Questions for brainy Adult CFer's about new study drugs

P

petersymons

Guest
the drug is called meveol . Hopefully it will revolutionise the treatment of infections.
<br />www.alaxia-pharma.eu/meveol
 

Ender

New member
Hey there,

From my understanding, these drugs are targeted at people with specific mutations whereas the cftr protein is at the cell surface, but not folded correctly. These drugs correct the protein, thus restoring proper function. It won`t reverse damage, but i suspect that with reduced mucus and inflammation, people should get some lung function back even if they are pretty low (as long as there isn`t tooo much scarring).

For people that have culture bugs their whole life, i don`t think you`ll get rid of them, but it should be easier to treat. Look at people who have had transplants and still have problems with the bugs they cultured before. Hopefully though with the mucus in check, the bugs won`t be much of a problem and the cycle of infection, inflammation, mucus production will lesson dramatically.

One anti infective medicine though that i have high hopes for is a nano emulsion already proven to kill most bugs that kill us, and is in phase 3 studies for other infectious diseases. Looks really promising!

<a target=_blank class=ftalternatingbarlinklarge href="http://nanotechnologytoday.blogspot.com/2009/03/nanoemulsion-potent-against-superbugs.html
">http://nanotechnologytoday.blo...-superbugs.html
</a>

from the website:

In cell cultures in the lab, the U-M scientists tested a nanoemulsion against 150 bacterial strains that attack cystic fibrosis patients. The emulsion proved effective at killing all of them, including one-third that are resistant to many antibiotics and 13 percent that resist all antibiotics.

They then tested the nanoemulsion against several bacterial strains grown in biofilms and sputum, to more closely simulate conditions in a patient's body. Antibiotics often can't penetrate biofilms and sputum unless given at high doses with unacceptable side effects.

"We saw, not surprisingly, that greater concentrations of nanoemulsion were required to kill the bacteria, but we saw no strains that were resistant," LiPuma says. Whether humans can tolerate those concentrations well remains to be seen.

LiPuma's lab, funded by the Cystic Fibrosis Foundation as a national reference lab, has collected more than 30,000 strains of bacteria from the lungs of cystic fibrosis patients. The lab receives samples from around the world for analysis.

What's next

The University of Michigan has filed for patent protection on the CF nanoemulsion, and licensed this technology to Ann Arbor-based NanoBio Corporation. Baker is a founder and equity holder of NanoBio. NanoBio and LiPuma's lab will cooperate in the next steps toward bringing the treatment to market. LiPuma is optimistic that if animal and human trials go well, a nanoemulsion treatment for cystic fibrosis infections could be available in as little as five years. ##

Hope that helps a bit
Kiel
 

Ender

New member
Hey there,

From my understanding, these drugs are targeted at people with specific mutations whereas the cftr protein is at the cell surface, but not folded correctly. These drugs correct the protein, thus restoring proper function. It won`t reverse damage, but i suspect that with reduced mucus and inflammation, people should get some lung function back even if they are pretty low (as long as there isn`t tooo much scarring).

For people that have culture bugs their whole life, i don`t think you`ll get rid of them, but it should be easier to treat. Look at people who have had transplants and still have problems with the bugs they cultured before. Hopefully though with the mucus in check, the bugs won`t be much of a problem and the cycle of infection, inflammation, mucus production will lesson dramatically.

One anti infective medicine though that i have high hopes for is a nano emulsion already proven to kill most bugs that kill us, and is in phase 3 studies for other infectious diseases. Looks really promising!

<a target=_blank class=ftalternatingbarlinklarge href="http://nanotechnologytoday.blogspot.com/2009/03/nanoemulsion-potent-against-superbugs.html
">http://nanotechnologytoday.blo...-superbugs.html
</a>

from the website:

In cell cultures in the lab, the U-M scientists tested a nanoemulsion against 150 bacterial strains that attack cystic fibrosis patients. The emulsion proved effective at killing all of them, including one-third that are resistant to many antibiotics and 13 percent that resist all antibiotics.

They then tested the nanoemulsion against several bacterial strains grown in biofilms and sputum, to more closely simulate conditions in a patient's body. Antibiotics often can't penetrate biofilms and sputum unless given at high doses with unacceptable side effects.

"We saw, not surprisingly, that greater concentrations of nanoemulsion were required to kill the bacteria, but we saw no strains that were resistant," LiPuma says. Whether humans can tolerate those concentrations well remains to be seen.

LiPuma's lab, funded by the Cystic Fibrosis Foundation as a national reference lab, has collected more than 30,000 strains of bacteria from the lungs of cystic fibrosis patients. The lab receives samples from around the world for analysis.

