My takeaway from the NACFC

[edan]

Hi everyone.  I was fortunate enough to be able to attend a few key meetings on Thurday evening at the NACFC in Anaheim.  In case anyone is interested, here are a few of my takeaways. I cannot for sure say my takeaways were accurate, but
I am pretty confident I understood correctly....<img src="i/expressions/face-icon-small-smile.gif" border="0"><br>
1.) There are studies that have linked a reduction in CFTR activity to
smoking. I heard this a year ago, but it was reinforced during the keynote
presentation by a prominent doctor from Heidelberg (sp?) Clearly, this would
open the total market potential for the drugs, making pharmaceutical companies
more interested in doing this high throughput screening to find a compound that
works. Yeah!!<br>
2.) Pfizer, GSK, Novartis, and other major pharma companies are currently
initiating projects to find a Vertex-like drug. The CFF has been seeding these
companies to get it all started.<br>
3.) Much research has been done on ENAC (impaired mucus clearance in cystic
fibrosis (CF) airways is a result of Airway Surface Liquid depletion (ASL)
caused by excessive Na(+) absorption through the epithelial sodium channel
(ENaC)). The compromized CFTR gene is what causes ENAC to work improperly.
Since ENaC works the same for all CF patients, regardless of mutation, a
one-size-fits-all drug may be able to be developed. As you well know, if
science goes only after CFTR mutations, there will have to be MANY, MANY drugs
in order to cover the entire CF population.<br>
4.) Another company other than Vertex will be announcing a compound
shortly.<br>
5.) Although some have thought that babies with CF are born with normal
lungs, studies have shown that some newborns already have air trapping and even
bronchiecstasis (sp?) This was brought up because they are trying to figure out
when to initiate taking drugs like Kalydeco. Clearly, the earlier the better in
many cases.   I hope some of this was helpful.  Please comment if you have more to add and/or correct. Cheers!Edan. <br>

 

[edan]

Hi everyone. I was fortunate enough to be able to attenda few key meetings on Thurdayevening at the NACFC in Anaheim. In case anyone is interested, here are a few of my takeaways. I cannot for sure say my takeaways were accurate, but
I am pretty confident I understood correctly....<img src="i/expressions/face-icon-small-smile.gif" border="0"><br>
1.) There are studies that have linked a reduction in CFTR activity to
smoking. I heard this a year ago, but it was reinforced during the keynote
presentation by a prominent doctor from Heidelberg (sp?) Clearly, this would
open the total market potential for the drugs, making pharmaceutical companies
more interested in doing this high throughput screening to find a compound that
works. Yeah!!<br>
2.) Pfizer, GSK, Novartis, and other major pharma companies are currently
initiating projects to find a Vertex-like drug. The CFF has been seeding these
companies to get it all started.<br>
3.) Much research has been done on ENAC (impaired mucus clearance in cystic
fibrosis (CF) airways is a result of Airway Surface Liquid depletion (ASL)
caused by excessive Na(+) absorption through the epithelial sodium channel
(ENaC)). The compromized CFTR gene is what causes ENAC to work improperly.
Since ENaC works the same for all CF patients, regardless of mutation, a
one-size-fits-all drug may be able to be developed. As you well know, if
science goes only after CFTR mutations, there will have to be MANY, MANY drugs
in order to cover the entire CF population.<br>
4.) Another company other than Vertex will be announcing a compound
shortly.<br>
5.) Although some have thought that babies with CF are born with normal
lungs, studies have shown that some newborns already have air trapping and even
bronchiecstasis (sp?) This was brought up because they are trying to figure out
when to initiate taking drugs like Kalydeco. Clearly, the earlier the better in
many cases. I hope some of this was helpful. Please comment if you have more to add and/or correct.Cheers!Edan.<br>

 

[edan]

<p>Hi everyone. I was fortunate enough to be able to attenda few key meetings on Thurdayevening at the NACFC in Anaheim. In case anyone is interested, here are a few of my takeaways. I cannot for sure say my takeaways were accurate, but
I am pretty confident I understood correctly....<img src="i/expressions/face-icon-small-smile.gif" border="0"><p><br>
<p>1.) There are studies that have linked a reduction in CFTR activity to
smoking. I heard this a year ago, but it was reinforced during the keynote
presentation by a prominent doctor from Heidelberg (sp?) Clearly, this would
open the total market potential for the drugs, making pharmaceutical companies
more interested in doing this high throughput screening to find a compound that
works. Yeah!!<p><br>
<p>2.) Pfizer, GSK, Novartis, and other major pharma companies are currently
initiating projects to find a Vertex-like drug. The CFF has been seeding these
companies to get it all started.<p><br>
<p>3.) Much research has been done on ENAC (impaired mucus clearance in cystic
fibrosis (CF) airways is a result of Airway Surface Liquid depletion (ASL)
caused by excessive Na(+) absorption through the epithelial sodium channel
(ENaC)). The compromized CFTR gene is what causes ENAC to work improperly.
Since ENaC works the same for all CF patients, regardless of mutation, a
one-size-fits-all drug may be able to be developed. As you well know, if
science goes only after CFTR mutations, there will have to be MANY, MANY drugs
in order to cover the entire CF population.<p><br>
<p>4.) Another company other than Vertex will be announcing a compound
shortly.<p><br>
<p>5.) Although some have thought that babies with CF are born with normal
lungs, studies have shown that some newborns already have air trapping and even
bronchiecstasis (sp?) This was brought up because they are trying to figure out
when to initiate taking drugs like Kalydeco. Clearly, the earlier the better in
many cases. <p><p><p>I hope some of this was helpful. Please comment if you have more to add and/or correct.<p><p>Cheers!<p>Edan.<p><br>

