Alex's Lemonade Stand Foundation for Childhood Cancer

Diffuse midline glioma (DMG) is a universally fatal childhood brain malignancy with no sufficient treatment options. With brain cancer emerging as the leading cause of cancer-related death in children and young adults, new treatments are desperately needed. Our research program has developed a new treatment called RNA nanoparticle vaccines (RNA-NPs) to treat DMG and other cancers. RNA-NPs are a personalized immunotherapy treatment that activates the body’s own immune system to recognize and kill cancer cells.

Mentor Name: Elias Sayour

Harnessing the immune system for the treatment of cancer through PD-1 checkpoint inhibitor has shown considerable promise in a number of solid tumors. However, outcomes remain poor for brain tumor patients. Since brain tumors are the number one cause of cancer-related deaths in children, finding ways to overcome treatment resistance in this population is paramount.

Background

Despite aggressive multimodality treatment, pediatric brain tumors are the leading cause of cancer related deaths in children. The development of efficacious therapies against these tumors is of paramount importance. Immunotherapeutic approaches against solid tumors can be curative with a demonstrably high degree of specificity, and adoptive T cell therapy using tumor infiltrating lymphocytes has proven to be the most efficacious platform against metastatic disease.

Human hepatoblastoma (HB) is the most common pediatric liver cancer in the U.S.A. More than 30 percent of patients die after a 10-year period. In addition, the existing therapies are very costly. For example, liver transplantation is estimated to cost approximately $150,000 to $200,000 per patient. Moreover, the shortage of donor livers for patients awaiting transplant and graft rejection post-transplantation significantly limit the widespread usage of this method. Thus, alternative therapies are still warranted.