Katharina Maisel, PhD

Lymphangioleiomyomatosis (LAM) is a rare disease in which abnormal cells begin to grow out of control and eventually damage the lungs. LAM is usually diagnosed during reproductive years and often during or after pregnancy. The current treatment only stops progression of the disease and about 30% of patients with LAM do not respond to it. Therefore, new avenues for treating LAM are needed. Recent work, including our own, has shown that LAM has some similarities to cancer, including that LAM many suppress the immune response that would normally destroy any cancer-like cells. We found that using checkpoint inhibitors that have had significant success as cancer immunotherapies could improve survival in a mouse model of LAM. Based on this work, we have explored the use of another strategy for immune activation: adjuvants. These are materials often used in vaccine formulations and also explored as cancer immunotherapies, which stimulate immune cells to become activated. We have found that auxiliary CpG effectively improves survival in a mouse model of LAM. However, this survival is incomplete. We will investigate how CpG modulates the LAM immune response, and we will develop different strategies to overcome this. The findings will provide the foundation for translating this treatment to patients.