Funded by the Dick Vitale Pediatric Cancer Research Fund
Recent advances have shown that it is possible to use a patient’s own immune system to fight cancer. Osteosarcoma, the most common bone cancer in children and young adults, is one cancer type that has not responded to immune-based treatments. Most patients who relapse die when the osteosarcoma spreads to the lung, and it is critically important to design new treatments to prevent these young lives from being lost.
Dr. Ligon’s team analyzed osteosarcoma samples from human patients and found that while immune cells are present in osteosarcoma lung tumors, they are kept at the outside of the tumor because the tumor has several ways to “exclude” these immune cells. In collaboration with Dr. Sayour, Dr. Ligon’s team proposes to use a new immune-based therapy called an RNA nanoparticle vaccine, which may be able to reprogram the tumor and allow immune cells to kill the cancer.
Based on promising data from the lab and from treating small animals such as dogs with osteosarcoma, Dr. Ligon proposes a clinical trial of this treatment in human patients with osteosarcoma which has spread to the lungs. He proposes to establish that this treatment is safe and find the right dose for future clinical trials. He will also perform studies on blood and tumor samples to understand how the vaccine works against osteosarcoma. This clinical trial will study a new treatment for a cancer that currently is incurable and help us understand how this new treatment works to help design future studies.