 

[Mommafirst]

Hi Edan, I was there too. You did a great job. when I have time to review my notes, I can post some of the other things I heard -- I was at all three plenary sessions and a variety of other ones as well.

 

[Mommafirst]

Hi Edan, I was there too. You did a great job. when I have time to review my notes, I can post some of the other things I heard -- I was at all three plenary sessions and a variety of other ones as well.

 

[Mommafirst]

Hi Edan, I was there too. You did a great job. when I have time to review my notes, I can post some of the other things I heard -- I was at all three plenary sessions and a variety of other ones as well.

 

[ymikhale]

Thank you for posting, it is really helpful. My dd's'doctor was there too but I doubt she will give me as detailed report!

 

[ymikhale]

Thank you for posting, it is really helpful. My dd's'doctor was there too but I doubt she will give me as detailed report!

 

[ymikhale]

Thank you for posting, it is really helpful. My dd's'doctor was there too but I doubt she will give me as detailed report!

 

[edan]

Hi Heather. Can't wait to hear what you have learned. I wish I could have been there for the whole thing!

ymikale - nice to hear from you. I hope all is well.

 

[edan]

Hi Heather. Can't wait to hear what you have learned. I wish I could have been there for the whole thing!

ymikale - nice to hear from you. I hope all is well.

 

[edan]

Hi Heather. Can't wait to hear what you have learned. I wish I could have been there for the whole thing!
<br />
<br />ymikale - nice to hear from you. I hope all is well.
<br />
<br />

 

[rmotion]

<br>Thanks for the update!Q In regards to the smoking. Is this the nicotine issue we were talking about. A reduction in CFTR is
not good or do you mean something else. Or are they saying CF's should not smoke? <br> 

 

[rmotion]

<br>Thanks for the update!Q In regards to the smoking. Is this the nicotine issue we were talking about. A reduction in CFTR is
not good or do you mean something else. Or are they saying CF's should not smoke?<br>

 

[rmotion]

<p><br>Thanks for the update!<p>Q In regards to the smoking. Is this the nicotine issue we were talking about. A reduction in CFTR is
not good or do you mean something else. Or are they saying CF's should not smoke?<p><p><br>

 

[bigstar]

Please we need more references of what happened in NACFC . Links could be very helpful! Im very excited to know that new pharmaceutical companies are getting in the game! If you have any info about their new drugs please post!

PS I am requesting again from the moderators a new section where we we will be able to discuss about researc and new drugs. Thank you!

 

[bigstar]

Please we need more references of what happened in NACFC . Links could be very helpful! Im very excited to know that new pharmaceutical companies are getting in the game! If you have any info about their new drugs please post!

PS I am requesting again from the moderators a new section where we we will be able to discuss about researc and new drugs. Thank you!

 

[bigstar]

Please we need more references of what happened in NACFC . Links could be very helpful! Im very excited to know that new pharmaceutical companies are getting in the game! If you have any info about their new drugs please post!
<br />
<br />PS I am requesting again from the moderators a new section where we we will be able to discuss about researc and new drugs. Thank you!

 

[Mommafirst]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>rmotion</b></i>

Thanks for the update!

Q In regards to the smoking. Is this the nicotine issue we were talking about. A reduction in CFTR is
not good or do you mean something else. Or are they saying CF's should not smoke?

 


 </end quote></div>

<div><br></div><div>In regards to the smoking, they are finding that long term smokers seem to have a similar diminishing function of the CFTR protein to that of CF patients.   So basically, by smoking they are giving themselves a disease similar to CF.</div>

 

[Mommafirst]

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>rmotion</b></i>

Thanks for the update!

Q In regards to the smoking. Is this the nicotine issue we were talking about. A reduction in CFTR is
not good or do you mean something else. Or are they saying CF's should not smoke?




</end quote>

<br>In regards to the smoking, they are finding that long term smokers seem to have a similar diminishing function of the CFTR protein to that of CF patients. So basically, by smoking they are giving themselves a disease similar to CF.