A nitric oxide therapy that AIT Therapeutics‘ is developing to treat respiratory diseases eradicated a drug-resistant bacteria in one cystic fibrosis patient and reduced it in another, according to a Phase 2 clinical trial.
The patients received the treatment under the Food and Drug Administration’s compassionate use program, which allows people to receive an experimental therapy in extraordinary circumstances. The two people in the AIT trial received nitric oxide for infections of Mycobacterium abscessus complex, or MABSC, after failing to respond to other treatments.
In addition to being effective, the study showed that the therapy was safe and that patients tolerated it well.
The study, published in the Pediatric Infectious Disease Journal, was titled “Compassionate Nitric Oxide Adjuvant Treatment of Persistent Mycobacterium Infection in Cystic Fibrosis Patients.”
MABSC, a group of rapidly growing non-tuberculous mycobacteria, is one of the most antibiotic-resistant pathogens found in CF patients.
In the Phase 2 trial, researchers gave the patients intermittent administrations of nitric oxide. NO is able to counter a lot of bacteria, and is safe and well tolerated, previous studies had shown.
The main objective of the trial was to see if AIT’s nitric oxide formulation could eliminate mycobacteria in CF patients. It eradicated Mycobacterium abscessus in one patient and greatly reduced the bacteria count in the other.
“We were pleased to see such dramatic results in these two patients with Mycobacterium abscessus complex,” Steve Lisi, chief executive officer of AIT Therapeutics, said in a press release.
“This condition is very difficult to treat and these patients had exhausted all other treatment options. The encouraging results demonstrate our proprietary Nitric Oxide therapy could be a potential treatment for these patients.” Lisi added.
Researchers said additional trials need to be conducted to confirm the results, however.
NO plays a key role in a range of biological functions. When it is at specific concentrations in airways, it appears to bolster immune system functioning.
Lab studies have suggested that NO can eliminate bacteria, fungi, yeast and parasites. Scientists believe it could also help neutralize the multi-drug-resistant strains of bacteria that have become a global public health concern in the past few decades.
The trial results prompted the team to conclude that “intermittent inhalations with 160 ppm NO are well tolerated, safe and result in significant reduction of Mycobacterium abscessus load. It may constitute an adjuvant therapeutic approach for CF patients with Mycobacterium abscessus lung disease.”