Breakthrough drug delivery system transports genetic therapies directly to the lungs

Scientists have made a key breakthrough for treating respiratory diseases by developing a new drug delivery system that transports genetic therapies directly to the lungs, opening promising possibilities for patients with conditions like lung cancer and cystic fibrosis.

The research, led by Gaurav Sahay of Oregon State University's College of Pharmacy, was conducted in collaboration with Oregon Health & Science University and the University of Helsinki. Findings were published in a pair of papers, in Nature Communications and the Journal of the American Chemical Society.

Scientists created and tested more than 150 different materials and discovered a new type of nanoparticle that can safely and effectively carry messenger RNA and gene-editing tools to lung cells. In studies with mice, the treatment slowed the growth of lung cancer and helped improve lung function that had been limited by cystic fibrosis, a condition caused by one faulty gene.

Researchers also developed a chemical strategy to build a broad library of lung-targeting lipids used in the nanocarriers. These materials form the foundation for the new drug delivery system and could be customized to reach different organs in the body, Sahay said.

The streamlined synthesis method makes it easier to design future therapies for a wide range of diseases. These results demonstrate the power of targeted delivery for genetic medicines. We were able to both activate the immune system to fight cancer and restore function in a genetic lung disease, without harmful side effects."

Gaurav Sahay, Oregon State University's College of Pharmacy

Oregon State's K. Yu Vlasova, D.K. Sahel, Namratha Turuvekere Vittala Murthy, Milan Gautam and Antony Jozic were co-authors of the Nature Communications paper, which also included scientists from OHSU and the University of Helsinki. OSU's Murthy, Jonas Renner, Milan Gautam, Emily Bodi and Antony Jozic teamed with Sahay on the other study.

"Our long-term goal is to create safer, more effective treatments by delivering the right genetic tools to the right place," said Sahay. "This is a major step in that direction."

These studies were funded by the Cystic Fibrosis Foundation, the National Cancer Institute and the National Heart, Lung and Blood Institute.