5T4-CAR-NKT cell therapy for pediatric rhabdomyosarcoma

Pediatric rhabdomysarcoma is a deadly disease affecting children and adolescents. While current radiation and chemotherapy can provide effective long-term tumour control in ~70% of patients, they are very toxic. Patients commonly suffer from decreased cognitive capabilities, fertility problems and even susceptibility to other types of cancers later in life. And for ~30% of RMS patients, there are no good treatment options. Therefore, there is urgent need to reduce the toxicity of RMS therapies and improve their effectiveness. The Mahoney lab is dedicated to discovering and developing new ways to treat RMS by harnessing the power of the immune system to attack and kill RMS cells. One promising approach is by engineering immune cells to express a synthetic protein called a "chimeric antigen receptor", or CAR. Immune cells engineered with CARs are able to better recognize and kill cancer cells. For my postdoctoral project, I propose to test the feasibility of using CAR technology to focus the immune system toward RMS. Additionally, I propose to evaluate whether two other therapies known to generate immune responses toward cancer will enhance the ability of CAR-engineered immune cells to destroy RMS tumours. If successfully, my research aims to provide a strong justification for testing CAR technology in human patients with RMS.