2013 Breath of Life Gala
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.
Did you know?
- CFF receives no federal funding. 100% from donors.
- CFF uses donated funds efficiently. About 90 cents of each dollar raised goes directly to research.
- CFF is innovative and pioneered the field of venture philanthropy. They partner with small biotechs to provide some startup money, bonuses for research milestones, and then share in the revenue stream if treatments make it to market. Fantastic approach.
- CFF “stewards” FDA to be sure that new therapies get Federal approval in record time. The new treatment Kalydeco was approved in around 18 months, which is phenomenal. They do this by designing studies the right way, presenting only clear results, and helping FDA staff consultants for review.
- Some new CF treatments are showing promise for other diseases, especially COPD, and potentially even Alzheimer’s.