April 2, 2009 7:00 pm ~ 9:00 pm
Pre-Event Cocktail Reception
The Pelican Bar and Grille Hyatt Regency Pier Sixty Six
2301 S.E. 17th Street Causeway, Ft. Lauderdale, FL
Please RSVP to Vicki Swain by March 26th
954.739.5006 vswain@cff.org
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March 25, 2009
March 24, 2009
CFF In The News
“Patient Power”
Forbes showcased the Foundation’s leadership and influence in driving drug development for a rare disease. The story highlighted the Foundation’s progress through the eyes of one CF patient, Emily Schaller. She said of the Foundation: “They’re the reason I’m alive.”
Forbes, September 15, 2008
“Breathing Easier”
A cover story in Chemical and Engineering News highlighted the Foundation’s drug development
efforts. The story notes that a stream of new drugs that treat the core defect in CF could reach patients in the next few years, thanks to the ambitious efforts of the Foundation.
C&EN, September 1, 2008
“PTC Therapeutics Announces $25 Million Award From CFFT for Development of PTC124”
PTC Therapeutics, Inc. and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate
of the CF Foundation, announced the expansion of an existing collaboration to support the development of PTC124, a new investigational drug for the treatment of CF. PTC will receive up to $25 million from CFFT in support of clinical trials.
CTP Therapeutics, July 16, 2008
“Transave Announces Positive Phase II Results for Once daily ArikaceTM in the Treatment of CF Patients Who Have Pseudomonas Lung Infections”
Transave, Inc., announced positive Phase 2 results for its lead investigational drug, Arikace™.
The inhaled compound is being developed for the treatment of CF patients who have lung infections due to the bacterium Pseudomonas aeruginosa. Cystic Fibrosis Foundation Therapeutics, Inc., provided a $1.7 million award to support the development of Arikace.
Transave, June 13, 2008
“Inspire Announces Positive Results From Phase III Clinical Trial of Denufosol”
Inspire Pharmaceuticals, Inc., announced positive results from a Phase 3 clinical trial of denufosol, an inhaled therapy for the treatment of CF. Denufosol is designed to improve chloride
transport in the airways and enhance mucus hydration and clearance. The data from the trial is
particularly encouraging because it brings the CF community one step closer to a novel treatment that addresses the basic defect in CF.
Inspire, June 6, 2008
“Drug Makers Turning to Nonprofits for Cash”
The Boston Globe featured a story on the front-page of its business section about the Foundation’s efforts to develop effective therapies for CF. It mentioned the Foundation has awarded $300 million to biotech companies for this purpose, and announced an expanded $37.7 million collaboration with EPIX Pharmaceuticals.
The Boston Globe, April 7, 2008
“A Healthy Gamble for CF”
An article in BusinessWeek online showcased the Foundation’s unique business model and promising investments in biotech companies. The story highlighted the positive early results of an ongoing clinical trial of VX-770, an oral drug that targets the core defect in CF being developed by Vertex Pharmaceuticals.
BusinessWeek, April 3, 2008
“Vertex Reports Positive Results for Phase II Clinical Trial of VX-770”
Data from a Phase 2a clinical trial in CF patients who carry the G551D gene mutation showed that an oral dose of VX-770 for 14 days resulted in improved lung function, improved nasal potential difference measures, and sweat chloride levels. Vertex reported that, “while these are early data, it is unprecedented for an investigational oral compound for the treatment of CF
to have such a marked effect on multiple measures of CF disease activity.”
Forbes, March 27, 2008
“Landmark Clinical Trials Initiated for Drugs Designed to Repair Defective CF Protein”
In a major milestone for the Foundation, two drugs that target the core defect in CF have entered clinical trials. Vertex Pharmaceuticals, in collaboration with the Foundation, began a Phase 2 trial for VX-770 in late 2007, and in early 2008, began evaluating VX-809 in healthy volunteers. The two drugs were developed as a result of an historic collaboration, which harnesses high-throughput screening for drug discovery.
Vertex, January 2008
“Breath of Fresh Air for Cystic Fibrosis Drug Pipeline”
Pharmaceutical Approvals Monthly, a leading biotech publication, ran a story on the success of the Foundation’s drug discovery and development pipeline. The outlook for CF drug development is “a very different story than even five years ago,” said Robert J. Beall, Ph.D., president and CEO of the Foundation.
