Inhaled Aztreonam turned down by FDA

Craigsmom · Sep 23, 2008

I agree, it seems like some people are really getting rich off of playing with peoples lives!!! In 1989 when my son was diagnosed I can remember hearing it wont be long before a cure is found ha! It makes me really wonder why would anyone find a cure, bc it would stop so many people from making so much monnneeeyy. The important thing should be to find a cure to stop all the suffering CFers have to go through, all the financial problems it causes trying to make sure they can get the drugs needed. Its hard to believe that TOBI, CREON, etc cost so much!!
Its is really sad to live in the US and we have all these concerns.

Craigsmom · Sep 23, 2008

I agree, it seems like some people are really getting rich off of playing with peoples lives!!! In 1989 when my son was diagnosed I can remember hearing it wont be long before a cure is found ha! It makes me really wonder why would anyone find a cure, bc it would stop so many people from making so much monnneeeyy. The important thing should be to find a cure to stop all the suffering CFers have to go through, all the financial problems it causes trying to make sure they can get the drugs needed. Its hard to believe that TOBI, CREON, etc cost so much!!
Its is really sad to live in the US and we have all these concerns.

Craigsmom · Sep 23, 2008

I agree, it seems like some people are really getting rich off of playing with peoples lives!!! In 1989 when my son was diagnosed I can remember hearing it wont be long before a cure is found ha! It makes me really wonder why would anyone find a cure, bc it would stop so many people from making so much monnneeeyy. The important thing should be to find a cure to stop all the suffering CFers have to go through, all the financial problems it causes trying to make sure they can get the drugs needed. Its hard to believe that TOBI, CREON, etc cost so much!!
Its is really sad to live in the US and we have all these concerns.

Craigsmom · Sep 23, 2008

I agree, it seems like some people are really getting rich off of playing with peoples lives!!! In 1989 when my son was diagnosed I can remember hearing it wont be long before a cure is found ha! It makes me really wonder why would anyone find a cure, bc it would stop so many people from making so much monnneeeyy. The important thing should be to find a cure to stop all the suffering CFers have to go through, all the financial problems it causes trying to make sure they can get the drugs needed. Its hard to believe that TOBI, CREON, etc cost so much!!
Its is really sad to live in the US and we have all these concerns.

Craigsmom · Sep 23, 2008

I agree, it seems like some people are really getting rich off of playing with peoples lives!!! In 1989 when my son was diagnosed I can remember hearing it wont be long before a cure is found ha! It makes me really wonder why would anyone find a cure, bc it would stop so many people from making so much monnneeeyy. The important thing should be to find a cure to stop all the suffering CFers have to go through, all the financial problems it causes trying to make sure they can get the drugs needed. Its hard to believe that TOBI, CREON, etc cost so much!!
 Its is really sad to live in the US and we have all these concerns.

Mockingbird · Sep 23, 2008

I don't think that's true. Keep in mind there's still a lot about the human body doctors don't understand. We still don't know exactly how the CFTR protein works.

I beileve a lot of people are working hard to find new treatments for CF, and money is not their primary motivation. The doctors at my own clinic are always extremely busy because they're involved in both research and patient care. Also, I have a friend who is going for her PhD, and I know her well enough to say her motivation is not money, either.

It's just that, as humans, we do have limitations as far as how much we understand and how much we can do. It's not as simple as looking into a microscope; this world still has a lot of mysteries science has yet to discover.

Mockingbird · Sep 23, 2008

I don't think that's true. Keep in mind there's still a lot about the human body doctors don't understand. We still don't know exactly how the CFTR protein works.

I beileve a lot of people are working hard to find new treatments for CF, and money is not their primary motivation. The doctors at my own clinic are always extremely busy because they're involved in both research and patient care. Also, I have a friend who is going for her PhD, and I know her well enough to say her motivation is not money, either.

It's just that, as humans, we do have limitations as far as how much we understand and how much we can do. It's not as simple as looking into a microscope; this world still has a lot of mysteries science has yet to discover.

Mockingbird · Sep 23, 2008

I don't think that's true. Keep in mind there's still a lot about the human body doctors don't understand. We still don't know exactly how the CFTR protein works.

