Bronchiectasis is a chronic lung condition characterised by abnormal and permanent widening of the bronchi (the airways that lead to the lungs), often resulting in recurrent infections and respiratory symptoms such as cough, sputum production, and shortness of breath. Although there is no cure for bronchiectasis, there are several new (emerging) treatments that will likely help manage the symptoms and improve the quality of life for people with this condition. Particularly, the emergence of antibiotic-resistant bacteria, including Pseudomonas aeruginosa, has made it increasingly difficult to effectively treat bacterial infections in patients with bronchiectasis. As a result, there has been ongoing research focused on developing new antibiotics and other treatments to target these infections and the inflammation they cause.
Brensocatib
Brensocatib is a new anti-inflammatory treatment that is being studied for its potential use in bronchiectasis. It is a selective oral inhibitor of dipeptidyl peptidase 1 (DPP1), an enzyme that is involved in neutrophil activation and inflammation.
Brensocatib works by blocking the activity of DPP1, which reduces the activation of neutrophils and the release of inflammatory cytokines. Neutrophils are a type of white blood cell that plays a key role in the immune response to infection. However, excessive activation of neutrophils can lead to inflammation and tissue damage, which is seen in many chronic respiratory diseases, including bronchiectasis.
Clinical trials have shown that brensocatib reduces the frequency of exacerbations in patients with bronchiectasis. In a phase 2 trial, patients who received brensocatib had a 60% reduction in the rate of exacerbations compared to placebo. Brensocatib was also well-tolerated, with no serious adverse events reported.
Currently, brensocatib is undergoing phase 3 clinical trials to further evaluate its safety and effectiveness in patients with bronchiectasis. If the results of these trials are positive, brensocatib could become a new treatment option for patients with bronchiectasis.
Arikayce
Arikayce is a liposomal formulation of the antibiotic amikacin that is approved for the treatment of nontuberculous mycobacterial (NTM) lung infections in patients with limited treatment options. NTM are a group of bacteria commonly found in the environment that can cause chronic lung infections in individuals with underlying lung conditions, such as bronchiectasis and cystic fibrosis.
Arikayce works by delivering amikacin directly to the lungs in a liposomal formulation, which allows for sustained release of the drug and enhanced penetration into the lung tissue. The liposomal formulation also helps to reduce systemic exposure and minimize the risk of side effects associated with systemic antibiotic therapy.
Clinical trials have demonstrated the efficacy of Arikayce in the treatment of NTM lung infections. In a randomized, double-blind, placebo-controlled phase 3 trial, Arikayce was shown to significantly improve sputum culture conversion rates and reduce exacerbations in patients with refractory NTM lung infections compared to placebo.
Arikayce is administered via a nebulizer once daily, which allows for targeted delivery of the drug to the lungs. However, there are some potential risks associated with the use of Arikayce, including the risk of respiratory adverse events, such as coughing and shortness of breath.
In addition, Arikayce is expensive and requires close monitoring of patients due to the risk of nephrotoxicity and ototoxicity associated with amikacin use. Therefore, it is typically reserved for patients with refractory NTM lung infections who have not responded to other treatment options.
Despite these limitations, Arikayce offers a promising new treatment option for patients with limited options for the treatment of NTM lung infections. Ongoing research is focused on identifying patient populations who may benefit most from this therapy and optimizing treatment protocols to minimize the risks associated with its use.
Phage therapy
Phage therapy is an experimental treatment that involves the use of bacteriophages, which are viruses that infect and kill bacteria. The therapy has shown promise as a potential treatment for bacterial infections in various medical conditions and may be beneficial in bronchiectasis.
Phage therapy offers a potential alternative to antibiotics by targeting specific bacterial strains without affecting the surrounding healthy bacteria. The treatment involves isolating bacteriophages that can effectively infect and kill the specific bacteria causing the infection. These phages are then administered to the patient to eliminate the bacteria and clear the infection.
In a small pilot study, patients with bronchiectasis who received phage therapy had a significant reduction in bacterial load and improved lung function compared to those who received standard antibiotic therapy. Phage therapy has also been effective in treating chronic Pseudomonas aeruginosa infections in patients with cystic fibrosis.
However, more research is needed to determine the safety and effectiveness of phage therapy in bronchiectasis. One of the challenges of phage therapy is the need to identify the specific phages that can effectively target the bacteria causing the infection, as well as the potential for bacterial resistance to develop over time.
Therefore, phage therapy is an experimental treatment that offers a potential alternative to antibiotics for the treatment of bacterial infections in patients with bronchiectasis. More research is needed to determine its safety and effectiveness in large patient populations. If studies show that phage therapy is a safe and effective treatment for bronchiectasis, it could offer a new approach to managing multi-drug resistant bacterial infections in these patients.
Lefamullin
Lefamullin is a novel antibiotic that is currently being developed. It is specifically designed to target the bacteria that commonly infect the lungs of people with bronchiectasis, including Pseudomonas aeruginosa and Staphylococcus aureus.
In a phase 2 clinical trial, Lefamullin demonstrated promising results in treating patients with bronchiectasis who were infected with Pseudomonas aeruginosa. The study found that Lefamullin was effective in reducing the bacterial load in the lungs of patients, as well as improving lung function and reducing the frequency of exacerbations.
Lefamullin works by inhibiting bacterial protein synthesis, which is necessary for the bacteria to grow and replicate. By targeting this specific process, Lefamullin is able to kill the bacteria without harming the patient’s healthy cells.
One of the key advantages of Lefamullin is its ability to penetrate the mucus layer in the lungs that often impedes other antibiotics. This allows Lefamullin to reach the bacteria directly and exert its antimicrobial effect.
Another advantage of Lefamullin is its favorable safety profile. In the phase 2 clinical trial, Lefamullin was well-tolerated by patients, with no serious adverse events reported.
While Lefamullin is still undergoing clinical trials, it shows promising potential as a new treatment option for patients with bronchiectasis. If approved, Lefamullin could provide an effective and safe treatment option for those with this chronic lung condition.