The Antibacterial Resistance Leadership Group (ARLG), funded by the National Institute of Allergy and Infectious Diseases (NIAID), has launched an early-stage clinical trial to assess the use of a potential bacteriophage (or “phage”) therapy in adults with cystic fibrosis (CF).

The trial, announced by the National Institutes of Health, will determine whether the investigational treatment is safe and able to reduce the amount of Pseudomonas aeruginosa in the participants’ lungs.

“The prevalence of antibiotic resistance is concerning, and the need for more effective therapeutics for vulnerable populations, such as people with cystic fibrosis, is especially urgent,” NIAID director Anthony S. Fauci, MD, said. “Although research on bacteriophage therapy may still be in its infancy in the United States, we hope that this study, and others like it, could open the doors to a new type of therapy for difficult-to-treat bacterial infections.”

The experimental phage therapeutic, known as WRAIR-PAM-CF1, is a cocktail composed of 4 bacteriophage viruses that naturally infect and kill P aeruginosa by taking over its cellular processes. The viruses included are specific to the bacterium and do not attack human cells.

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P aeruginosa is a serious and potentially deadly bacterium often acquired in healthcare settings, and it is a common cause of exacerbations in CF. Multidrug-resistant strains are on the rise, and only a few antibiotics are available to treat these infections.

The clinical trial aims to enroll up to 72 adults with CF who harbor P aeruginosa in their lungs from 16 specialized centers around the United States. They will receive the phage cocktail via an intravenous infusion at one of 3 dosages. The researchers will collect data on the safety and activity of the phages in terms of killing bacteria and their impact on lung function and quality of life.

The trial will start as a phase 1b trial and, if no serious adverse events occur, additional participants will be recruited for a phase 2 trial in which they will receive either the optimal dose of the cocktail as determined in phase 1 or a placebo.

Reference

NIH-supported clinical trial of phage therapy for cystic fibrosis begins. News release. National Institutes of Health; October 4, 2022.