Statins Show Promise as a Treatment for Severe Lung Disease

Researchers Explore Statins as a Treatment for Pulmonary Alveolar Proteinosis

Understanding Pulmonary Alveolar Proteinosis

Pulmonary alveolar proteinosis (PAP) is a rare lung disease affecting approximately seven individuals per ten million, as reported by the PAP Foundation. The condition primarily occurs in adults aged 30 to 50, with men being more susceptible than women.

Causes and Symptoms of PAP

PAP arises when surfactant, a substance that helps keep the air sacs in the lungs open, accumulates and obstructs these sacs. This blockage hinders air from entering the lungs and impairs oxygen transfer into the bloodstream, leading to progressive breathlessness. The condition is mainly attributed to a decrease in factors necessary for surfactant clearance and maturation within the lungs. Common symptoms include shortness of breath, persistent cough, fatigue, fever, and weight loss.

Current Treatment Options

The primary treatment for PAP is whole lung lavage, a procedure performed under general anesthesia. During this treatment, excess surfactant is flushed out of the lungs using saline. This procedure must be repeated whenever lung function deteriorates, often resulting in significant discomfort for patients.

Statins as a Novel Therapeutic Approach

A recent study published in Nature Communications by researchers at Cincinnati Children’s Hospital Medical Center in Ohio has investigated the use of statins to treat PAP. Statins, commonly used to lower cholesterol, were found to reduce cholesterol buildup in the air sacs and improve the condition of patients with PAP.

The researchers conducted a trial with a 58-year-old woman suffering from severe PAP, who had not responded well to traditional lung washing methods. Following six months of statin treatment, the patient reported significant improvements in her breathing difficulties and was able to discontinue the use of her oxygen concentrator. Remarkably, after three and a half years of statin therapy, the surfactant accumulation associated with PAP was completely resolved, eliminating her need for whole lung lavage.

Implications for Future Research

This promising study paves the way for future research into statin therapy for PAP, potentially leading to clinical trials involving larger patient populations. The findings suggest a new direction for the treatment of this challenging lung disease.

Reference

McCarthy C. et al. Statin as a novel pharmacotherapy of pulmonary alveolar proteinosis. Nature Communications. 2018;9:3127. DOI 10.1038/s41467-018-05491-z.