CF Foundation Awards Over $2 Million for Cystic Fibrosis Treatment Development

The Cystic Fibrosis Foundation is dedicated to finding a cure for the rare, genetic disorder. They fund more CF research than any other organization, and their latest effort is a portable treatment for nontuberculous mycobacteria (NTM). They will grant up to $2.17 million to Beyond Air for the therapy.

About Cystic Fibrosis (CF)

Cystic fibrosis is a genetic disorder that is characterized by progressive damage to the respiratory and digestive systems. Those with cystic fibrosis do not have the slippery mucus that is normally found in the lungs, instead they have thick and sticky mucus which builds up in their system. This buildup causes clogs in the airways, which then traps bacteria and causes breathing problems, infections, lung damage, and respiratory failure. It can also block digestive enzymes, which makes it difficult to absorb nutrients. Cystic fibrosis is a recessive disorder, meaning that the mutated gene must be passed down by both parents. The gene responsible for this condition affects the protein that regulates salt movement. The mutation in the gene varies in severity as well.

Symptoms of cystic fibrosis affect the respiratory and digestive systems. They include:

  • Persistent coughing and wheezing
  • Shortness of breath
  • Difficulty exercising
  • Frequent lung infections
  • Stuffy noses
  • Trouble with gaining weight
  • Constipation
  • Male infertility
  • Salty-tasting skin
  • Exercise intolerance

About the Research

Through the use of the money awarded by the CF Foundation, Beyond Air will develop their portable inhaled nitric oxide therapy, which will be used to treat NTM in CF patients. This bacterial infection is typically difficult to treat, and what treatment is administered leads to side effects. Because of this, there must be new therapies made available.

In order to create a viable therapy, Beyond Air plans to launch a clinical trial to evaluate the safety and efficacy of a high dosage when taken with the current standard of care. The study will run for 12 weeks, with 20 participants enrolling in Australia. These patients will receive the therapy at home through a generator that creates air from ambient air.

Why It’s Important

Cystic fibrosis patients, along with their physicians, have to remain vigilant when it comes to infections, as they can contribute to disease progression. To handle these infections, patients are often put on antibiotics for extended periods of times, sometimes for multiple years. This treatment regimen can lead to adverse effects, and it also puts patients at an increased risk of antibiotic-resistant infections.

Hopefully, Beyond Air’s new therapy is proven to be a viable treatment for nontuberculous mycobacteria in cystic fibrosis patients.

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