CF Foundation Tops $100M in Funds to Improve Infections Treatment
Note: This story has been updated to clarify that the Cystic Fibrosis Foundation has funded more than $100 million in various studies through its Infection Research Initiative, not that it has raised funding for future research.
The Cystic Fibrosis Foundation (CFF) has funded more than $100 million in various studies to date through its Infection Research Initiative, designed to improve the detection and treatment of chronic infections in cystic fibrosis (CF).
The funding, now exceeding $109 million, was awarded in little more than three years — two years ahead of the project’s initial five-year goal.
“Reaching this goal doesn’t mean we have reached the finish line,” Tiffany Burnett, co-lead of the Infection Research Initiative and senior director of biopharma programs at the Cystic Fibrosis Foundation, said in a press release.
“We will maintain this momentum and continue to fund any science that we believe holds real promise to address infections,” Burnett said, adding, “Chronic infections are a serious threat to the health of people with CF, and we continue to need improved detection and more effective treatments.”
Most CF patients develop complications from infections that put them at risk of worsening lung disease. A type of antibiotic-resistant bacterial strain, called methicillin-resistant Staphylococcus aureus (MRSA), is of special concern as persistent infections are linked with rapid lung decline and lower survival among patients.
Launched in 2018, the Infection Research Initiative marked the CFF’s continued efforts to aid the development of new and non-traditional ways to treat antibiotic-resistant infections, as well as to improve detection and diagnosis. Additionally, the initiative is working to enhance current therapies.
More than 20 industry programs — all focused on developing new treatments — have so far been funded by the initiative, with grants totaling $60 million. This has fueled a 67% average annual increase since 2018 in the number of foundation-supported studies.
Six studies currently are investigating ways to treat common CF infections, including those triggered by Pseudomonas, a bacteria commonly found in the environment. Another five studies are focused on infections caused by nontuberculous mycobacteria — a group of bacteria related to the one that causes tuberculosis — and two are on MRSA infections.
New “non-traditional” strategies to address hard-to-treat bacterial infections are being tested, according to the CFF, including the use of nitric oxide (NO), IV gallium, and bacteriophage therapies.
NO is a gas that is produced naturally in the body and acts as a vasodilator — that is, it prompts blood vessels to relax and widen — but also plays a key role in the immune response against microbes.
Prior research shows NO could help eliminate disease-causing bacteria in CF and improve lung function.
Gallium, a molecule similar to iron, has been shown to kill antibiotic-resistant strains of Pseudomonas aeruginosa. With its into-the-vein (intravenous) use approved by the U.S. Food and Drug Administration for diagnostic purposes, further studies are assessing its effectiveness for treating infection in people with CF.
The CFF has funded the development of bacteriophage (phage) therapies, which have reached clinical testing. Phages are viruses that kill specific bacterial strains, and serve as an alternative to antibiotics. Most importantly, phages don’t affect beneficial bacteria or human cells.
In addition, the foundation has allocated close to $6 million to identify technologies that do not use sputum to detect when a person with CF is infected. This is particularly important for patients being treated with Vertex Pharmaceuticals’ Trikafta, known to have limited sputum production. Alternative diagnostic tests are critical to assess the effectiveness of new therapies and monitor response to treatment, according to the CF Foundation.
As part of the CFF-supported academic research, results from the STOP2 — Standardized Treatment of Pulmonary Exacerbations II — clinical trial (NCT02781610) showed that a shorter regimen of 10 days of intravenous antibiotics is not inferior to a 14-day regimen in treating pulmonary exacerbation (worsening) in early responders, or patients who responded quickly to the treatment. The same trend was seen for later responders, with 21 days of treatment showing no higher effectiveness than 14 days.
These findings support the use of shorter intravenous (IV) antibiotic regimens for people with CF, which will reduce the risk of antibiotic resistance and of side effects.
A new pilot study involving children and adolescents, called Streamlined Treatment of Pulmonary Exacerbations in Pediatrics, or STOP-PEDS (NCT04608019), is now underway. This study, which enrolled 121 participants with CF, ages 6 to 18, will assess whether antibiotic treatment added immediately with airway clearance may benefit pediatric patients.
The outcomes of this trial will help researchers determine the feasibility of a future larger study comparing the early versus later addition of antibiotics in controlling mild pulmonary exacerbations in pediatric CF.
The CF Foundation also is supporting the STOP3 program, which will investigate whether an intravenous antibiotic combo — a broad-spectrum aminoglycoside (such as gentamicin) added to a beta-lactam antibiotic (a class that includes penicillin) — works better than beta lactam alone for fighting Pseudomonas infections.
The study expects to show that the combination therapy provides no benefit, which would be important given the negative effects of aminoglycosides on hearing and kidney function.
Looking ahead, the foundation is committed to boosting research for less common infections in CF, such as those caused by the Aspergillus fungus. A large-scale study, expected to start next year, will help evaluate the true burden of fungal infections, and build the infrastructure to develop future therapies and diagnoses for this particular type of infection.
Since three main microbial agents affecting CF patients — Aspergillus, MRSA, and Pseudomonas — also affect the general population, the foundation’s efforts will likely benefit people outside the CF community.
Further, the CFF will keep supporting the PASTEUR Act, bipartisan federal legislation in the U.S. to enhance the development of new antibiotics and to promote appropriate use of existing ones.