I beileve a lot of people are working hard to find new treatments for CF, and money is not their primary motivation. The doctors at my own clinic are always extremely busy because they're involved in both research and patient care. Also, I have a friend who is going for her PhD, and I know her well enough to say her motivation is not money, either.

It's just that, as humans, we do have limitations as far as how much we understand and how much we can do. It's not as simple as looking into a microscope; this world still has a lot of mysteries science has yet to discover.

Mockingbird · Sep 23, 2008

I don't think that's true. Keep in mind there's still a lot about the human body doctors don't understand. We still don't know exactly how the CFTR protein works.

I beileve a lot of people are working hard to find new treatments for CF, and money is not their primary motivation. The doctors at my own clinic are always extremely busy because they're involved in both research and patient care. Also, I have a friend who is going for her PhD, and I know her well enough to say her motivation is not money, either.

It's just that, as humans, we do have limitations as far as how much we understand and how much we can do. It's not as simple as looking into a microscope; this world still has a lot of mysteries science has yet to discover.

Mockingbird · Sep 23, 2008

I don't think that's true. Keep in mind there's still a lot about the human body doctors don't understand. We still don't know exactly how the CFTR protein works.
 
 I beileve a lot of people are working hard to find new treatments for CF, and money is not their primary motivation. The doctors at my own clinic are always extremely busy because they're involved in both research and patient care. Also, I have a friend who is going for her PhD, and I know her well enough to say her motivation is not money, either.
 
 It's just that, as humans, we do have limitations as far as how much we understand and how much we can do. It's not as simple as looking into a microscope; this world still has a lot of mysteries science has yet to discover.

just1more · Sep 23, 2008

A cure is not forthcoming, and it is not due to money directly. It is due to simple science.

As we all know, there are over 1500 known mutations which are known to cause a disease called CF. From a cure standpoint, this is 1500 DIFFERENT diseases that need cured. While technology to fix one could be used to fix others possibly, at the end of the day you would have to 'cure' each defect. Would a treatment that fixes a given mutation (even DF508) be considered a cure?

However, if you look at treatment the odds are better. There are maybe a dozen or two (my guess) primary symptoms and causes of death from CF. Logically, it is eaiser to spend your resources tackling this much smaller group of symptoms looking for the best treatment option.

Ultimately, there will be a successful genetic treatment option, but it will first come for something with a much smaller target to hit. A prime example is Hemophilia B with 1 mutation to try and repair and an instant pass/fail result for your treatment; or Parkinsons which while a bit more complex, is nowhere near CF and has millions of potential customers to offset the costs.

just1more · Sep 23, 2008

A cure is not forthcoming, and it is not due to money directly. It is due to simple science.

As we all know, there are over 1500 known mutations which are known to cause a disease called CF. From a cure standpoint, this is 1500 DIFFERENT diseases that need cured. While technology to fix one could be used to fix others possibly, at the end of the day you would have to 'cure' each defect. Would a treatment that fixes a given mutation (even DF508) be considered a cure?

However, if you look at treatment the odds are better. There are maybe a dozen or two (my guess) primary symptoms and causes of death from CF. Logically, it is eaiser to spend your resources tackling this much smaller group of symptoms looking for the best treatment option.

Ultimately, there will be a successful genetic treatment option, but it will first come for something with a much smaller target to hit. A prime example is Hemophilia B with 1 mutation to try and repair and an instant pass/fail result for your treatment; or Parkinsons which while a bit more complex, is nowhere near CF and has millions of potential customers to offset the costs.

just1more · Sep 23, 2008

A cure is not forthcoming, and it is not due to money directly. It is due to simple science.

As we all know, there are over 1500 known mutations which are known to cause a disease called CF. From a cure standpoint, this is 1500 DIFFERENT diseases that need cured. While technology to fix one could be used to fix others possibly, at the end of the day you would have to 'cure' each defect. Would a treatment that fixes a given mutation (even DF508) be considered a cure?

However, if you look at treatment the odds are better. There are maybe a dozen or two (my guess) primary symptoms and causes of death from CF. Logically, it is eaiser to spend your resources tackling this much smaller group of symptoms looking for the best treatment option.

