BETHESDA, Md. -- Today, the Cystic Fibrosis Foundation announced that it has awarded up to $2.17 million to Beyond Air® to support the development of a portable inhaled nitric oxide treatment for nontuberculous mycobacteria (NTM), difficult-to-treat bacteria that infect the lungs of people with cystic fibrosis. NTM infections are becoming increasingly prevalent among people with CF, and current standard of care involves lengthy courses of antibiotics that are associated with serious side effects.
Nitric oxide plays a key role in the body's immune system, making germs easier to kill by breaking down protective layers formed by bacteria called biofilms. People with CF have lower levels of nitric oxide in their airways, which are associated with chronic infection and reduced lung function. Researchers believe that increasing levels of nitric oxide in the body could help eliminate bacteria and increase lung function in people with CF.
“The treatment regimen for NTM infections can be grueling -- often requiring aggressive antibiotics for up to two years,” said JP Clancy, MD, vice president of clinical research at the Foundation. “Creating new and better therapies that can be better tolerated and can reduce the side effect and treatment burden is particularly important for people with CF battling these difficult-to-treat bacteria.”
The funding will support a clinical trial to determine whether high doses of inhaled nitric oxide are safe and effective when used in conjunction with current standard-of-care therapies, including amikacin and azithromycin. The 12-week pilot study is being conducted in Australia and is expected to enroll 20 total CF and non-CF patients with one of two different NTM strains -- Mycobacterium avium complex or Mycobacterium abscessus complex.
Participants in the study will receive nitric oxide from a generator that produces the gas from ambient air and will be used at home. In other therapeutic settings, nitric oxide is administered via expensive and cumbersome cylinders that restrict its use to hospitals.
The CF Foundation's Commitment to Infection Research
People with CF who have chronic infections are at greater risk for worsening lung disease, and infection remains a top concern of both patients and clinicians. Many individuals also suffer severe side effects from long-term antibiotic use, such as hearing loss, and are at increased risk of developing antibiotic-resistant infections.
In 2018, the CF Foundation dedicated at least $100 million to its Infection Research Initiative as part of a sweeping effort to advance research into bacterial, viral, and fungal infections. The Infection Research Initiative is a comprehensive approach to improve outcomes associated with infections through enhanced detection, diagnosis, prevention, and treatment.
The Foundation already has committed more than $79 million to the initiative, and it will continue to invest in any science that it believes holds real promise to address infections. Currently, the Foundation is funding more than 15 industry programs to develop treatments for CF-related infections.
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit cff.org.