. Kalydeco (VX770) has the ability to go direct to that mutation and "activate"it. Is doing this with good results. The Delta F508 mutation lays at the bottom of (lower on) the gene and Kalydeco cannot access it in its present location. Still under study is VX809. It is expected/hoped that VX809 will locate the Delta F508 mutation and move/relocate it to the top of the gene (like G551D). At that time Kalydeco would be introduced to the Delta F508 and "activate" it as it would activate G551D.
You can go to CFF.ORG/drugs in pipeline for info on this subject. You will find that the CFF is currently researching many avenues. Most of wich we don't know anything about.
There is even a study for people like me (OLD PEOPLE with CF and limited lung damage). The theory is that we have an natural immunity that protects our lungs from "CF damage. If this "immunity" can be identified and duplicated, it may be produced to protect other CF patients.
Bill
I don't pretend to be an expert on this subject.
The G551D mutation sits on the top of the gene. Kalydeco (VX770) has the ability to go direct to that mutation and "activate"it. Is doing this with good results. The Delta F508 mutation lays at the bottom of (lower on) the gene and Kalydeco cannot access it in its present location. Still under study is VX809. It is expected/hoped that VX809 will locate the Delta F508 mutation and move/relocate it to the top of the gene (like G551D). At that time Kalydeco would be introduced to the Delta F508 and "activate" it as it would activate G551D.
You can go to CFF.ORG/drugs in pipeline for info on this subject. You will find that the CFF is currently researching many avenues. Most of wich we don't know anything about.
There is even a study for people like me (OLD PEOPLE with CF and limited lung damage). The theory is that we have an natural immunity that protects our lungs from "CF damage. If this "immunity" can be identified and duplicated, it may be produced to protect other CF patients.
Bill
The G551D mutation sits on the top of the gene. Kalydeco (VX770) has the ability to go direct to that mutation and "activate"it. Is doing this with good results. The Delta F508 mutation lays at the bottom of (lower on) the gene and Kalydeco cannot access it in its present location. Still under study is VX809. It is expected/hoped that VX809 will locate the Delta F508 mutation and move/relocate it to the top of the gene (like G551D). At that time Kalydeco would be introduced to the Delta F508 and "activate" it as it would activate G551D.
You can go to CFF.ORG/drugs in pipeline for info on this subject. You will find that the CFF is currently researching many avenues. Most of wich we don't know anything about.
There is even a study for people like me (OLD PEOPLE with CF and limited lung damage). The theory is that we have an natural immunity that protects our lungs from "CF damage. If this "immunity" can be identified and duplicated, it may be produced to protect other CF patients.
Bill
Bill, I am really interested in that study! I know they have identified some modifier genes that they feel impact the disease course for some with CF. I'd very much like to hear about this study too.
Re. the original post- no, Kalydeco on it's own cannot help you unless you have a mutation that affects protein function right at the surface of the cell. Right now it's approved for those with G551D, and it will next be trialed for those with other gating mutations (since they are the same kind of defect) and some others that are similar in nature- where CFTR is there, at the cell surface in sufficient quantity.
If you have DF508, the problem is more complex. The protein is degrading before it can even reach the surface of the cell, so you need what is called a 'corrector' (enter 809 or 661), and if one/both of them are successful in restoring enough function, Kalydeco- the potentiator- can then open the 'gate' at the cell surface. If all of this works, THEN we hope to see results for those with DF508 similar in nature to those with G551D. But as early 809 results indicated, more dose tweaking is needed, and/or 661 may end up being the better choice, and/or the two correctors may need to be used together to achieve the level of function needed for meaningful results. Only time is going to tell.
Re. the original post- no, Kalydeco on it's own cannot help you unless you have a mutation that affects protein function right at the surface of the cell. Right now it's approved for those with G551D, and it will next be trialed for those with other gating mutations (since they are the same kind of defect) and some others that are similar in nature- where CFTR is there, at the cell surface in sufficient quantity.
If you have DF508, the problem is more complex. The protein is degrading before it can even reach the surface of the cell, so you need what is called a 'corrector' (enter 809 or 661), and if one/both of them are successful in restoring enough function, Kalydeco- the potentiator- can then open the 'gate' at the cell surface. If all of this works, THEN we hope to see results for those with DF508 similar in nature to those with G551D. But as early 809 results indicated, more dose tweaking is needed, and/or 661 may end up being the better choice, and/or the two correctors may need to be used together to achieve the level of function needed for meaningful results. Only time is going to tell.
Bill, I am really interested in that study! I know they have identified some modifier genes that they feel impact the disease course for some with CF. I'd very much like to hear about this study too.
Re. the original post- no, Kalydeco on it's own cannot help you unless you have a mutation that affects protein function right at the surface of the cell. Right now it's approved for those with G551D, and it will next be trialed for those with other gating mutations (since they are the same kind of defect) and some others that are similar in nature- where CFTR is there, at the cell surface in sufficient quantity.
If you have DF508, the problem is more complex. The protein is degrading before it can even reach the surface of the cell, so you need what is called a 'corrector' (enter 809 or 661), and if one/both of them are successful in restoring enough function, Kalydeco- the potentiator- can then open the 'gate' at the cell surface. If all of this works, THEN we hope to see results for those with DF508 similar in nature to those with G551D. But as early 809 results indicated, more dose tweaking is needed, and/or 661 may end up being the better choice, and/or the two correctors may need to be used together to achieve the level of function needed for meaningful results. Only time is going to tell.
Re. the original post- no, Kalydeco on it's own cannot help you unless you have a mutation that affects protein function right at the surface of the cell. Right now it's approved for those with G551D, and it will next be trialed for those with other gating mutations (since they are the same kind of defect) and some others that are similar in nature- where CFTR is there, at the cell surface in sufficient quantity.
If you have DF508, the problem is more complex. The protein is degrading before it can even reach the surface of the cell, so you need what is called a 'corrector' (enter 809 or 661), and if one/both of them are successful in restoring enough function, Kalydeco- the potentiator- can then open the 'gate' at the cell surface. If all of this works, THEN we hope to see results for those with DF508 similar in nature to those with G551D. But as early 809 results indicated, more dose tweaking is needed, and/or 661 may end up being the better choice, and/or the two correctors may need to be used together to achieve the level of function needed for meaningful results. Only time is going to tell.