Milestones in Cystic Fibrosis Medicine

The 20th century saw a great deal of progress in the field of medicine with the advent of advancements such as antibiotics, vaccines, organ transplants, and genetic research. Advancements in medical knowledge affected the prognosis of cystic fibrosis quite notably over the past 60 years. Consider that in the 1930s, most patients with CF died in infancy; thirty years ago, the life expectancy rose to twenty years; and today, the median life expectancy is 37 years. As CF patients live longer and longer, the medical advancements have been coming more and more rapidly. Here is a brief overview of some of the most important milestones in research of cystic fibrosis treatments:

• 1938: Dorothy Andersen first recognizes cystic fibrosis as a specific disease in her paper “Cystic fibrosis of the pancreas and its relation to celiac disease”
• 1950’s: first CF clinics are founded to treat patients; sweat tests are developed as a diagnostic tool
• 1955: Shwachman describes the method that became the foundation for modern treatments: early diagnosis, active early treatment of chest infections, and attention to nutrition. Additionally, first use of pancreatin began to aid digestion of fats, and the US National CF Research Foundation (later known a the Cystic Fibrosis Foundation) was founded
• 1960’s: chest physiotherapy began to be regularly used with CF patients
• 1972: Nutritionists realized that low fat diets were detrimental to CF patients and focused on consumption of additional calories
• 1976: Danish medical centers begin using intravenous antibiotics to treat P. Aeruginosa
• 1980: Hopfer and colleagues present a paper at the Toronto International CF Congress suggesting that abnormal epithelial electrolyte transport may play a critical role in the disease
• 1983: Pancrease, the first acid-resistant microspheres for digestive enzymes, was first introduced, greatly improving the quality of life for pancreatic-insufficient patients
• 1985: First use of nebulized colomycin
• 1989: Tsui, Collins, and Riordan identified the CF gene and called it the cystic fibrosis conductance regulator (CFTR). The most common mutation DF508 was also identified.
• 1990: First double lung transplant was performed
• 1992: Introduction of high lipase enzymes, greatly reducing the number of capsules patients need to take with each meal
• 1994: First drug specifically designed for CF, Pulmozyme, was approved by the FDA
• 1996: Inhalation of hypertonic saline is found to improve sputum clearance and improve pulmonary function
• 1999: Flutter device is developed as an aid for airway clearance that patients can use in their homes without assistance
• 2000: Scientists supported by the Cystic Fibrosis Foundation map the entire genetic structure of the most common cause of CF lung infections-the Pseudomonas aeruginosa bacterium
• 2003: Scientists at Structural GenomiX, Inc. determine the 3-dimensional structure of a portion of the CFTR protein opening the door to more drug discovery opportunities
• 2006: The first compound for CF, discovered through high-throughput screening at Vertex Pharmaceuticals, begins clinical trials
• 2007: VX-770, a potentiator candidate developed by Vertex Pharmaceuticals to restore function of the CFTR protein for patients with the G551D mutation, entered Phase 2 testing
• 2008: Vertex Pharmaceuticals announces promising results for the VX-770 drug, and announces studies of an additional drug VX-809 which may restore CFTR function for patients with the DF508 mutation

As you can see, CF medicine has come a long way in the past half-century, and the progress has been steadily picking up momentum over the past twenty years. Many additional medications and treatments are being researched thanks to the dedication of foundations such as the CFF. The scientific progress is inspiring for anyone in the medical field, and especially offers hope to CF patients and their families. We are excited to see what else is in store.

For further reading, you can find a very thorough history of CF article here. Additional information on historical research milestones is available through the CFF website here.