Pharm Approvals Monthly, December 2007
“Foundation Launches New Collaboration With AMRI”
CFFT, the nonprofit affiliate of the CF Foundation, announced a four-year research collaboration with AMRI, worth up to $23.7 million. The project aims to identify novel drugs that address the core CF defect.
AMRI, October 4, 2007
“Venture Philanthropy on the Rise”
An article in CenterWatch Monthly reported on the trend of venture philanthropy nationwide and identified the CF Foundation as the charitable organization spending the most on drug research and development. In fact, the Foundation will spend $48 million on drug research in 2007, three times more than any other disease foundation in the country.
CenterWatch, August 2007
“$22M to Help FoldRx Develop Drug for Cystic Fibrosis”
An article in The Boston Globe featured the CF Foundation’s new $22 million biotech collaboration with FoldRx Pharmaceuticals to discover and develop new compounds to treat the core defect in CF. In 2007 alone, the story said, the Foundation has earmarked $48 million for drug research with biotech companies as it seeks to stock its drug pipeline with promising treatments.
The Boston Globe, August 13, 2007
“Gilead Announces Expanded Access Program for Aztreonam Lysine for Inhalation for Patients With Cystic Fibrosis”
Gilead Sciences, Inc. announced the launch of an early access program Aug. 1, 2007 to provide its investigational therapy, aztreonam lysine for inhalation, to patients with cystic fibrosis and Pseudomonas aeruginosa infection who have limited treatment options and are at risk for disease progression.
Gilead, July 9, 2007
“A Different Sort of Venture”
The Foundation was prominently featured on National Public Radio’s Marketplace program. The
show, heard by 4.5 million listeners, credited the Foundation for bringing promising CF drugs to
market and helping people with CF live longer.
NPR, June 8, 2007
Forbes showcased the Foundation’s leadership and influence in driving drug development for a rare disease. The story highlighted the Foundation’s progress through the eyes of one CF patient, Emily Schaller. She said of the Foundation: “They’re the reason I’m alive.”
Forbes, September 15, 2008
“Breathing Easier”
A cover story in Chemical and Engineering News highlighted the Foundation’s drug development
efforts. The story notes that a stream of new drugs that treat the core defect in CF could reach patients in the next few years, thanks to the ambitious efforts of the Foundation.
C&EN, September 1, 2008
“PTC Therapeutics Announces $25 Million Award From CFFT for Development of PTC124”
PTC Therapeutics, Inc. and Cystic Fibrosis Foundation Therapeutics, Inc., the nonprofit affiliate
of the CF Foundation, announced the expansion of an existing collaboration to support the development of PTC124, a new investigational drug for the treatment of CF. PTC will receive up to $25 million from CFFT in support of clinical trials.
CTP Therapeutics, July 16, 2008
“Transave Announces Positive Phase II Results for Once daily ArikaceTM in the Treatment of CF Patients Who Have Pseudomonas Lung Infections”
Transave, Inc., announced positive Phase 2 results for its lead investigational drug, Arikace™.
The inhaled compound is being developed for the treatment of CF patients who have lung infections due to the bacterium Pseudomonas aeruginosa. Cystic Fibrosis Foundation Therapeutics, Inc., provided a $1.7 million award to support the development of Arikace.
Transave, June 13, 2008
“Inspire Announces Positive Results From Phase III Clinical Trial of Denufosol”
Inspire Pharmaceuticals, Inc., announced positive results from a Phase 3 clinical trial of denufosol, an inhaled therapy for the treatment of CF. Denufosol is designed to improve chloride
transport in the airways and enhance mucus hydration and clearance. The data from the trial is
particularly encouraging because it brings the CF community one step closer to a novel treatment that addresses the basic defect in CF.
Inspire, June 6, 2008
“Drug Makers Turning to Nonprofits for Cash”
The Boston Globe featured a story on the front-page of its business section about the Foundation’s efforts to develop effective therapies for CF. It mentioned the Foundation has awarded $300 million to biotech companies for this purpose, and announced an expanded $37.7 million collaboration with EPIX Pharmaceuticals.
The Boston Globe, April 7, 2008
“A Healthy Gamble for CF”
An article in BusinessWeek online showcased the Foundation’s unique business model and promising investments in biotech companies. The story highlighted the positive early results of an ongoing clinical trial of VX-770, an oral drug that targets the core defect in CF being developed by Vertex Pharmaceuticals.
