Aridis Pharmaceuticals has released the data from the first phase of its clinical trial of AR-501. This treatment is inhaled to clear lung infections that are commonly experienced by those with cystic fibrosis. Researchers are excited by the data so far, as it demonstrates that AR-501 was safe and helped to clear the airways. If this treatment becomes available to CF patients, they will have a safe and easy therapy that they will be able to administer themselves.
About Cystic Fibrosis
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 affects one in every 2,500 to 3,500 Caucasian babies, and it is even rarer in other ethnicities. 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, and exercise intolerance.
Diagnosis often comes at birth, as screening for cystic fibrosis is standard across the United States. In order to complete a screening, doctors must perform blood sampling, genetic testing, and sweat tests. After a diagnosis is obtained, doctors select the correct treatment. The goal is to control infections, remove mucus from the lungs, prevent intestinal blockage, and provide proper nutrition. Methods include antibiotics, anti-inflammatory medications, medicine to induce the coughing up of mucus, bronchodilators, pancreatic enzymes, exercise therapy, chest physical therapy, vest therapy, lung transplants, and surgery to remove bowel blockages, remove nasal polyps, and place a feeding tube.
AR-501 is a self-administered, inhaled form of gallium citrate. It is an antimicrobial that intends to interfere with iron and disrupt pathways, but does so in a way that is distinct from past CF treatments. In fact, it has been shown to be effective against antibiotic resistant strains of bacteria.
About the Study
This trial was randomized, double-blind, and placebo controlled in the effort to test the pharmocokinetics and safety of AR-501. 48 healthy people and 48 people with cystic fibrosis participated in this trial, coming from 18 sites across the United States. The healthy participants were separated into six cohorts and given one ascending dose or multiple ascending doses.
Key findings include:
- No serious adverse events were reported
- Adverse events that were reported were mild, balanced between those who received the placebo and those who received AR-501, and resolved
- AR-501 was well-tolerated
- All doses have been cleared to move to the next portion of the trial
- Changes in airflow were found through spirometry and were not dose related
These results are very exciting for researchers and cystic fibrosis patients, as AR-501 would be a convenient and effective treatment.