Grant: Travel Awards - Institute Community Support

Lymphangioleiomyomatosis (LAM) is a rare lung disease that predominantly strikes women during reproductive years. In patients, aggressive LAM cells invade the lungs, causing destruction of essential tissue and leading to respiratory failure. LAM is driven by genetic mutations which alter molecular pathways and allow the LAM cells to grow and cause severe damage. Unfortunately, the current treatment regime for LAM has limited efficacy and is unable to rid patients of disease. The goal of this project is to develop an improved therapeutic option. To do so, we have simulated human lungs in the lab using novel chemical techniques. In parallel, we have established new cellular models of LAM which accurately reflect the human disease. By combining our synthetic lungs and LAM cellular models, we have been able to recapitulate the invasive nature of this lung disease. We have now harnessed this system to screen hundreds of Health Canada-approved therapeutics, looking for drugs that selectively eliminate LAM cells. From this drug screen, we have identified a subset of candidates which show promising therapeutic potential. We are now attempting to better understand how these drugs eliminate LAM cells, and importantly, if they continue to demonstrate efficacy when tested in an animal model of the disease. We will then nominate our most promising therapeutic for clinical trials. Considering all these drugs have already received Health Canada approval, we anticipate rapid clinical translation. Importantly, this works seeks to advance essential, life saving therapeutic options for LAM patients.