You are using an out of date browser. It may not display this or other websites correctly.
You should upgrade or use an alternative browser.
You should upgrade or use an alternative browser.
Can someone help me
- Thread starter anonymous
- Start date
Scarlett81
New member
Yeah I posted about it a few weeks ago, and its a very interesting article. The problem was people couldn't access it b/c you need to be a member of Forbes.com or something like that. But-you can be a member of Forbes.com for free, and click an option so you don't receive any junk emails either.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Scarlett81
New member
Yeah I posted about it a few weeks ago, and its a very interesting article. The problem was people couldn't access it b/c you need to be a member of Forbes.com or something like that. But-you can be a member of Forbes.com for free, and click an option so you don't receive any junk emails either.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Scarlett81
New member
Yeah I posted about it a few weeks ago, and its a very interesting article. The problem was people couldn't access it b/c you need to be a member of Forbes.com or something like that. But-you can be a member of Forbes.com for free, and click an option so you don't receive any junk emails either.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Just go to their website, click on archives (or something similar to that, like a recent articles search) and scroll down to health related articles. Then you'll find the one on CF. When you click to read it it says you have to be a member. Just fill in the info, it takes a minute and its free.
Sorry to disappoint, but the Forbes article was about what is being researched and not new meds or treatments that have been released to the public. The stuff mentioned in the article in not yet available. They've been working on this for years and will likely work on it for several more.
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
Sorry to disappoint, but the Forbes article was about what is being researched and not new meds or treatments that have been released to the public. The stuff mentioned in the article in not yet available. They've been working on this for years and will likely work on it for several more.
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
Sorry to disappoint, but the Forbes article was about what is being researched and not new meds or treatments that have been released to the public. The stuff mentioned in the article in not yet available. They've been working on this for years and will likely work on it for several more.
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
I have posted my own thoughts on the article and an abbreviated version of the Forbes article <a target=_blank class=ftalternatingbarlinklarge href="http://livingwellwithcf.blogspot.com/2006/09/undeserving-adults.html">here on my blog</a>.
I tend not to get overly excited about the new drugs or pin my hopes on them. For now, tobra, merrem and fortaz do the trick to get me over the hump when I need it--as was recently the case. I know that new drugs can't be cranked out like cotton candy, and I don't expect this to happen. I pray for a cure, but I'm not holding my breath (ha!) waiting for it. God gives me what I need to get through in my current state of being. The doctors, treatments, etc. have all been integral in getting me to today. And if I should happen to have more tomorrows added by some other means, then that will be a blessing too.
My husband recently asked me if I thought there would be a cure in my lifetime and I said "if I live to be 75, maybe!" Incidentally that's another 50 or so years that I'd have to wait.
Below is a response from a doctor with CF and his thoughts on the drugs and the Forbes article.
<i>As a CF adult, back around the days of the gene discovery, I remember something that was said: "We will have the cure in 10-15 years." Well, for anyone doing the math, that would have been around 1999-2004 and we are still waiting. Going into medicine, learning about genetic engineering, drug development, etc, I remember about 5 years after the discovery saying, they will probably have a treatment, something to control the disease, not to cure it. Cure can be an interesting word, what do we expect from a cure, to stop the disease (what I would consider a cure as a physician but not probably as a patient with my current lung function), or something to reverse everything that has happened to us (hmmm, sorry but don't see this any time soon considering the current state of medicine, not just CF medicine)? Unfortunately, the protein that comes from the "defective" CF gene has proven entirely more complicated than ever imagined. It isn't the only "culprit." There are also many proteins that the CFTR interacts with that play a role in the disease. Hence why one person with two copies of delta F508 (the most common gene) can have such different disease courses.
I remember when UTP was talked about . . . it was supposed to cause minimum side effects but great effects . . . unfortunately it hasn't gone far. CTX was another drug trialed but seems to have disappeared. A drug used for other things . . . amiloride (I think) really didn't go anywhere.
They are finally beginning to understand the protein and hence more about CF itself in the lungs and other organs. The complicated puzzle is slowly being put together, albeit the amount of time is frustrating. So for the moment we have to put our "stock" in tobramyacin, other antibiotics, azithromycin, sodium chloride treatments to buy us all more time for the "cure" to come about. Truly, the more we find out in medicine, the more confusing things get.
All I know is that the rate of discovery and the hope is light years ahead of when I was a child. After all, the disease really has been around for hundreds of years but didn't even get a name until the 1940's. Just over sixty years ago, most children didn't make it into their teens. Now many make it into the 30's and 40's. Twenty years ago the median age was maybe into the 20's. I never thought I would make it through medical school and residency at that time. The hope of things continuing to improve was all that got me through, and it is what continues to give me hope today, it is all I have!</i>
here's the entire article<br>
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."
here's the entire article<br>
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."
here's the entire article<br>
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."
<br>
<b>Health<br>
Breathing Easier<br>
Mary Ellen Egan 09.18.06</b><br>
<br>
Two new drugs may be the first to halt the progression of a deadly
disease.<br>
Tanner Buck is 6 years old. He will be lucky to be alive at age 40.
