Wednesday, March 30, 2011
" I have such a social little girl on my hands, whom I don't want to ever have to tell " I chose ___________________ because you have C.F." I don't want her illness to define her, or have her life filled with choices and options that were tailored to C.F. I want her to do things that everyone else said she couldn't, I want her to build lasting relationships with people who support her and lift her up while she is hurting.
Tuesday, March 29, 2011
Monday, March 28, 2011
Sunday, March 27, 2011
Friday, March 25, 2011
Just thought I'd share this nice little story about Genetech that was featured on the CFF Website. The more sponsors that we can get to do stuff like Genetech is doing, the faster we will reach our cure. If you or someone you know works for a company who might be interested in partnering with the CFF during this exciting time when we are so close to a cure, please don't hesitate to contact me or anyone at your local CFF office.
Genentech: Making CF History
Genentech has been a generous corporate partner of the Cystic Fibrosis Foundation for more than a decade, and today, its employees are more committed than ever to making CF a thing of the past.
“Participating is important to Genentech because we know that the money raised goes back to funding the research we’re so passionate about,” says Sheri Morin, senior director of sales and marketing. “It only makes sense for us to be involved!”
Genentech is both a national sponsor of Great Strides and a supporter of local walks in Northern California, where the biotech company is headquartered. During Great Strides season, Genentech encourages its 11,000 employees to join a team or make a donation.
Caring for the CF Community
Team Genentech at the Great Strides walk in San Francisco, California.
Genentech is also committed to ensuring all people living with CF and their families have access to the information and treatment support they need to stay as healthy as possible.
Many of the educational resources the CF Foundation provides to the community are made possible with significant support from Genentech.
For example, in 2010, with an unrestricted educational grant from Genentech, the Foundation established a CF education webcast series to bring the cystic fibrosis community together in a virtual forum to learn from CF experts nationwide.
In addition, the company supports research that aims to identify new ways that doctors can help patients stick to their prescribed treatment regimens.
In 2009, Genetech contributed a total of nearly $700,000 to the support the search for a cure.
“Genentech is a company that has long demonstrated it cares about the CF community,” says C. Richard Mattingly, executive vice president and chief operating officer of the CF Foundation. “We are so grateful for everything they do to support better health and a brighter future for people with cystic fibrosis.”
Wednesday, March 23, 2011
Please join the Division of Pulmonary Medicine for a day of scientific discovery and celebration. The symposium will feature scientific breakthroughs in the field of Cystic Fibrosis led by researchers throughout the country and at Cincinnati Children’s. We will also celebrate the establishment of the Thomas Boat Chair in Pulmonary Medicine and the appointment of Dr. John Clancy to the chair.
10:00 am Welcome and IntroductionRaouf S. Amin, MDDirector, Division of Pulmonary Medicine
10:15 am Cystic Fibrosis Science at Cincinnati Children’sArnold Strauss, MDPhysician-in-ChiefDirector, Cincinnati Children’s Research Foundation10:25 am Cystic Fibrosis ProgressThomas Boat, MDExecutive Associate Dean, University of Cincinnati College of MedicineCEO, University of Cincinnati Physicians10:55 am The Network Development of Cystic Fibrosis Basic andClinic Science: The Role of the FoundationRobert J. Beall, PhDPresident and CEO, Cystic Fibrosis Foundation11:35 am Breakthrough in Animal Models of Cystic Fibrosis DiseaseMichael Welsh, MDDirector, University of Iowa Cystic Fibrosis Research Center1:20 pm New Paradigms of Inflammation in Cystic FibrosisChristopher Karp, MD, PhDDirector, Cincinnati Children’s Cystic Fibrosis Foundation2:00 pm Bringing Quality Improvement to Cystic Fibrosis ClinicalCare and Applying to Clinical ResearchGerald T. O’Connor, PhD, ScDProfessor, Medicine and Health Policy and Clinical Practice atDartmouth University2:50 pm The Realities of Cystic Fibrosis Disease, Current and Future TargetsMichael Konstan, MDProfessor and Chairman of Pediatrics, Case Western Reserve UniversitySchool of Medicine, Rainbow Babies and Children’s Hospital3:30 pm Cystic Fibrosis Modulators – Are They Enough?John Paul Clancy, MDDirector, Clinical and Translational Research, Cincinnati Children’s
Monday, March 21, 2011
- Spread the word!! Share Drew's video. Here's the link: http://66roses.blogspot.com/2011/01/doin-it-for-drew.html
- Donate to our Great Strides team by clicking HERE. If you can only spare $5, we will appreciate your $5. If you can spare $500, we will appreciate that too.
