The fight against respiratory diseases has just received a significant boost. Scientists have developed a groundbreaking new drug delivery system that targets genetic therapies directly to the lungs. This innovative approach has opened promising possibilities for patients suffering from conditions like lung cancer and cystic fibrosis, offering a glimmer of hope for those in need.

Led by Gaurav Sahay from Oregon State University’s College of Pharmacy, this research was conducted in collaboration with Oregon Health & Science University and the University of Helsinki. The findings were published in two separate papers: one in Nature Communications and another in the Journal of the American Chemical Society.

Through an extensive study involving over 150 different materials, researchers discovered a novel type of nanoparticle that can safely and effectively carry messenger RNA and gene-editing tools to lung cells. In experiments with mice, this treatment demonstrated remarkable results – slowing down the growth of lung cancer and improving lung function that had been compromised by cystic fibrosis.

Cystic fibrosis is a genetic condition caused by one faulty gene, which severely limits lung function in patients. The new drug delivery system has shown great promise in addressing this issue, with researchers developing a chemical strategy to build a broad library of lung-targeting lipids used in the nanocarriers. These materials form the foundation for the innovative treatment and can be customized to reach different organs in the body.

“The streamlined synthesis method makes it easier to design future therapies for a wide range of diseases,” Sahay explained. “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.”

The studies were funded by esteemed organizations such as the Cystic Fibrosis Foundation, the National Cancer Institute, and the National Heart, Lung and Blood Institute. This groundbreaking research has brought hope for patients with respiratory diseases, and its long-term implications could revolutionize the treatment of various conditions.

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

The diabetes pill dapagliflozin has shown promising results in reducing liver scarring in a clinical trial published in The BMJ. The study found that treatment with dapagliflozin improved metabolic dysfunction-associated steatohepatitis (MASH), a condition where excess fat accumulates in the liver, leading to inflammation, and liver fibrosis, a build-up of scar tissue.

The trial involved 154 adults diagnosed with MASH after a liver biopsy at six medical centers in China. Almost half had type 2 diabetes, and almost all had liver fibrosis. The participants were randomly assigned to receive either 10 mg of dapagliflozin or a matching placebo once daily for 48 weeks.

The results showed that treatment with dapagliflozin improved MASH in 53% of participants without worsening of fibrosis, compared to 30% in the placebo group. Resolution of MASH without worsening of fibrosis occurred in 23% of participants in the dapagliflozin group, compared to 8% in the placebo group.

Fibrosis improvement without worsening of MASH was also reported in 45% of participants in the dapagliflozin group, compared to 20% in the placebo group. The percentage of participants who discontinued treatment due to adverse events was 1% in the dapagliflozin group and 3% in the placebo group.

The researchers acknowledged that the trial was conducted in a Chinese population, which limits its broader generalizability, and that female and older patients were under-represented. However, they pointed out that results were consistent after further analyses, suggesting they are robust.

The study’s findings indicate that dapagliflozin may affect key aspects of MASH by improving both steatohepatitis and fibrosis. Large-scale and long-term trials are needed to further confirm these effects.

In the coming years, researchers expect exciting developments in the field of pharmacological treatment for MASH, with more drugs becoming available and therapeutic decisions becoming increasingly tailored to individual patient profiles. Ideally, such treatments should provide cardiovascular benefit, have an established safety profile, and be accessible to broad and diverse patient populations.

The production of human proteins has long been a costly and labor-intensive process, often relying on mammalian cells. However, a team led by Dr. Markus Napirei at Ruhr University Bochum has successfully produced the human protein DNase1 using yeast cells, a breakthrough that could revolutionize the field.

DNase1 is an enzyme used to treat cystic fibrosis and other conditions, but its production in mammalian cells has been limited by high costs and effort. The new method uses Pichia pastoris, a type of yeast fungus, to produce the protein, which can be stably integrated into the yeast genome and released as desired.

“This is the result of years of work, and could lay the groundwork for the manufacture of human DNase1 in yeast as a biological agent,” says Dr. Napirei. The research was published in PLOS ONE on April 29, 2025.

The advantages of using yeast cells over mammalian cells are clear: cost-effective culture conditions, high reproduction rates without the need to immortalize cells, and lower susceptibility to pathogens. In his doctoral thesis, Jan-Ole Krischek successfully expressed human DNase1 in Pichia pastoris, cleaned it, and characterized it for the first time.

One of the surprising findings was that the yeast produced considerably less human DNase1 than the mouse DNase1 used as a guide, despite sharing 82 percent of their primary structure. This is partly due to specific folding behaviors of the two proteins, explains Dr. Napirei.

DNase1 has been used for over 60 years to treat various conditions, including cystic fibrosis. The enzyme degrades cell-free DNA that can induce symptoms of illness. Inhaled DNase1 liquifies DNA-laden bronchial mucus, making it easier to cough up. Its potential use in other pathological processes is vast, particularly in the removal of neutrophil extracellular traps (NETs) and microthrombi that contain high levels of NET components.

Dr. Napirei suggests that DNase1 could be used to better dissolve microthrombi containing DNA, an application currently being explored in clinical studies. Another potential use is in dissolving thrombosis of a cerebral artery in the case of ischemic strokes.

This breakthrough has significant implications for the production and use of human proteins, particularly DNase1. The ability to produce this enzyme using yeast cells could lead to more cost-effective and efficient treatment options for patients, ultimately improving their quality of life.