A Leader in Lung Development and Disease Research

Researchers in our Division of Pulmonary Biology—one of the largest programs of its kind in the U.S.—have profoundly impacted modern lung biology, pulmonary medicine and neonatal care.

Our scientists study the molecular and cellular processes that regulate lung development and investigate the causes of common and rare lung diseases. Not only have our findings led to new diagnostic tools—such as clinical lab tests for autoimmune pulmonary alveolar proteinosis (PAP) and kaposiform vascular anomalies—but we’ve also paved the way for lifesaving and life-changing surfactant, gene, and cell therapies.

As we continue building upon previous discoveries (and making new ones), we aim to further reduce perinatal deaths caused by lung dysfunction and to identify more therapeutic targets for lung disease in children and adults.

Collaboration Leads to Innovation

In the Division of Pulmonary Biology, research doesn't take place in a vacuum. Our investigators work with many other individuals and groups from Cincinnati Children’s and the University of Cincinnati (UC) School of Medicine. These include:

• Clinicians and researchers from the Division of Neonatology (within our nationally recognized Perinatal Institute) and the Division of Pulmonary Medicine—ranked No. 1 in the nation by U.S. News & World Report (2024-2025)
• Clinicians and researchers from the UC Division of Pulmonary, Critical Care and Sleep Medicine
• Experts from the Center for Stem Cell & Organoid Medicine (CuSTOM), the Division of Biomedical Informatics, and graduate programs specializing in Development, Stem Cells & Regenerative Medicine, Immunology, and more

Our scientists also team up with organizations outside Cincinnati Children’s. For instance, we frequently lead studies through federally funded research networks such as the National Institutes of Health (NIH) Progenitor Cell Translational Consortium, the Rare Lung Diseases Consortium, and all three phases of the LungMAP consortium. Additionally, some of our faculty hold leadership positions in international health organizations such as the LAM Foundation, Cystic Fibrosis Foundation, PAP Foundation, Alpha-1 Foundation, and TBX4Life.

These internal and external partnerships help us accelerate the translational research process. Together, we’re able to move our scientific discoveries out of the lab and into the hands of physicians and their patients.

A History of Groundbreaking Research

Over the last several decades, our investigators have made many important breakthroughs that directly impact patient outcomes. For example:

• Some of our researchers discovered surfactant B and C genes and proteins in the early 1980s. They used their findings to help develop lifesaving surfactant therapy for infants with respiratory distress syndrome and to identify some of the genetic causes of lung diseases in infants and children.
• An NIH-funded study co-led by members of our team resulted in Food and Drug Administration (FDA) approval of the drug Sirolimus to treat lymphangioleiomyomatosis, or LAM disease.
• Our scientists were the first to identify hereditary PAP as a new genetic disease caused by mutations in CSF2RA and CSF2RB. Building upon these findings, we developed a type of lung gene/cell therapy called pulmonary macrophage transplantation (PMT). In 2023, we launched the first-in-human PMT clinical trial. In parallel, we have led phase 1, phase 2, and phase 3 clinical trials of inhaled GM-CSF.

Leveraging New Technology

Because Cincinnati Children’s is home to experts in bioinformatics, single-cell genomics, artificial intelligence, and other advanced disciplines, our researchers have begun taking a genome-wide approach to decipher the molecular networks underlying lung biology. With faculty specializing in nearly every lung cell lineage—including epithelial, endothelial, mesenchymal, and immune cells—we’re also utilizing a multi-lineage approach.

This shift is essential because the functions of nearly 50 different lung cell types aren’t compartmentalized. Physical and chemical signaling, along with other cellular activities, are interconnected. This means lung development and disease onset are likely products of system-level processes rather than single-gene (or single-cell) resilience or deterioration.

To that end, we’re combining artificial intelligence and mechanistic experimentation to better understand the entire genome and its influence on all pertinent lung cell types.

Pulmonary Research by the Numbers