Trial For Cystic Fibrosis Drug AZLI To Treat Chronic Burkholderia Species Infection Completed

There is some good news for cystic fibrosis patients who suffer with Chronic Burkholderia Species Infection. A Phase 3b clinical trial for Aztreonam, a therapeutic for use in CF patients with Burkholderia infections, was recently sponsored by Gilead Sciences, Inc. Gilead Sciences is a research based biopharmaceutical company engaged in CF drug discovery and development.

The Phase 3b trial was designed to test the safety of Gilead’s new drug for treating the infection in cystic fibrosis patients, as well as to see if it was effective in minimizing the infection. The study utilized a randomized, double-blind, placebo-controlled two-part trial in order to extract the best data possible for the use of continuous Aztreonam for Inhalation Solution (AZLI) in patients with CF with chronic Burkholderia species infection.  The study consisted of a 24-week randomized phase, and a 24-week open-label phase. Primary and secondary efficacy analyses were conducted for the 24-week randomized phase only.  Safety data were collected for both the randomized and open-label phases. Pulmonary function was measured with spirometry and the revised Cystic Fibrosis Questionnaire (CFQ-R) was used to evaluate the quality of life.  The CFQ-R is a validated, patient-reported outcome tool that is used to measure health-related quality of life for both children and adults with CF.

The results of the study showed that AZLI treated patients had fewer colony forming units (CFU) of Burkholderia relative to baseline and improved lung function. In addition, researchers reported no safety issues, meaning that AZLI has crossed a critical threshold in moving towards possibly becoming a viable treatment for the infection in CF patients.

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Beta-lactam antibiotics and Aztreonam

Beta-lactam antibiotics are one of the most prescribed drugs and are classified based upon a common chemical structure known as a beta-lactam ring.  Examples of beta-lactam drugs include penicillins, cephalosporins, cephamycins and carbapenems.  Aztreonam is a member of this group and works much like other beta-lactam antibiotics such as penicillin.  It inhibits mucopeptide synthesis in the bacterial cell wall which prevents peptidoglycan crosslinking.  This inhibits cell wall synthesis and eventually causes the bacterial cell to lyse and die.

Aztreonam for Inhalation Solution (AZLI) is used to treat bacterial infections such as Pseudomonas aeruginosa and Burkholderia species infections in patients with cystic fibrosis (CF).

Chronic Burkholderia infections

Individuals with CF are highly susceptible to chronic pulmonary infection due to certain Burkholderia bacteria.  In particular, Burkholderia cepacia is a problem for CF patients.  These bacteria are catalase-producing, lactose-nonfermenting, Gram-negative bacteria that are made up of 18 different species of which B. cepacia is one of them.  B. cepacia is of note because it is an important human pathogen that most often causes pneumonia particularly in immunocompromised individuals such as patients with CF.

Burkholderia is commonly found in soil and water and capable of surviving for long periods of time in moist environments.  It is easily spread from person-to-person particularly in hospitals and clinics.  For that reason, patients who are infected with Burkholderia are isolated from uninfected patients to limit spread of the bacteria as it can cause a rapid decline in lung function and result in death.

Lung infections for CF patients can be deadly. Read about how [adrotate banner=”12″] can help!