BusinessWeek, April 3, 2008
“Vertex Reports Positive Results for Phase II Clinical Trial of VX-770”
Data from a Phase 2a clinical trial in CF patients who carry the G551D gene mutation showed that an oral dose of VX-770 for 14 days resulted in improved lung function, improved nasal potential difference measures, and sweat chloride levels. Vertex reported that, “while these are early data, it is unprecedented for an investigational oral compound for the treatment of CF
to have such a marked effect on multiple measures of CF disease activity.”
Forbes, March 27, 2008
“Landmark Clinical Trials Initiated for Drugs Designed to Repair Defective CF Protein”
In a major milestone for the Foundation, two drugs that target the core defect in CF have entered clinical trials. Vertex Pharmaceuticals, in collaboration with the Foundation, began a Phase 2 trial for VX-770 in late 2007, and in early 2008, began evaluating VX-809 in healthy volunteers. The two drugs were developed as a result of an historic collaboration, which harnesses high-throughput screening for drug discovery.
Vertex, January 2008
“Breath of Fresh Air for Cystic Fibrosis Drug Pipeline”
Pharmaceutical Approvals Monthly, a leading biotech publication, ran a story on the success of the Foundation’s drug discovery and development pipeline. The outlook for CF drug development is “a very different story than even five years ago,” said Robert J. Beall, Ph.D., president and CEO of the Foundation.
Pharm Approvals Monthly, December 2007
“Foundation Launches New Collaboration With AMRI”
CFFT, the nonprofit affiliate of the CF Foundation, announced a four-year research collaboration with AMRI, worth up to $23.7 million. The project aims to identify novel drugs that address the core CF defect.
AMRI, October 4, 2007
“Venture Philanthropy on the Rise”
An article in CenterWatch Monthly reported on the trend of venture philanthropy nationwide and identified the CF Foundation as the charitable organization spending the most on drug research and development. In fact, the Foundation will spend $48 million on drug research in 2007, three times more than any other disease foundation in the country.
CenterWatch, August 2007
“$22M to Help FoldRx Develop Drug for Cystic Fibrosis”
An article in The Boston Globe featured the CF Foundation’s new $22 million biotech collaboration with FoldRx Pharmaceuticals to discover and develop new compounds to treat the core defect in CF. In 2007 alone, the story said, the Foundation has earmarked $48 million for drug research with biotech companies as it seeks to stock its drug pipeline with promising treatments.
The Boston Globe, August 13, 2007
“Gilead Announces Expanded Access Program for Aztreonam Lysine for Inhalation for Patients With Cystic Fibrosis”
Gilead Sciences, Inc. announced the launch of an early access program Aug. 1, 2007 to provide its investigational therapy, aztreonam lysine for inhalation, to patients with cystic fibrosis and Pseudomonas aeruginosa infection who have limited treatment options and are at risk for disease progression.
Gilead, July 9, 2007
“A Different Sort of Venture”
The Foundation was prominently featured on National Public Radio’s Marketplace program. The
show, heard by 4.5 million listeners, credited the Foundation for bringing promising CF drugs to
market and helping people with CF live longer.
NPR, June 8, 2007
Labels:
Education
What Is Cystic Fibrosis?
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States (70,000 worldwide). A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that:
• clogs the lungs and leads to life-threatening lung infections; and
• obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
In the 1950s, few children with cystic fibrosis lived to attend elementary school. Today, advances in research and medical treatments have further enhanced and extended life for children and adults with CF. Many people with the disease can now expect to live into their 30s, 40s and beyond.
Symptoms of Cystic Fibrosis
People with CF can have a variety of symptoms, including:
• very salty-tasting skin;
• persistent coughing, at times with phlegm;
• frequent lung infections;
• wheezing or shortness of breath;
• poor growth/weight gain in spite of a good appetite; and
• frequent greasy, bulky stools or difficulty in bowel movements.
Statistics
• About 1,000 new cases of cystic fibrosis are diagnosed each year.
• More than 70 percent of patients are diagnosed by age two.
• More than 44 percent of the CF patient population is age 18 or older.
• Currently, the predicted median age of survival is 37 years, more than double what it was 25 years ago.
Testing for Cystic Fibrosis
Genetic Carrier Testing
More than 10 million Americans are symptomless carriers of the defective CF gene. Genetic carrier testing can help detect carriers, who could pass CF onto their children. To have cystic fibrosis, a child must inherit one copy of the defective CF gene from each parent. Each time two carriers of the CF gene have a child, the chances are:
• 25 percent the child will have CF;
• 50 percent the child will carry the CF gene but not have CF; and
• 25 percent the child will not carry the gene and not have CF.