Ultimately, there will be a successful genetic treatment option, but it will first come for something with a much smaller target to hit. A prime example is Hemophilia B with 1 mutation to try and repair and an instant pass/fail result for your treatment; or Parkinsons which while a bit more complex, is nowhere near CF and has millions of potential customers to offset the costs.

just1more · Sep 23, 2008

A cure is not forthcoming, and it is not due to money directly. It is due to simple science.

As we all know, there are over 1500 known mutations which are known to cause a disease called CF. From a cure standpoint, this is 1500 DIFFERENT diseases that need cured. While technology to fix one could be used to fix others possibly, at the end of the day you would have to 'cure' each defect. Would a treatment that fixes a given mutation (even DF508) be considered a cure?

However, if you look at treatment the odds are better. There are maybe a dozen or two (my guess) primary symptoms and causes of death from CF. Logically, it is eaiser to spend your resources tackling this much smaller group of symptoms looking for the best treatment option.

Ultimately, there will be a successful genetic treatment option, but it will first come for something with a much smaller target to hit. A prime example is Hemophilia B with 1 mutation to try and repair and an instant pass/fail result for your treatment; or Parkinsons which while a bit more complex, is nowhere near CF and has millions of potential customers to offset the costs.

just1more · Sep 23, 2008

A cure is not forthcoming, and it is not due to money directly. It is due to simple science.
 
 As we all know, there are over 1500 known mutations which are known to cause a disease called CF. From a cure standpoint, this is 1500 DIFFERENT diseases that need cured. While technology to fix one could be used to fix others possibly, at the end of the day you would have to 'cure' each defect. Would a treatment that fixes a given mutation (even DF508) be considered a cure?
 
 However, if you look at treatment the odds are better. There are maybe a dozen or two (my guess) primary symptoms and causes of death from CF. Logically, it is eaiser to spend your resources tackling this much smaller group of symptoms looking for the best treatment option.
 
 Ultimately, there will be a successful genetic treatment option, but it will first come for something with a much smaller target to hit. A prime example is Hemophilia B with 1 mutation to try and repair and an instant pass/fail result for your treatment; or Parkinsons which while a bit more complex, is nowhere near CF and has millions of potential customers to offset the costs.

ladybug · Sep 23, 2008

I think there are limitations to humans, as well as some red tape that do factor into whether or not drugs get passed and how quickly (no matter how much we'd like to believe otherwise). And, oftentimes I dont' believe its the docs and people who work directly with patients that hold things up, but big pharmaceutical companies AND the FDA. Be it for money or just to avoid lawsuits if even the tiniest thing were to ever go wrong.

I think the biggest disappointment I have is not that we're not getting a "cure" as promised, but that these pipeline drugs take WAY too long to get to us. Too much red tape, too much cost, too much everything.

Wasn't TOBI the last great drug that came out for CF? Wasn't that in the 90's? So, in about 20 years, we've had one great drug (that doesn't even work for many) come out. Not too impressive.

Plus, when you figure things like glutathione and all the "natural" trials that have been ongoing for years (my clinic highly recommends NOT taking things until they are approved and I follow their warnings), it seems like nothing new is every coming out for CF.

Then, when we are promised something great, something like this happens. Even if it was not a cure, Azli stood to help many many people from deterioriating health (that's how I understood it anyway). Now, those of us who can't yet get it, will simply continue what we've been doing the past two decades and hope we don't decline too much to be helped by new meds when/if they do come out.

Its just very unfortunate.

ladybug · Sep 23, 2008

I think there are limitations to humans, as well as some red tape that do factor into whether or not drugs get passed and how quickly (no matter how much we'd like to believe otherwise). And, oftentimes I dont' believe its the docs and people who work directly with patients that hold things up, but big pharmaceutical companies AND the FDA. Be it for money or just to avoid lawsuits if even the tiniest thing were to ever go wrong.

I think the biggest disappointment I have is not that we're not getting a "cure" as promised, but that these pipeline drugs take WAY too long to get to us. Too much red tape, too much cost, too much everything.

Wasn't TOBI the last great drug that came out for CF? Wasn't that in the 90's? So, in about 20 years, we've had one great drug (that doesn't even work for many) come out. Not too impressive.

Plus, when you figure things like glutathione and all the "natural" trials that have been ongoing for years (my clinic highly recommends NOT taking things until they are approved and I follow their warnings), it seems like nothing new is every coming out for CF.

