Development of Immunostimulatory DNA as a Novel Therapy in Pediatric Acute Lymphoblastic Leukemia

Background

Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. While current therapies have resulted in significant improvements in survival, relapsed ALL does not usually respond to chemotherapy. If a child does eventually respond to more intensive chemotherapy, bone marrow transplant offers the best chance for cure, although with significant risks of severe illness or death.

Project Goal
We are investigating a therapy that stimulates the immune system, much like an infection would. Our hope is that with this treatment, the child’s body would attack his or her own leukemia cells in a similar way to how the immune system would attack a bacteria or virus. This would provide a form of treatment to children with high-risk leukemia that will avoid some of the toxic effects of traditional chemotherapy or bone marrow transplant. In addition, we hope that this vaccine-like effect would provide long-term protection from relapse for these children.

Working at the Children’s Hospital of Philadelphia, we are touched by Alex Scott and her lemonade stand on a daily basis. She lives on in the hearts of every staff member who had the privilege to care for her. Alex was a true hero, and I am incredibly grateful to have the opportunity to provide a scientific contribution to help other children fighting cancers in her honor.