Newborn Screening
Newborns screened for cystic fibrosis can benefit from early diagnosis and treatment, which can:
• Improve growth;
• Improve lung function;
• Reduce hospital stays; and
• Add years to life.
While newborn screening is not a definitive diagnostic test for cystic fibrosis, it may lead to tests that can rule out or confirm a CF diagnosis. The CF Foundation and the Centers for Disease Control and Prevention recommend screening for cystic fibrosis for all newborns.
Sweat Testing
If a person exhibits symptoms of CF, a doctor may order a sweat test. This simple, painless test is the best way to diagnose cystic fibrosis. It measures the concentration of salt in a person’s sweat, and a high salt level indicates CF. Sweat tests should be done at a CF Foundation-accredited care center where strict guidelines help ensure accurate results.
Treatments for Cystic Fibrosis
People living with cystic fibrosis must follow a regular treatment routine to stay healthy and maintain optimal lung function. Most often, treatment for CF begins with techniques to trigger strong coughs that help loosen and clear thick mucus from the airways. Nutrition and drug therapies are also important for children and adults with cystic fibrosis.
The Cystic Fibrosis Foundation
The mission of the Cystic Fibrosis Foundation, a nonprofit donor-supported organization, is to assure the development of the means to cure and control CF and to improve the quality of life for those with the disease.
Thanks to the dedication and financial backing of our supporters—patients, families and friends, medical professionals, researchers, volunteers, individual donors, corporations and staff—we are making a difference.
• clogs the lungs and leads to life-threatening lung infections; and
• obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.
In the 1950s, few children with cystic fibrosis lived to attend elementary school. Today, advances in research and medical treatments have further enhanced and extended life for children and adults with CF. Many people with the disease can now expect to live into their 30s, 40s and beyond.
Symptoms of Cystic Fibrosis
People with CF can have a variety of symptoms, including:
• very salty-tasting skin;
• persistent coughing, at times with phlegm;
• frequent lung infections;
• wheezing or shortness of breath;
• poor growth/weight gain in spite of a good appetite; and
• frequent greasy, bulky stools or difficulty in bowel movements.
Statistics
• About 1,000 new cases of cystic fibrosis are diagnosed each year.
• More than 70 percent of patients are diagnosed by age two.
• More than 44 percent of the CF patient population is age 18 or older.
• Currently, the predicted median age of survival is 37 years, more than double what it was 25 years ago.
Testing for Cystic Fibrosis
Genetic Carrier Testing
More than 10 million Americans are symptomless carriers of the defective CF gene. Genetic carrier testing can help detect carriers, who could pass CF onto their children. To have cystic fibrosis, a child must inherit one copy of the defective CF gene from each parent. Each time two carriers of the CF gene have a child, the chances are:
• 25 percent the child will have CF;
• 50 percent the child will carry the CF gene but not have CF; and
• 25 percent the child will not carry the gene and not have CF.
Newborn Screening
Newborns screened for cystic fibrosis can benefit from early diagnosis and treatment, which can:
• Improve growth;
• Improve lung function;
• Reduce hospital stays; and
• Add years to life.
While newborn screening is not a definitive diagnostic test for cystic fibrosis, it may lead to tests that can rule out or confirm a CF diagnosis. The CF Foundation and the Centers for Disease Control and Prevention recommend screening for cystic fibrosis for all newborns.
Sweat Testing
If a person exhibits symptoms of CF, a doctor may order a sweat test. This simple, painless test is the best way to diagnose cystic fibrosis. It measures the concentration of salt in a person’s sweat, and a high salt level indicates CF. Sweat tests should be done at a CF Foundation-accredited care center where strict guidelines help ensure accurate results.
Treatments for Cystic Fibrosis
People living with cystic fibrosis must follow a regular treatment routine to stay healthy and maintain optimal lung function. Most often, treatment for CF begins with techniques to trigger strong coughs that help loosen and clear thick mucus from the airways. Nutrition and drug therapies are also important for children and adults with cystic fibrosis.
The Cystic Fibrosis Foundation
The mission of the Cystic Fibrosis Foundation, a nonprofit donor-supported organization, is to assure the development of the means to cure and control CF and to improve the quality of life for those with the disease.
Thanks to the dedication and financial backing of our supporters—patients, families and friends, medical professionals, researchers, volunteers, individual donors, corporations and staff—we are making a difference.
Labels:
Education
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