Then, when we are promised something great, something like this happens. Even if it was not a cure, Azli stood to help many many people from deterioriating health (that's how I understood it anyway). Now, those of us who can't yet get it, will simply continue what we've been doing the past two decades and hope we don't decline too much to be helped by new meds when/if they do come out.

Its just very unfortunate.

ladybug · Sep 23, 2008

I think there are limitations to humans, as well as some red tape that do factor into whether or not drugs get passed and how quickly (no matter how much we'd like to believe otherwise). And, oftentimes I dont' believe its the docs and people who work directly with patients that hold things up, but big pharmaceutical companies AND the FDA. Be it for money or just to avoid lawsuits if even the tiniest thing were to ever go wrong.

I think the biggest disappointment I have is not that we're not getting a "cure" as promised, but that these pipeline drugs take WAY too long to get to us. Too much red tape, too much cost, too much everything.

Wasn't TOBI the last great drug that came out for CF? Wasn't that in the 90's? So, in about 20 years, we've had one great drug (that doesn't even work for many) come out. Not too impressive.

Plus, when you figure things like glutathione and all the "natural" trials that have been ongoing for years (my clinic highly recommends NOT taking things until they are approved and I follow their warnings), it seems like nothing new is every coming out for CF.

Then, when we are promised something great, something like this happens. Even if it was not a cure, Azli stood to help many many people from deterioriating health (that's how I understood it anyway). Now, those of us who can't yet get it, will simply continue what we've been doing the past two decades and hope we don't decline too much to be helped by new meds when/if they do come out.

Its just very unfortunate.

ladybug · Sep 23, 2008

I think there are limitations to humans, as well as some red tape that do factor into whether or not drugs get passed and how quickly (no matter how much we'd like to believe otherwise). And, oftentimes I dont' believe its the docs and people who work directly with patients that hold things up, but big pharmaceutical companies AND the FDA. Be it for money or just to avoid lawsuits if even the tiniest thing were to ever go wrong.

I think the biggest disappointment I have is not that we're not getting a "cure" as promised, but that these pipeline drugs take WAY too long to get to us. Too much red tape, too much cost, too much everything.

Wasn't TOBI the last great drug that came out for CF? Wasn't that in the 90's? So, in about 20 years, we've had one great drug (that doesn't even work for many) come out. Not too impressive.

Plus, when you figure things like glutathione and all the "natural" trials that have been ongoing for years (my clinic highly recommends NOT taking things until they are approved and I follow their warnings), it seems like nothing new is every coming out for CF.

Then, when we are promised something great, something like this happens. Even if it was not a cure, Azli stood to help many many people from deterioriating health (that's how I understood it anyway). Now, those of us who can't yet get it, will simply continue what we've been doing the past two decades and hope we don't decline too much to be helped by new meds when/if they do come out.

Its just very unfortunate.

ladybug · Sep 23, 2008

I think there are limitations to humans, as well as some red tape that do factor into whether or not drugs get passed and how quickly (no matter how much we'd like to believe otherwise). And, oftentimes I dont' believe its the docs and people who work directly with patients that hold things up, but big pharmaceutical companies AND the FDA. Be it for money or just to avoid lawsuits if even the tiniest thing were to ever go wrong.
 
 I think the biggest disappointment I have is not that we're not getting a "cure" as promised, but that these pipeline drugs take WAY too long to get to us. Too much red tape, too much cost, too much everything.
 
 Wasn't TOBI the last great drug that came out for CF? Wasn't that in the 90's? So, in about 20 years, we've had one great drug (that doesn't even work for many) come out. Not too impressive.
 
 Plus, when you figure things like glutathione and all the "natural" trials that have been ongoing for years (my clinic highly recommends NOT taking things until they are approved and I follow their warnings), it seems like nothing new is every coming out for CF.
 
 Then, when we are promised something great, something like this happens. Even if it was not a cure, Azli stood to help many many people from deterioriating health (that's how I understood it anyway). Now, those of us who can't yet get it, will simply continue what we've been doing the past two decades and hope we don't decline too much to be helped by new meds when/if they do come out.
 
 Its just very unfortunate.

Inhaled Aztreonam turned down by FDA

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member

New member