Alex's Lemonade Stand Foundation for Childhood Cancer

Background
Immunotherapy with genetically modified T cells has the potential to improve outcomes for children with cancer. We have recently developed a new class of antigen-specific T cells, Engager T cells, that not only recognize tumor cells, but also redirect bystander T cells to tumors. This was achieved by genetically modifying T cells to secrete bispecific T-cell engagers. However, cancer patients also have a large reservoir of NK cells that could be harnessed to destroy malignant cells.

Background

Using the patient's own immune system to fight cancers is a promising approach to improve outcomes for pediatric cancer patients who do not benefit from current therapies. However, the body's immune defenses against cancers often fail because the cancers do not induce or actively inhibit immunity. Cancer treatments consisting of the infusion of T cells (one component of the patient's own immune system) that recognize CD19, a molecule present on many blood cancers, have shown promise in early clinical studies.

Our aim is to develop potent immune-based therapies for high-risk neuroblastoma that produce complete tumor responses without toxicity and without compromising quality of life.

We have already demonstrated that T-cells genetically modified to recognize the GD2 protein expressed on neuroblastoma cells can safely eliminate local relapsed neuroblastoma, but complete responses of bulky or metastatic tumors were not obtained, likely because tumor-specific T-cells travel poorly to tumor sites and because neuroblastomas are immunosuppressive.