Grant: Banting Postdoctoral Fellowship Program

Lymphangioleiomyomatosis is a devastating form of cancer, characterized by the abnormal growth of cells resembling smooth muscle cells in the lungs of affected patients. This disease primarily affects young to middle aged women, and causes lung tissue destruction, respiratory failure and death within 10-20 years. Treatment options for this disease are very limited, largely because we have not been able to efficiently grow these tumour cells in culture to study their molecular defects. To provide effective treatment options for LAM we desperately need a cellular model that mimics the behaviour of LAM tumour cells. This will allow us to determine the defects in these cells that cause disease and, more importantly, to identify novel genes and proteins that can be targeted therapeutically to treat LAM. Thus, through my research I will generate pluripotent stem cells from tumour cells of patients who have LAM, and I will grow and manipulate these cells in culture to model the phenotype of the cells that cause their disease. This will allow us to generate LAM cells in large quantities, and to therefore perform extensive analyses to identify the underlying molecular defects that cause LAM and to identify new potential drug targets. Our ability to generate stem cells directly from patients who have LAM and to model their disease in a dish provides us with an invaluable opportunity to provide treatment options to affected individuals in a patient-specific manner. This personalized medicine approach makes this project particularly exciting and offers tremendous hope that our work will lead to highly significant advances for the treatment of LAM tumours and will greatly improve the survival rate and quality of life of affected patients.