Breaking News!!!!!!!!!!!!!!!!!!!!!!!

my65roses4me · Mar 27, 2009

I received this email from CFF.org.
It sounds so promising!!

March 25, 2009

The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis - a faulty gene and its protein product, called CFTR.

Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a "corrector" and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would.

The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients.

"Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "We are encouraged by Vertex's rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF."

"The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."

VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.

Read Vertex's press release.

my65roses4me · Mar 27, 2009

I received this email from CFF.org.
It sounds so promising!!

March 25, 2009

The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis - a faulty gene and its protein product, called CFTR.

Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a "corrector" and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would.

The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients.

"Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "We are encouraged by Vertex's rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF."

"The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."

VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.

Read Vertex's press release.

my65roses4me · Mar 27, 2009

I received this email from CFF.org.
It sounds so promising!!

March 25, 2009

The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis - a faulty gene and its protein product, called CFTR.

Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a "corrector" and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would.

The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients.

"Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "We are encouraged by Vertex's rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF."

"The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."

VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.

Read Vertex's press release.

my65roses4me · Mar 27, 2009

I received this email from CFF.org.
It sounds so promising!!

March 25, 2009

The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis - a faulty gene and its protein product, called CFTR.

Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a "corrector" and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would.

The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients.

"Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "We are encouraged by Vertex's rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF."

"The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."

VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.

Read Vertex's press release.

my65roses4me · Mar 27, 2009

I received this email from CFF.org.
 It sounds so promising!!
 
 March 25, 2009
 
 The Cystic Fibrosis Foundation announced today the initiation of a Phase 2a clinical trial for VX-809, a compound designed to treat the basic defect in cystic fibrosis - a faulty gene and its protein product, called CFTR.
 
 Developed by Vertex Pharmaceuticals Incorporated, in collaboration with the Foundation, VX-809 is known as a "corrector" and aims to move defective CFTR protein to its proper place in the cell. The protein is then poised to move chloride out of the cell and potentially allow it to function as a healthy cell would.
 
 The Phase 2a trial is designed primarily to evaluate the safety and tolerability of multiple doses of VX-809 in patients with CF. In addition to assessing safety, the trial will also evaluate whether VX-809 affects key measures of how well the protein is functioning. The trial is expected to enroll approximately 90 patients with the Delta F508 mutation of CF, the most common mutation in CF patients.
 
 "Today, CF treatment involves the use of multiple drugs that address the symptoms of CF, however no therapy currently exists that targets the underlying cause of this disease," said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. "We are encouraged by Vertex's rapid progress in advancing two novel therapies aimed at correcting the basic defect in CF. We believe that VX-770, which is expected to enter registration studies in the first half of this year, and VX-809, which today entered Phase 2 clinical development, have the potential to play a significant role in changing the future course of this disease for people with CF."
 
 "The advancement of VX-809 into Phase 2 development demonstrates our commitment to improve care for patients with CF," said Freda Lewis-Hall, M.D., executive vice president, medicines development, and chief medical officer of Vertex. "This Phase 2a trial is designed primarily to provide a safety, tolerability and pharmacokinetic evaluation for VX-809. Any additional signals observed in this trial, including changes in sweat chloride and nasal potential difference, that indicate VX-809 has an effect on measures of CFTR function may be highly informative and encouraging for planning future clinical trials with VX-809."
 
 VX-809 was discovered as part of a collaboration between Vertex Pharmaceuticals and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate of the Foundation. Initiated in 1998, the Foundation has invested $76 million in CF research and development efforts with Vertex.
 
 Read Vertex's press release.

mamaScarlett · Mar 27, 2009

Yes I just received the same email.
Though I do keep news like this in perspective-it still is a ways off....roadblocks could come up...I could not qualify...ect
I was very happy to read this today.
Whenever I sit down to my dreaded therapy, I know I'll always feel better after doing it and have a sense of accomplishment. The past few days though, I've just been feeling "this just takes too much time".
Seeing this email today though gave me the oomph to keep doing this boring thing day after day after day...
When we get a breakthrough treatment, its going to preserve our existing lung function.
Its motivation for us to keep taking care of ourselves, and have a little more hope that big things are just around the corner.

I spoke about this drug with my doc a few weeks ago. I rarely ask my doc about breakthrough or "C" word drugs (cure)-bc I never want to put my doc in a position where she has to give me false hope. Rather for her to give me motivation to keep working. But this time, she brought it up to me. She said this is the first time she can actually say in her cf career that drugs to correct our defect are realistically coming out within our near future.

mamaScarlett · Mar 27, 2009

Yes I just received the same email.
Though I do keep news like this in perspective-it still is a ways off....roadblocks could come up...I could not qualify...ect
I was very happy to read this today.
Whenever I sit down to my dreaded therapy, I know I'll always feel better after doing it and have a sense of accomplishment. The past few days though, I've just been feeling "this just takes too much time".
Seeing this email today though gave me the oomph to keep doing this boring thing day after day after day...
When we get a breakthrough treatment, its going to preserve our existing lung function.
Its motivation for us to keep taking care of ourselves, and have a little more hope that big things are just around the corner.

