Alvelestat for Bronchiolitis Obliterans Syndrome

This trial tests the safety and effectiveness of a new drug, alvelestat (MPH966), for individuals with bronchiolitis obliterans syndrome (BOS), a lung condition that can occur after a stem cell transplant. Researchers aim to determine if alvelestat can improve lung function by reducing the activity of neutrophil elastase, which may contribute to BOS. The trial seeks adults who have undergone a stem cell transplant and now have BOS along with chronic graft versus host disease (cGVHD), which can cause breathing difficulties in daily life. Participants will take the drug in varying doses to identify the most effective one and monitor changes in their lung health over time. As a Phase 1, Phase 2 trial, this research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to contribute to groundbreaking advancements in BOS treatment.

The trial does not specify that you must stop taking all current medications, but if you are on azithromycin, you need to stop it at least 2 weeks before joining. If you are on systemic therapy for cGVHD, you must be on stable or tapering doses for the previous 4 weeks.

Research shows that alvelestat (MPH966) is being tested for safety in people with bronchiolitis obliterans syndrome (BOS). Earlier studies found that alvelestat, a pill, may help with BOS symptoms by reducing the activity of a substance called neutrophil elastase.

These studies examined how well people handle the drug and what side effects it might cause. So far, results suggest that alvelestat is generally well-tolerated by patients. The main goal has been to find the best dose that works effectively while minimizing side effects.

Researchers are excited about Alvelestat for bronchiolitis obliterans syndrome because it offers a novel approach by targeting neutrophil elastase, an enzyme linked to lung damage. Current treatments like corticosteroids and immunosuppressants mainly aim to reduce inflammation and suppress the immune system, but they don't specifically address the enzyme activity contributing to the disease progression. Alvelestat's ability to directly inhibit neutrophil elastase represents a potential breakthrough by tackling the underlying cause of tissue damage, promising more effective management of the condition.

Research has shown that alvelestat (MPH966), the investigational treatment in this trial, might help treat bronchiolitis obliterans syndrome (BOS) by blocking an enzyme called neutrophil elastase, which may be involved in this condition. Earlier studies found that alvelestat is generally safe and could help stabilize the disease in people with advanced BOS. Specifically, improvements appeared in certain biomarkers, indicating how the body responds to the treatment. While more research is needed, these early results suggest that alvelestat might improve lung function in people with BOS.¹²⁶⁷⁸