For me, as for many physician-scientists, the story began with a patient. As a third-year medical student at the University of Iowa, I entered a clinic room and saw a young girl coughing and struggling to breathe. Her prognosis was dismal, and the lack of effective treatments was disheartening. That girl remains deeply embedded in my memory.

Decades earlier, Dorothy Andersen, a pathologist at Babies Hospital at Columbia-Presbyterian, identified fluid-filled cysts surrounded by scars in the pancreas in autopsies of children originally thought to have celiac disease. In 1938, she named the disease “cystic fibrosis of the pancreas” (1). She and others rapidly recognized the severe bronchiectasis and autosomal recessive inheritance that are associated with CF.

A breakthrough came after the New York heat wave of 1948, when children with CF were brought to the hospital with severe dehydration and heat prostration. Paul di Sant’Agnese discovered that people with CF lose excessive salt into sweat, which led to a diagnostic test — the sweat Cl– test — that became the cornerstone of CF diagnosis (2).

Increased diagnostic accuracy and recognition of the disease further revealed that CF affects multiple organs. In addition to pancreas and lung destruction, it causes liver disease, meconium ileus, male sterility, and CF-related diabetes. However, it is the lung disease, with recalcitrant bacterial infections, profuse inflammation, and copious mucus production, that causes most of the morbidity and mortality. In the 1960s, few children survived beyond their teenage years.

Progress for people with CF came when physicians, scientists, and caregivers began newborn screening programs and established CF clinical centers and a CFF Therapeutics Development Network. In addition, they developed better therapies, including pancreatic enzyme replacement, nutritional support, chest percussion, aggressive antibiotic therapy (including inhaled antibiotics), inhaled DNase, inhaled hypertonic saline, and lung transplantation. By the end of the 1980s median survival had increased to approximately 18 years.