- WALK WITH US!! Click on that link above that says HERE and join our team
- Hold a fundraiser for us! I know lots of creative people. Surely some of you can come up with some creative way to raise some money to help us.
- Sign up to walk with us in a city near you. Here is the link to a list of cities that Team Doin' It For Drew is walking in, as well as a link to find a walk in a city near you:http://66roses.blogspot.com/p/great-strides.html
- Ask your company if they are willing to support Cystic Fibrosis by offering matching gifts on donations, or by making a corporate donation. You never know how many people you can reach until you ask. The worst they can say is no.
- Become an Advocate for CF. It takes but a moment of your time to send a pre-drafted letter to your congressman/woman, petitioning them to get federal support for Cystic Fibrosis. Imagine if we had government funding for research instead of just the money that I'm able to raise!! Click here to read more about becoming an advocate: BECOME AN ADVOCATE
- Say a prayer for little Drew. He needs it. And if you have one to spare, I could probably use one too.
Friday, March 18, 2011
Philadephia, PA - May 21
Honolulu, HI - May 28
New London, CT - May 14
Charleston, SC - May 14
San Francisco - May 21
Vertex's Treatment for Cystic Fibrosis
Its VX-770 is the first pill designed to repair an inherited flaw based on the discovery of the gene mutation that causes the disease
By Meg Tirrell
Then in December 2009, Schaller started taking VX-770, an experimental drug from Vertex Pharmaceuticals (VRTX). Now the self-described exercise enthusiast says she can run 10 to 12 miles with barely any breathing problems. "This past year has been the coolest of my life," says Schaller, who runs the Rock CF Foundation, a nonprofit near Detroit that helps fund research on the disease, which afflicts 30,000 Americans. "It was a whole new feeling that I hadn't felt since I was a little kid."For the better part of two decades, Emily Schaller could barely take a deep breath without lapsing into coughing fits. At the age of 18 months, Schaller was diagnosed with cystic fibrosis, a hereditary disease that chokes the airways with sticky mucus, making victims susceptible to infections and other problems that often kill them before they turn 40. Schaller, now 29, says doctors told her parents, "Enjoy her, because she probably won't live long enough to graduate from high school." By age 13, she was suffering the full effects of the disease, which required her to take various medications and wear a vest that vibrates to loosen her congested lungs.
Vertex's drug is in the third and final stage of testing required for U.S. Food and Drug Administration approval. In a study released in February, Vertex reported that the drug increased breathing function by 10.5 percent over a year, reduced patients' need for antibiotics, and led to an average weight gain of seven pounds. "We were euphoric," says Robert Beall, chief executive officer of the Cystic Fibrosis Foundation in Bethesda, Md., which helped fund development of the Vertex drug. The results "suggest we've already added five years of life to these patients."
The CF gene, discovered in 1989, was one of the first found using new gene-hunting techniques that have led to better understanding of the biology of dozens of illnesses. The genetic research is helping scientists close in on treatments for cancer, diabetes, and other disorders that target glitches that occur during an individual's lifetime. VX-770 is the first pill designed to repair an inherited flaw based on the discovery of the mutation that causes the disease.