What's next

The University of Michigan has filed for patent protection on the CF nanoemulsion, and licensed this technology to Ann Arbor-based NanoBio Corporation. Baker is a founder and equity holder of NanoBio. NanoBio and LiPuma's lab will cooperate in the next steps toward bringing the treatment to market. LiPuma is optimistic that if animal and human trials go well, a nanoemulsion treatment for cystic fibrosis infections could be available in as little as five years. ##

Hope that helps a bit
Kiel
 

Ender

New member
Hey there,
<br />
<br />From my understanding, these drugs are targeted at people with specific mutations whereas the cftr protein is at the cell surface, but not folded correctly. These drugs correct the protein, thus restoring proper function. It won`t reverse damage, but i suspect that with reduced mucus and inflammation, people should get some lung function back even if they are pretty low (as long as there isn`t tooo much scarring).
<br />
<br />For people that have culture bugs their whole life, i don`t think you`ll get rid of them, but it should be easier to treat. Look at people who have had transplants and still have problems with the bugs they cultured before. Hopefully though with the mucus in check, the bugs won`t be much of a problem and the cycle of infection, inflammation, mucus production will lesson dramatically.
<br />
<br />One anti infective medicine though that i have high hopes for is a nano emulsion already proven to kill most bugs that kill us, and is in phase 3 studies for other infectious diseases. Looks really promising!
<br />
<br /><a target=_blank class=ftalternatingbarlinklarge href="http://nanotechnologytoday.blogspot.com/2009/03/nanoemulsion-potent-against-superbugs.html
<br />">http://nanotechnologytoday.blo...-superbugs.html
<br /></a>
<br />
<br />from the website:
<br />
<br />In cell cultures in the lab, the U-M scientists tested a nanoemulsion against 150 bacterial strains that attack cystic fibrosis patients. The emulsion proved effective at killing all of them, including one-third that are resistant to many antibiotics and 13 percent that resist all antibiotics.
<br />
<br />They then tested the nanoemulsion against several bacterial strains grown in biofilms and sputum, to more closely simulate conditions in a patient's body. Antibiotics often can't penetrate biofilms and sputum unless given at high doses with unacceptable side effects.
<br />
<br />"We saw, not surprisingly, that greater concentrations of nanoemulsion were required to kill the bacteria, but we saw no strains that were resistant," LiPuma says. Whether humans can tolerate those concentrations well remains to be seen.
<br />
<br />LiPuma's lab, funded by the Cystic Fibrosis Foundation as a national reference lab, has collected more than 30,000 strains of bacteria from the lungs of cystic fibrosis patients. The lab receives samples from around the world for analysis.
<br />
<br />What's next
<br />
<br />The University of Michigan has filed for patent protection on the CF nanoemulsion, and licensed this technology to Ann Arbor-based NanoBio Corporation. Baker is a founder and equity holder of NanoBio. NanoBio and LiPuma's lab will cooperate in the next steps toward bringing the treatment to market. LiPuma is optimistic that if animal and human trials go well, a nanoemulsion treatment for cystic fibrosis infections could be available in as little as five years. ##
<br />
<br />Hope that helps a bit
<br />Kiel
 

missT

Member
Kiel, from everything that I have learned I would think they would work exactely like you said but I dont think that is the case. Each class of mutation has a different problem with the CFTR channel. In nonsense mutations that end with X there is no functioning protein (class 1). That is why I feel the vertex and combo drugs ARE working because it is a different level of CFTR malfunctioning...meaning that the protein is already there it is just folded wrong.
Your info on the new drug to kill all our bug strains is quite interesting but I really wonder what the side effects would be. I cringe when I read about our poor CFer's who cant hear because of Tobi.
 

missT

Member
Kiel, from everything that I have learned I would think they would work exactely like you said but I dont think that is the case. Each class of mutation has a different problem with the CFTR channel. In nonsense mutations that end with X there is no functioning protein (class 1). That is why I feel the vertex and combo drugs ARE working because it is a different level of CFTR malfunctioning...meaning that the protein is already there it is just folded wrong.
Your info on the new drug to kill all our bug strains is quite interesting but I really wonder what the side effects would be. I cringe when I read about our poor CFer's who cant hear because of Tobi.
 

missT

Member
Kiel, from everything that I have learned I would think they would work exactely like you said but I dont think that is the case. Each class of mutation has a different problem with the CFTR channel. In nonsense mutations that end with X there is no functioning protein (class 1). That is why I feel the vertex and combo drugs ARE working because it is a different level of CFTR malfunctioning...meaning that the protein is already there it is just folded wrong.
<br />Your info on the new drug to kill all our bug strains is quite interesting but I really wonder what the side effects would be. I cringe when I read about our poor CFer's who cant hear because of Tobi.
 

dasjsmum

New member
yes, that's right. I know the G551D has an open channel but closed gate thingo that the chloride passess through. The DF508 has a closed channel and gate.The 770 opens the gate so people's bodies act the way people without cf bodies act.

