Cystic fibrosis patients got their ‘miracle drug’ thanks to federal research funding – new Trump administration rules may cut future miracles short
Prior to effective treatments, it was difficult for children with cystic fibrosis to live full lives. Chris Carlson/AP Photo The hardest disease to care for when I was training as a pediatrician in the late 1970s was cystic fibrosis , a disease in which abnormally thick mucus secretions block the lungs’ airways and make it progressively harder to breathe.
In the late 1970s, cystic fibrosis was a devastating disease with no cure. It caused thick mucus to block the lungs, making breathing increasingly difficult over time. Patients often suffered from chronic pneumonia, antibiotic-resistant infections, and complications like diabetes, malnutrition, and heart failure. Treatments were limited to physical therapy to clear mucus, drugs to open airways, and high-dose antibiotics. The median age of death for children with cystic fibrosis in 1979 was just 15 years, making it a heartbreaking condition that robbed young patients of adulthood. The disease was progressive and inevitably fatal, leaving families and doctors with few options to slow its progression.
A major shift occurred in 2012 when the drug Kalydeco (ivacaftor) was approved by the U.S. Food and Drug Administration (FDA). This was followed by other breakthrough drugs: Orkambi (2015), Symdeko (2018), Trikafta (2019), and Alyftrek (2024). These drugs, called cystic fibrosis transmembrane conductance regulator (CFTR) modulators, target the root cause of the disease by correcting the defective protein produced by the CFTR gene. As a result, the median survival age for children born today with cystic fibrosis has risen to 66 years. For example, Mary Vought, whose daughter has cystic fibrosis, stated that these drugs have given her daughter the chance to live a full life.
The development of cystic fibrosis drugs was made possible by decades of research funded by both public and private sources. The National Institutes of Health (NIH), a U.S. government agency, played a critical role by funding over 6,200 research papers on cystic fibrosis biology between 1985 and 2012. This research helped identify the CFTR gene and understand how it causes the disease. The NIH provided $3.2 billion for basic research and $940 million for applied research during this period. The Cystic Fibrosis Foundation, a nonprofit organization, also contributed $200 million to drug discovery and development, while the biotech company Vertex Pharmaceuticals invested undisclosed amounts to create and commercialize these drugs.
Despite the success of cystic fibrosis drugs, federal funding for research is now at risk due to policies proposed by the Office of Management and Budget (OMB), led by Russell Vought. These policies aim to increase political control over federal research grants by limiting peer review—a process where experts evaluate research proposals—and restricting topics eligible for funding. While Congress temporarily blocked these changes, several federal agencies, including the NIH, are still pursuing parts of this agenda. Such restrictions could slow down or complicate future medical breakthroughs by reducing funding, training opportunities, and global research collaboration.
Developing a new drug typically requires 20 to 30 years of basic lab research followed by 8 to 9 years of clinical testing. On average, each drug costs $1.4 billion in NIH funding and up to $1.4 billion in industry funding. This long-term commitment from scientists, advocates, legislators, and companies is essential for advancing treatments. The current threats to federal research funding could disrupt this process, making it harder to find cures for diseases like cystic fibrosis, cardiovascular disease, cancer, and dementia. The Cystic Fibrosis Foundation has emphasized the importance of a fully functional NIH to continue advancing treatments for the disease.
