A "cure"? Please explain...

kitomd21 · Aug 5, 2008

What are your thoughts regarding the two following treatments? They are posted at the CFF website under drug development pipeline. As they are in the later phases toward approval, don't these seem extremely promising? Am I a fool for viewing them as a "cure"?

PROTEIN ASSIST/REPAIR

This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs.

PTC 124 (PTC Therapeutics) - PTC 124 is a novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It has been demonstrated to be safe, orally available and well-tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results.

RESTORE SALT TRANSPORT

The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium & chloride) along the cell surface.

Denufosol (Inspire Pharmaceuticals, Inc.) - Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.

kitomd21 · Aug 5, 2008

What are your thoughts regarding the two following treatments? They are posted at the CFF website under drug development pipeline. As they are in the later phases toward approval, don't these seem extremely promising? Am I a fool for viewing them as a "cure"?

PROTEIN ASSIST/REPAIR

This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs.

PTC 124 (PTC Therapeutics) - PTC 124 is a novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It has been demonstrated to be safe, orally available and well-tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results.

RESTORE SALT TRANSPORT

The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium & chloride) along the cell surface.

Denufosol (Inspire Pharmaceuticals, Inc.) - Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.

kitomd21 · Aug 5, 2008

What are your thoughts regarding the two following treatments? They are posted at the CFF website under drug development pipeline. As they are in the later phases toward approval, don't these seem extremely promising? Am I a fool for viewing them as a "cure"?

PROTEIN ASSIST/REPAIR

This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs.

PTC 124 (PTC Therapeutics) - PTC 124 is a novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It has been demonstrated to be safe, orally available and well-tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results.

RESTORE SALT TRANSPORT

The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium & chloride) along the cell surface.

Denufosol (Inspire Pharmaceuticals, Inc.) - Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.

kitomd21 · Aug 5, 2008

What are your thoughts regarding the two following treatments? They are posted at the CFF website under drug development pipeline. As they are in the later phases toward approval, don't these seem extremely promising? Am I a fool for viewing them as a "cure"?

PROTEIN ASSIST/REPAIR

This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs.

PTC 124 (PTC Therapeutics) - PTC 124 is a novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It has been demonstrated to be safe, orally available and well-tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results.

RESTORE SALT TRANSPORT

The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium & chloride) along the cell surface.

Denufosol (Inspire Pharmaceuticals, Inc.) - Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.

kitomd21 · Aug 5, 2008

What are your thoughts regarding the two following treatments? They are posted at the CFF website under drug development pipeline. As they are in the later phases toward approval, don't these seem extremely promising? Am I a fool for viewing them as a "cure"?
 
 PROTEIN ASSIST/REPAIR
 
 This therapy is designed to correct the function of the defective CFTR protein made by the CF gene to allow chloride and sodium (salt) to move properly in cells lining the lungs and other organs.
 
 PTC 124 (PTC Therapeutics) - PTC 124 is a novel, small molecule compound, that promotes the read-through of premature truncation codons in the CFTR mRNA. It has been demonstrated to be safe, orally available and well-tolerated in a Phase 1 single-dose trial in healthy volunteers. A Phase 2 trial in CF patients conducted in the United States and Israel demonstrated safety and encouraging biological results.
 
 
 
 RESTORE SALT TRANSPORT
 
 The goal of this approach is to hydrate thick CF mucus in the lungs by correcting the amount of salt (sodium & chloride) along the cell surface.
 
 Denufosol (Inspire Pharmaceuticals, Inc.) - Denufosol is being used to correct the ion transport defect in CF. A recently completed Phase 2 trial to determine the effect of drug on pulmonary function in CF patients demonstrated efficacy. The first of two FDA-mandated Phase 3 trials has completed enrollment. The second Phase 3 trial will be a multi-national one and is scheduled to begin in 2008.

Rebjane · Aug 5, 2008

A "cure

Both of these drugs show promise, in my opinion. There is reason for hope. I don't think "cure" in the sense that the CF is gone forever, but these treatments and drugs in the future may make the life of a CF'er closer to manageable. The goal is to maintain lung fuction and prevent damage for the most quality of life. Cure to me means that the CF is completely gone, which in a genetic disease, would mean correcting the CFTR in every cell in the person's body, which I don't see that happening in the near future. Hopefully I didn't read too much into your question but I do think reseachers are on to something with these drugs.

Rebjane · Aug 5, 2008

A "cure

Both of these drugs show promise, in my opinion. There is reason for hope. I don't think "cure" in the sense that the CF is gone forever, but these treatments and drugs in the future may make the life of a CF'er closer to manageable. The goal is to maintain lung fuction and prevent damage for the most quality of life. Cure to me means that the CF is completely gone, which in a genetic disease, would mean correcting the CFTR in every cell in the person's body, which I don't see that happening in the near future. Hopefully I didn't read too much into your question but I do think reseachers are on to something with these drugs.

Rebjane · Aug 5, 2008

A "cure

Both of these drugs show promise, in my opinion. There is reason for hope. I don't think "cure" in the sense that the CF is gone forever, but these treatments and drugs in the future may make the life of a CF'er closer to manageable. The goal is to maintain lung fuction and prevent damage for the most quality of life. Cure to me means that the CF is completely gone, which in a genetic disease, would mean correcting the CFTR in every cell in the person's body, which I don't see that happening in the near future. Hopefully I didn't read too much into your question but I do think reseachers are on to something with these drugs.

Rebjane · Aug 5, 2008

A "cure

Both of these drugs show promise, in my opinion. There is reason for hope. I don't think "cure" in the sense that the CF is gone forever, but these treatments and drugs in the future may make the life of a CF'er closer to manageable. The goal is to maintain lung fuction and prevent damage for the most quality of life. Cure to me means that the CF is completely gone, which in a genetic disease, would mean correcting the CFTR in every cell in the person's body, which I don't see that happening in the near future. Hopefully I didn't read too much into your question but I do think reseachers are on to something with these drugs.

Rebjane · Aug 5, 2008

A "cure

Both of these drugs show promise, in my opinion. There is reason for hope. I don't think "cure" in the sense that the CF is gone forever, but these treatments and drugs in the future may make the life of a CF'er closer to manageable. The goal is to maintain lung fuction and prevent damage for the most quality of life. Cure to me means that the CF is completely gone, which in a genetic disease, would mean correcting the CFTR in every cell in the person's body, which I don't see that happening in the near future. Hopefully I didn't read too much into your question but I do think reseachers are on to something with these drugs.

kitomd21 · Aug 5, 2008