@miss T The 809 is supposed (hopefully) to open the channel, then they are adding the 770 which opens the 'gate'. sorry about my rudimentary understanding, but that's basically how the drug works.

As far as mucus etc goes, my understanding is that cfer's have not enough mucus and it's too thick. If the chloride channel works correctly, the body should correct the mucus thickness etc that is the underlying issue with cf....is your trial double blind or open? Is everyone on the drug for sure, or could you be on placebo (therefore you wont notice a difference).I dont understand what the problem is with the W gene, so cant comment....you might be right.

Personally, I dont think that cfer's on the vertex or other that correct the defect will still have issues with pseudo etc.
 

dasjsmum

New member
yes, that's right. I know the G551D has an open channel but closed gate thingo that the chloride passess through. The DF508 has a closed channel and gate.The 770 opens the gate so people's bodies act the way people without cf bodies act.

@miss T The 809 is supposed (hopefully) to open the channel, then they are adding the 770 which opens the 'gate'. sorry about my rudimentary understanding, but that's basically how the drug works.

As far as mucus etc goes, my understanding is that cfer's have not enough mucus and it's too thick. If the chloride channel works correctly, the body should correct the mucus thickness etc that is the underlying issue with cf....is your trial double blind or open? Is everyone on the drug for sure, or could you be on placebo (therefore you wont notice a difference).I dont understand what the problem is with the W gene, so cant comment....you might be right.

Personally, I dont think that cfer's on the vertex or other that correct the defect will still have issues with pseudo etc.
 

dasjsmum

New member
yes, that's right. I know the G551D has an open channel but closed gate thingo that the chloride passess through. The DF508 has a closed channel and gate.The 770 opens the gate so people's bodies act the way people without cf bodies act.
<br />
<br />@miss T The 809 is supposed (hopefully) to open the channel, then they are adding the 770 which opens the 'gate'. sorry about my rudimentary understanding, but that's basically how the drug works.
<br />
<br />As far as mucus etc goes, my understanding is that cfer's have not enough mucus and it's too thick. If the chloride channel works correctly, the body should correct the mucus thickness etc that is the underlying issue with cf....is your trial double blind or open? Is everyone on the drug for sure, or could you be on placebo (therefore you wont notice a difference).I dont understand what the problem is with the W gene, so cant comment....you might be right.
<br />
<br />Personally, I dont think that cfer's on the vertex or other that correct the defect will still have issues with pseudo etc.
 

ymikhale

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

the drug is called meveol . Hopefully it will revolutionise the treatment of infections.

www.alaxia-pharma.eu/meveol</end quote></div>

This drug is still in preclinical development. It might be years before it comes to the market, assuming it does not fail in early stages
 

ymikhale

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

the drug is called meveol . Hopefully it will revolutionise the treatment of infections.

www.alaxia-pharma.eu/meveol</end quote>

This drug is still in preclinical development. It might be years before it comes to the market, assuming it does not fail in early stages
 

ymikhale

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>petersymons</b></i>
<br />
<br />the drug is called meveol . Hopefully it will revolutionise the treatment of infections.
<br />
<br />www.alaxia-pharma.eu/meveol</end quote>
<br />
<br />This drug is still in preclinical development. It might be years before it comes to the market, assuming it does not fail in early stages
 

missT

Member
Hi Joanna, the trial is blind (placebo/drug) so I have no idea what I am on. After a 48 weeks you get the real drug. I am trying to figure out how exactely our bodies react and also the disease process is halted...and to what extent.
 

missT

Member
Hi Joanna, the trial is blind (placebo/drug) so I have no idea what I am on. After a 48 weeks you get the real drug. I am trying to figure out how exactely our bodies react and also the disease process is halted...and to what extent.
 

missT

Member
Hi Joanna, the trial is blind (placebo/drug) so I have no idea what I am on. After a 48 weeks you get the real drug. I am trying to figure out how exactely our bodies react and also the disease process is halted...and to what extent.
 

saveferris2009

New member
Hey there - I think Kiel did a great job explaining how this drug works. And it sounds like you understand what's going on too.

Think link provides a FANTASTIC illustration! <a target=_blank class=ftalternatingbarlinklarge href="http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
">http://www.ptcbio.com/3.1.1_genetic_disorders.aspx
</a>
So do you have any other questions (per your PM?)? If so, let me know.

You may be on placebo, which is why you're not seeing results. The Phase II results on PCT 124 were pretty good! "Ataluren treatment was associated with reductions in cough frequency and improvements in pulmonary function tests."
 
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