I spoke about this drug with my doc a few weeks ago. I rarely ask my doc about breakthrough or "C" word drugs (cure)-bc I never want to put my doc in a position where she has to give me false hope. Rather for her to give me motivation to keep working. But this time, she brought it up to me. She said this is the first time she can actually say in her cf career that drugs to correct our defect are realistically coming out within our near future.

mamaScarlett · Mar 27, 2009

Yes I just received the same email.
Though I do keep news like this in perspective-it still is a ways off....roadblocks could come up...I could not qualify...ect
I was very happy to read this today.
Whenever I sit down to my dreaded therapy, I know I'll always feel better after doing it and have a sense of accomplishment. The past few days though, I've just been feeling "this just takes too much time".
Seeing this email today though gave me the oomph to keep doing this boring thing day after day after day...
When we get a breakthrough treatment, its going to preserve our existing lung function.
Its motivation for us to keep taking care of ourselves, and have a little more hope that big things are just around the corner.

I spoke about this drug with my doc a few weeks ago. I rarely ask my doc about breakthrough or "C" word drugs (cure)-bc I never want to put my doc in a position where she has to give me false hope. Rather for her to give me motivation to keep working. But this time, she brought it up to me. She said this is the first time she can actually say in her cf career that drugs to correct our defect are realistically coming out within our near future.

mamaScarlett · Mar 27, 2009

Yes I just received the same email.
Though I do keep news like this in perspective-it still is a ways off....roadblocks could come up...I could not qualify...ect
I was very happy to read this today.
Whenever I sit down to my dreaded therapy, I know I'll always feel better after doing it and have a sense of accomplishment. The past few days though, I've just been feeling "this just takes too much time".
Seeing this email today though gave me the oomph to keep doing this boring thing day after day after day...
When we get a breakthrough treatment, its going to preserve our existing lung function.
Its motivation for us to keep taking care of ourselves, and have a little more hope that big things are just around the corner.

I spoke about this drug with my doc a few weeks ago. I rarely ask my doc about breakthrough or "C" word drugs (cure)-bc I never want to put my doc in a position where she has to give me false hope. Rather for her to give me motivation to keep working. But this time, she brought it up to me. She said this is the first time she can actually say in her cf career that drugs to correct our defect are realistically coming out within our near future.

mamaScarlett · Mar 27, 2009

Yes I just received the same email.
 Though I do keep news like this in perspective-it still is a ways off....roadblocks could come up...I could not qualify...ect
 I was very happy to read this today.
 Whenever I sit down to my dreaded therapy, I know I'll always feel better after doing it and have a sense of accomplishment. The past few days though, I've just been feeling "this just takes too much time".
 Seeing this email today though gave me the oomph to keep doing this boring thing day after day after day...
 When we get a breakthrough treatment, its going to preserve our existing lung function.
 Its motivation for us to keep taking care of ourselves, and have a little more hope that big things are just around the corner.
 
 I spoke about this drug with my doc a few weeks ago. I rarely ask my doc about breakthrough or "C" word drugs (cure)-bc I never want to put my doc in a position where she has to give me false hope. Rather for her to give me motivation to keep working. But this time, she brought it up to me. She said this is the first time she can actually say in her cf career that drugs to correct our defect are realistically coming out within our near future.

saveferris2009 · Mar 27, 2009

posted this on my blog and twitter 7 days ago.

like khristina, it is what it is. but at least we'll get to check it out....

If all goes well the drug will be on the market in probably ~3-5 years. but it's something!

saveferris2009 · Mar 27, 2009

posted this on my blog and twitter 7 days ago.

like khristina, it is what it is. but at least we'll get to check it out....

If all goes well the drug will be on the market in probably ~3-5 years. but it's something!

saveferris2009 · Mar 27, 2009

posted this on my blog and twitter 7 days ago.

like khristina, it is what it is. but at least we'll get to check it out....

If all goes well the drug will be on the market in probably ~3-5 years. but it's something!

saveferris2009 · Mar 27, 2009

posted this on my blog and twitter 7 days ago.

like khristina, it is what it is. but at least we'll get to check it out....

If all goes well the drug will be on the market in probably ~3-5 years. but it's something!

saveferris2009 · Mar 27, 2009

posted this on my blog and twitter 7 days ago.
 
 like khristina, it is what it is. but at least we'll get to check it out....
 
 If all goes well the drug will be on the market in probably ~3-5 years. but it's something!

Sakem · Mar 28, 2009