Buck, a wiry little boy with long, lank cornsilk hair, is one of
30,000 young Americans with the fatal genetic disease known as
cystic fibrosis. It drowns the lungs in abnormally thick, sticky
mucus and devastates the pancreas, liver and other organs.<br>
In between soccer games, tree-climbing and other boyhood pursuits
in his hometown of Luck, Wis., Tanner spends part of each day just
fighting to breathe. His current treatments are difficult and
time-consuming--and aim only at easing the severe symptoms of
CF.<br>
<br>
Now two publicly held biotechs--Inspire Pharmaceuticals of Durham,
N.C. and Vertex Pharmaceuticals in Cambridge, Mass.--are testing
new compounds that are the first to target the underlying defects
that threaten the life of Tanner Buck. The boy took part in a
90-patient trial of Inspire's new drug in the spring and hopes to
reenroll; his mom says the main upside for him is that the
experimental regimen lets him watch an extra 45 minutes of TV each
day.<br>
<br>
Without a new breakthrough, Tanner squeezes life in between daily
therapy. To keep his lungs clear, he puts in half an hour twice a
day wearing a nebulizer mask and inhaling a mucus thinner
(Mucomyst) and a compound that opens up his airways (Albuterol).
While inhaling the compounds, he wears a vibrating vest that
loosens the mucus in his lungs, letting him expel it by
coughing.<br>
<br>
In addition to lung therapy Tanner and other people with CF take
enzyme pills with meals and snacks to free up the clogged channels
that normally carry enzymes to the intestines to digest food;
otherwise patients risk malnutrition. The existing regimens have
extended the life span of CF patients from 14 years in 1969 to 37
years today.<br>
<br>
Now researchers hope new compounds may halt the deadly progression
of cystic fibrosis. Inspire's drug, denufosol tetrasodium, emerged
in the early 1990s from research by Dr. Richard Boucher at the
University of North Carolina at Chapel Hill.<br>
The basic malfunction in CF involves a defect in the epithelial
cells (which cover and protect various organs, as well as the skin
and the digestive tract), particularly the cells lining the airways
of the lungs. These lining cells have channels on their outside
surface: One lets sodium ions (small charged molecules) flow into
the cell, while another pushes chloride ions out of the cell and
into the mucus on the airway surface.<br>
<br>
In CF patients the outflow of chloride is hindered, the inflow of
sodium unrestrained. This disrupts the delicate balance of salt and
water on the surface of the lungs' airways, preventing a normal
coating of fluid and mucus inside the lungs, pancreas and other
organ passageways.<br>
<br>
Patients with CF produce a defective form of a protein called CFTR
(cystic fibrosis transmembrane conductance regulator) that screws
up the inflow/outflow process. Boucher long suspected that
epithelial cells have an alternative chloride transport channel;
stimulate the right receptor, and he might jump-start the backup
channel.<br>
<br>
In 1991, after 15 years of searching, Boucher and his UNC lab coats
discovered a nucleotide called uridine triphosphate (UTP) that
activates this alternative chloride passageway. UTP turns on P2Y2
receptors--protein molecules on the surfaces of mucosal cells.
These receptors prompt the cells to secrete salt (which draws water
onto the airway surface) and prompt the hairlike cilia on cell
membranes to beat faster to sweep mucus out of the airways and into
the trachea, where it is disposed of by swallowing or coughing.<br>
<br>
UNC licensed the breakthrough to Inspire in 1995. Inspire raised $9
million to develop a compound that would mimic UTP, tapping venture
capitalists at Burr, Egan Deleage & Co. (now Alta
Communications), Domain Associates and Medical Science Partners.
The firm's scientists spent five years developing a synthetic
version of UTP, now called denufosol, which can be inhaled through
a nebulizer. Compound in hand, the company went public in August
2000; since then its shares have dropped 73%, in part because of
higher R&D costs.<br>
<br>
Vertex's compound, VX-770, would go a step further than Inspire's
drug. It tries to restore function not only to the lungs but also
to other organs affected by CF. VX-770 resulted from a five-year
collaboration between Vertex and the Cystic Fibrosis Foundation.
Vertex landed $13.3 million from the group to work on the compound
and received a total of $40 million in foundation grants (although
the organization doesn't own an equity stake in Vertex).<br>
<br>
Instead of switching on a backup chloride channel like Inspire's
drug, VX-770 works directly on the faulty CFTR channel. It targets
parts of the CFTR protein involved in the opening and closing of
the CFTR channel, propping open the protein to afford a more normal
flow of chloride.<br>
<br>
In young patients the pancreas and digestive tract also may benefit
from the drug, says Vertex Chief Medical Officer John Alam, because
the CFTR function is suspected to play a role there as well.<br>
<br>
"I think it's a terrific idea," says UNC's Boucher,
although he allows that VX-770 is only in early-stage trials.
"There's hope now that you could take a pill and fix CF in the
lungs, pancreas and other organs," he says. Patients may
benefit from both the Vertex compound and Inspire's denufosol, he
adds. "In college kids, who typically have significant amounts
of lung damage, denufosol can keep the healthy portions of the lung
healthy."<br>
<br>
If both approaches win federal approval, Inspire's drug would hit
the market several years ahead of Vertex's. In July Inspire began
enrolling patients for late-stage trials. Preliminary results could
come as early as 2008. When 6-year-old Tanner Buck took part in a
28-day trial of the Inspire remedy last May, his parents watched
for signs of improvement but saw none; it may be that Tanner was
unknowingly given a placebo in the blinded clinical trial.<br>
<br>
"He hasn't asked why he has CF or what it means for him,"
says his mom, Julie Buck. "And since he's so little, we don't
see a point in telling him the details. It isn't fair to tell him
he may die from the disease someday when we don't know what's going
to happen in the future." She adds, adamantly: "We're
hopeful that he will live well into adulthood."