AIT’s application for the treatment of lung diseases associated with Cystic Fibrosis (CF).
About Cystic Fibrosis
The Cystic Fibrosis Foundation estimates that approximately 70,000 people globally suffer from CF, with nearly half (approximately 30,000) in the United States. There are about 1,000 new cases per year in the United States.
The management of CF includes a variety of medications, physiotherapy and a high calorie, gh protein diet. Antibiotic to control lung infections is given by either oral or inhaled, or intra-venous during acute exacerbations. Lung transplantation is an option in severe cases.
Treatment continues throughout the patient’s life. Annual estimated cost of patient care ranges from $45,000 to $50,000. Repeated episodes of infection result in progressive lung injury, respiratory failure and ultimately death.
If NOxCF minimizes the complications of CF, it stands to have a major impact on the length and quality of life of CF patients.
Cystic Fibrosis (CF) is a genetic disorder that causes mucus to build up in the lungs, digestive tract and other areas of the body. CF affects mainly the lungs, but other systems are also involved. The most serious breathing problem results from frequent lung infections. NO has been found to be effective against several microbial and viral strains involved in severe lung pathologies, including those associated with CF.
Our NOxCF application is based on proprietary technology that utilizes inhalations of Nitric Oxide (NO) for several consecutive days on a monthly basis. NOxCF was successful in a Phase IIa clinical study. We plan to conduct a phase II clinical trial for NOxCF exacerbation as well as NOxCF NTM (Non-Tuberculous Mycobacterium) in the near future.
Limitations of Current Treatment Options for CF-Related Lung Infections
Although current therapies for CF-related lung infections have led to improvement in lung function, chronic bacterial infections associated with CF necessitate multiple courses of antibiotic therapy, which lead to antibiotic resistance. It is clear from recent developments that resistance develops faster than new antibiotics can be produced, evaluated and processed through regulatory approvals.
In addition, there is a lack of medical treatment for viral respiratory infections associated with CF (such as RSV), as antibiotic therapies are ineffective in the treatment of viruses.
AIT’s novel NO products meet the urgent need for better treatment strategies to combat both bacterial and viral infections.
Cystic Fibrosis causes thick, sticky mucus to build up in the lungs, digestive tract, and other areas of the body. The symptoms and the severity of the symptoms vary; some children will have symptoms at birth, while others may not have symptoms for weeks, months, or even years. The severity of symptoms also vary; while some children show only mild digestive and lung problems, others have severe food-absorption problems and life-threatening breathing complications.
Some of the most common symptoms are salty-tasting skin, frequent coughing, wheezing, bouts of pneumonia or sinusitis, difficulty in breathing that keeps getting worse, and a big appetite but poor weight gain.
Over time, the symptoms of Cystic Fibrosis can worsen and may include: Pancreatitis (a painful inflammation of the pancreas), Liver disease, Diabetes and Gallstones.
Cystic Fibrosis is caused by a defect in the gene that produces a protein called CFTR (cystic fibrosis conductance transmembrane regulator). The CFTR protein controls the flow of salt and water in and out of the cells in organs such as the lungs and pancreas.
To have Cystic Fibrosis, a person must inherit two copies of the defective Cystic Fibrosis gene — one copy from each parent.
People with only one copy of the defective Cystic Fibrosis gene are called carriers, but they do not have the disease themselves. Each time two Cystic Fibrosis carriers have a child, the chances are 25% (1 out of 4) for carrying a child with Cystic Fibrosis.
In a healthy person, the Cystic Fibrosis gene produces the protein CFTR (Cystic Fibrosis conductance Transmembrane Regulator), which is found in the cells that line different organs, such as the lungs and pancreas. The CFTR protein controls the movement of electrically charged particles, including chloride and sodium (components of salt), in and out of these cells.
In people with Cystic Fibrosis the CFTR gene is defective. As a result, the produced CFTR protein is flawed, and the salt balance in the body is disturbed. Because there is too little salt and water outside of the cells, the thin layer of mucus that helps keep the lungs free of bacteria becomes very thick and difficult to cough up. This thick mucus then clogs the airways and, without treatment, can lead to infection and inflammation that ultimately damage the lungs.
A genetic test can determine if someone carries a faulty CFTR gene, the gene responsible for Cystic Fibrosis. This can be done before or during pregnancy for either of the future parents.
During pregnancy, there are two tests that can determine if an unborn baby has Cystic Fibrosis:
- Chorionic villus sampling (CVS) tests a small piece of placenta early in pregnancy.
- Amniocentesis tests a small amount of the fluid around the baby in the uterus.
In both tests, genetic material from the fetus is collected and screened for the Cystic Fibrosis gene.
After birth Cystic Fibrosis is usually diagnosed by conducting a sweat test, which measures the amount of salts in a person’s sweat. For newborns, an immune-reactive trypsinogen test (IRT) may be used instead.
If the results of the sweat test or the IRT test are unclear, a genetic test is often used to confirm a diagnosis of Cystic Fibrosis.