Treatment options are limited for children and adolescents presenting with advanced cancers and many die from progressive disease. Chemotherapy is often ineffective for solid tumors and brain tumors that recur or progress after standard treatment. Although surgery or radiation therapy may prolong life, they are not always curative. Alternative treatment approaches such as immunotherapy, including T cells genetically modified to express a tumor targeting molecule called "chimeric antigen receptor" or "CAR", have been effective in curing some patients with B-cell leukemia and lymphoma. However, a subset of these children with high-risk disease experience relapse after CAR T-cell therapy due to...

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Treatment options are limited for children and adolescents presenting with advanced cancers and many die from progressive disease. Chemotherapy is often ineffective for solid tumors and brain tumors that recur or progress after standard treatment. Although surgery or radiation therapy may prolong life, they are not always curative. Alternative treatment approaches such as immunotherapy, including T cells genetically modified to express a tumor targeting molecule called "chimeric antigen receptor" or "CAR", have been effective in curing some patients with B-cell leukemia and lymphoma. However, a subset of these children with high-risk disease experience relapse after CAR T-cell therapy due to reasons inherent to the cancer itself or from limitations of the treatment method. The potential of CAR T cells and other immunotherapies in treating advanced solid tumors and brain tumors in children is yet to be explored optimally. The primary intent of our clinical research program is to develop effective immunotherapy approaches for these high-risk childhood cancers, with the focus on sarcomas, brain tumors, and therapy resistant B-cell leukemia. We have the resources and a structured research plan to accomplish our goals through: (1) conducting investigator-initiated clinical trials of CAR T cells to assess their safety and efficacy, (2) evaluating evidence-based combination immunotherapies with CAR T cells in well designed clinical trials, (3) improving children's access to experimental treatments, (4) contributing to scientific advancements by thorough examination of research samples from treated patients, (5) establishing sound scientific collaborations, and (6) preparing the next-generation of young clinicians to continue the research endeavors. We believe that this well-rounded approach will enable us to make a difference to children with cancer in the long term.

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