The annual CFF Meeting that Tricia and I attended last week took place at Inspire Pharmaceuticals, Inc. which recently initiated a second Phase 3 clinical trial evaluating denufosol tetrasodium inhalation solution for the treatment of Cystic Fibrosis. A few of the Inspire doctors/scientists spent several minutes explaining this new drug.
The basic issue that CF presents in the lungs is that it causes the thin layer of hydration that keeps normal lungs wet to dry up and become thick and sticky. Normally, this mucus is such that it is able to catch most dust, germs and other foreign stuff in your lungs and quickly move it out of your body. With CF, the mucus is so thick that most of what you breathe in never makes it back out. The mucus traps a lot of junk, and slowly becomes thicker and thicker. This causes a few major problems (and, understand that the following is an extremely simplistic way of explaining...only because it's ultimately too complicated for my brain to comprehend):
1) The thick mucus traps germs and bugs and bacteria and fungus which, if given enough time, can result in some really nasty/harmful sickness. This is why most CFer's become very sick fairly often.
2) The mucus also slowly begins to block the lungs natural ability to both absorb the O2 and get rid of the CO2 from the air being breathed in. This is why most CFer's have difficulty breathing.
Denufosol enhances the hydration and clearance of mucus from the lungs by helping chloride move across the cell membranes of the lungs. This new trial, named TIGER-2 (Transport of Ions to Generate Epithelial Rehydration), is enrolling 350 people with CF aged 5 years and older in the United States and Canada and at additional international sites as the trial proceeds. The trial aims to demonstrate improved lung function and decreased pulmonary exacerbations, and measure the need for other CF medications and quality of life.
So far, the results are extremely promising!
Inspire is one of the few pharmaceutical companies in the US that was organized to specifically develop drugs for Cystic Fibrosis. The CFF has been partnering with Inspire since it's beginning, and is one of the companies that receives funding from the CFF, which, of course, comes from people like you who generously give to help find a cure.
Inspire explained that, after Denufosol hits the market (assuming the trials go well and it is approved for manufacturing and distribution), their next major goal is to find the cure. But, the only way that they'll ever get that far is with additional financial support from the people who give to the CFF.
Please, if you haven't yet, consider joining us as we search for a cure by Donating to the Cystic Fibrosis Foundation.
Thanks!
Nate
16 comments:
Wow, I am the FIRST to comment. What a honor! :) All the happiness to you and your family Nate.
Annie
I am very exeicted about this drug...I am hoping it comes out on the market soon so it can help my child with CF...shes almost 4 and well in the last year she has had more lung involuement and yeah scary stuff!
Thanks for sharing this news and explaining it!
I heard about this new drug that doesn't just treat symptoms but CF itself. Excellent news!
You said that they were taking 350 to test it out... do they already have the 350 or is there a place where you can put in to be in the testing phase?
Very encouraging news!
How very exciting!!!
Hi Nate,
Can you please post somewhere the man's blog that is losing weight for Tricia? I have misplaced his blog and I would love to checkout his progress. Thanks so much!
If you want to join the trials, here is a link to the info:
http://clinicaltrials.gov/ct2/show/NCT00625612?term=denufosol&rank=3&show_locs=Y#locn
If that doesn't work, type in clinicaltrials.gov and you can search for any clinical trials involving CF
Wow...exciting!
how exciting & i love the name of the company as well! hope you have a lovely relaxing weekend, xx
That was very informative about both the disease and the company. We are praying for you guys.
Just wanted to share this local story from my hometown, Louisville Kentucky.
http://www.wave3.com/Global/story.asp?S=8768000&nav=menu31_3
I've followed your blog from the beginning & wish you and your family the best.
Shannon
Thanks for some reader-friendly info about CF, its good to know a little bit more about it. I pray for your family...god bless
What an answer to prayer.
The guy doing the 65lb for Patty Sue has a blog is at http://www.65lbs.blogspot.com/
Myra
I'm not sure if this is the same treatment the University of Western Ontario (located in London Ontario Canada) discovered.
Here is the transcipt
Breakthrough holds promise for cystic fibrosis this is from the UWO website
By Communications Staff
Wednesday, July 30, 2008
A University of Western Ontario professor studying cystic fibrosis (CF) has successfully corrected the defect which causes the overproduction of intestinal mucous in mice.
This discovery by Richard Rozmahel, an associate professor of Biochemistry, Pediatrics and Oncology at the Schulich School of Medicine & Dentistry and scientist with the Lawson Health Research Institute, has clear implications to understanding and treating this facet of the disease in humans.
Cystic fibrosis is a fatal, genetic disease affecting 70,000 children and adults worldwide, and characterized by an overproduction of mucous in the lungs and digestive system. Compared to the 1960's, when most patients died by age four, the median age of survival is now 37 years.
Rozmahel and his colleagues are identifying secondary genes that could contribute to CF, and measuring their impact on the disease. More specifically, they are investigating the potential of a gene found in mice, mCLCA3, which is similar to one in humans that exhibits abnormal levels in CF. The mCLCA3 gene is expressed by cells that produce and secrete mucous.
The researchers discovered that mCLCA3 plays an important role in the property of mucous, thereby allowing it to be cleared rather than result in the blockages that underlie CF. By correcting the abnormal levels of mCLCA3 in CF mice they were able to overcome the mucous lesions.
Whereas CF mice normally do not survive more than four weeks as a consequence of the mucous disease, the animals where mCLCA3 levels were corrected could live a normal lifespan.
"It's my hope to understand what is causing the exaggerated mucous production and secretion in CF patients," says Dr. Rozmahel. "From there, we can figure out ways to correct it."
From the London Free Press
London scientists working with mice have discovered that by correcting an abnormal gene linked to cystic fibrosis the mouse has a normal two-year lifespan instead of dying at four or six weeks.
The hope is the research will eventually lead to a new treatment for the fatal genetic disorder that hits one in 3,600 Canadians.
"The important aspect of this will be the translation of this research from the mouse model back into the clinical setting into actual cystic fibrosis patients," said Dr. Richard Rozmahel of the Lawson Research Institute.
Rozmahel's research team looked at more than 30,000 genes to find ones different in a normal mouse from one in cystic fibrosis mice.
One gene with a different expression in cystic fibrosis mice was mCLCA3, a gene similar to one found in humans with cystic fibrosis.
In the CF mice, it was only being expressed at one-third the level of healthy mice.
When the Lawson scientists brought the gene up to normal expression, the mucous blockages in the lungs and intestines were reduced.
"They could survive normally," said Rozmahel.
The next step in their research will be to pinpoint exactly how the gene CLCA3 functions, he said.
"We have shown CLCA3 is important to cystic fibrosis but the function of CLCA3 is not fully understood," said Rozmahel.
"Is it acting on the properties of the mucous to allow the mucous to be cleared better, is it working on the hydration of the mucous so that mucous has more fluid in it so it would be cleared better, or is it acting to decrease secretion of the mucous by these cells?"
Individuals born with cystic fibrosis used to survive only a few years, but drug therapies and lung transplants have pushed the median life expectancy to 37.
The most obvious problem for individuals with the disease is the build of mucous in the lungs, which provides a breeding ground for bacteria.
"In cystic fibrosis the mucous is not able to be cleared because it is so thick and sticky," Rozmahel said.
"If we were able to promote the clearance of mucous we would be able to correct at least part of the problem."
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