Development of new therapies for neuroblastoma

Division Of Basic Sciences - Nci

Investigators

Linked publications, trials & patents

Abstract

Update on goal 1. Harnessing tumor-targeting antibodies and NK cell activity in pediatric solid tumors: We developed next-generation antibody cytokine fusion proteins and will assess their therapeutic efficacy in preclinical neuroblastoma models. The aim of these studies is to translate these novel antibody cytokine fusion proteins to an investigator-initiated clinical trial for GD2-expressing malignancies. Update on goal 2. Optimizing cell-based immunotherapy for pediatric neuroblastoma: We have demonstrated preclinical activity of GPC2-targeting chimeric antigen receptor (CAR) T cell therapy in preclinical neuroblastoma models. This work will be translated into an investigator-initiated first-in-human clinical trial for patients with neuroblastoma in NCI's Pediatric Oncology Branch. To further optimize CAR T cell therapy, our laboratory focuses on CAR engineering to manipulate the CAR structure, conditionally alter CAR expression, and exploit the use of transgenic cytokines. We use genome-wide CRISPR screens to understand the mechanisms of tumor response to and evasion from adoptive CAR T therapy. Altogether, these efforts are geared towards finding new adoptive therapies to treat patients with neuroblastoma.