ATS Breathe Easy - ATS 2026: Delving into the Future of Rare Lung Disease

You're listening to the ATS Breathe Easy podcast brought to you by the American Thoracic Society.

Welcome back to ATS Now, live from ATS 2026.

I'm your host, Ugo Ezema.

We're returning to a question we opened with Dr. Tisha Wong.

Are we entering an era of disease-modifying therapy for rare lung diseases? We used autoimmune pulmonary alveolar proteinosis as the first lens.

Now we're using a second, lymphangioleomyomatosis, or LAM.

LAM is a rare cystic lung disease that almost exclusively affects women in their reproductive years, driven by TSC1 or TSC2.

These mutations cause progressive cystic destruction, and for decades, all we could offer was supportive care and transplant.

Then in 2011, the MILES trial showed serolimus stabilized lung function.

But serolimus is cytostatic.

It slows LAMB.

It doesn't cure it.

Come off it and the disease returns.