Cystic fibrosis is caused by mutations to a gene that, when working properly, creates proteins to help transport fluid in and out of cells. When the gene is defective and the proteins don't work, chloride, a component of salt, can't travel freely across cell membranes. That limits the flow of fluid outside the cell, causing mucus there to dry up and clog airways, allowing bacteria to gather.
VX-770, designed for a mutation carried by 4 to 5 percent of CF patients, enhances the function of the protein, creating a gateway that allows chloride to pass through the cell wall. About 90 percent of people with the disease, though, also suffer from a thornier problem: The protein never makes it to the cell wall. So Vertex is developing a second drug—VX-809—that helps transport the protein to the membrane. "To have optimal benefit, you need two compounds," says Eric Olson, head of Vertex's cystic fibrosis unit. "One to get the protein to the surface, and one to boost its activity once it's out there."
VX-809 is still in a so-called Phase II test as is the combined treatment. While VX-770 could win approval as early as 2012, any formula with both drugs likely won't be available for several years. "The combination therapy down the line is going to be the real driver," says Brian Skorney, an analyst with brokerage ThinkEquity in New York. Such a drug, he says, could eventually generate $1 billion a year in sales. On Feb. 23, when Vertex reported the VX-770 data, the company's Nasdaq-traded shares jumped 15 percent.
The medicines are the fruit of more than a decade of research. After the CF gene was discovered, scientists initially pursued gene therapy, trying to introduce a normal gene into cells to replace the faulty one. When that didn't work, the focus shifted to medicines such as VX-770 that could simply help defective proteins function properly. The Cystic Fibrosis Foundation asked drugmakers if they would screen compounds in their chemical libraries to determine whether one might affect the protein that helps move fluids in and out of cells.
Aurora Biosciences in San Diego agreed to test thousands of chemicals, eventually yielding compounds that led to the Vertex therapies. Vertex bought Aurora in 2001 for about $600 million and maintained the relationship with the CF Foundation, which supplied $75 million in funding for the program. "Discovering the genetic cause of a disease can lead you toward a therapy you never would have thought of otherwise," says Francis Collins, one of the CF gene's discoverers and now director of the National Institutes of Health.
The cystic fibrosis program, once an uncertain part of Vertex's future after the Aurora acquisition, has transformed the company into a diversified drugmaker, says Phil Nadeau, an analyst with Cowen & Co. in New York. Vertex is awaiting regulatory approval for Telaprevir, a medicine for hepatitis C that Nadeau estimates could draw more than $4 billion in annual revenue. "Now people see [Vertex] as more diversified," Nadeau says. "VX-770 is a second pillar the company can stand on."
The bottom line: If approved, Vertex's new cystic fibrosis pill will be the first treatment developed from genetic research for an inherited mutation.
If you missed my post on why this drug is good news for us, CLICK HERE to check it out.
That's about all the energy I have for this evening. It was a great week in our house. The nice weather allowed us to get outside for many hours on many occasions this week which was literally a breath of fresh air. Hope you'll sign up to walk with us!
Monday, March 14, 2011
Thursday, March 10, 2011
Defective protein production with premature termination of CFTR production. Class 1 mutations produce few or no functioning CFTR chloride channels.
Class 2 mutations
Defective trafficking of CFTR so that it does not reach the apical surface membrane where it can function.
Class 3 mutations
Defective regulation of CFTR even though it is able to reach the apical cell surface.
Class 4 mutations
CFTR reaches the apical surface but chloride transport through the channel is defective.
Class 5 mutations
Reduced production of functional CFTR. A small amount of functional CFTR may reach the surface.
So a couple of weeks ago I posted this article about a new and exciting drug which corrects the basic defect of one of the mutations of CF. The study was done on a mutation known as G551D. Well, mutation G551D falls into Class 3 of the mutation, the ones that have trouble regulating chloride. The new drug that should hit the market in later 2011 or early 2012 corrects this regulation problem, getting chloride in and out of the cells the way a normal persons would. Mutation R560T, one of Drew's mutation, also falls into Class 3. (I'll give you a moment to get excited if you can see where i'm going with this).....