Cystic fibrosis patients got their ‘miracle drug’ thanks to federal research funding – new Trump administration rules may cut future miracles short
The Trump administration exerted increased political control over NIH research funding, leading to major cuts and disruptions to lifesaving research.
Cystic fibrosis patients have been granted a "miracle drug" thanks to federal research funding, but new rules by the Trump administration could potentially cut off future breakthroughs. Prior to the advent of effective treatments, children with cystic fibrosis had a challenging life expectancy due to difficulty breathing caused by thick mucus secretions blocking their airways.
The disease had no cure, and the only available treatments consisted of physical therapy, airway dilation drugs, and high-dose antibiotics that led to antibiotic-resistant infections and other health complications.
The landscape of cystic fibrosis treatment shifted dramatically after the drug Kalydeco (ivacaftor) was introduced by Vertex Pharmaceuticals in 2012. This drug, followed by additional treatments like Orkambi, Symdeko, Trikafta, and Alyftrek, extended the median survival age for cystic fibrosis patients to 66 years. The foundation for these life-changing drugs was laid through decades of research funded by a combination of public and private investments, with the National Institutes of Health (NIH) playing a pivotal role in the early stages of discovery and understanding of cystic fibrosis genetics and biology.
However, the NIH funding that was essential for developing these drugs is now at risk due to policies proposed by the Office of Management and Budget. These policies aim to increase political control over federal research grants, limit peer review, and curtail research on certain topics. While Congress temporarily blocked these policies, some federal agencies, including the NIH, are still pursuing portions of this agenda.
The NIH had funded over 6,200 research papers on cystic fibrosis biology in the 27 years leading up to Kalydeco's approval and had invested approximately $3.2 billion in basic and $940 million in applied research for potential drugs between 1985 and 2012.
The Cystic Fibrosis Foundation also acknowledged the critical role of the NIH in their 2025 letter to Russell Vought and other Trump administration leaders, stressing the importance of ensuring the NIH's functionality for furthering cystic fibrosis research and developing new treatments. To ensure the continued development of groundbreaking drugs, a long-term, sustained commitment from the public, government, and industry is necessary.
This includes an average of $1.4 billion in NIH funding and as much as $1.4 billion in industry funding per drug. Protecting or expanding federal funding for basic research is crucial to finding cures for other life-threatening conditions, including cardiovascular disease, cancer, dementia, and more.
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