Center for Pediatric T Cell Therapy (CEPET)
Many children with malignant disease can be cured by conventional therapy, but only at the cost of severe and sometimes fatal treatment-related toxicity and with a significant risk of disease recurrence. We propose building an infrastructure that will allow us to extend the application of a highly targeted and evidently much less toxic biological therapy, which modifies the patient's own immune system to attack the tumor. With the aid of this infrastructure grant we will be able to build on our previous and published successes with this approach, and offer treatments for neuroblastoma, osteosarcoma, medulloblastoma and high grade glioblastoma, which are among the commonest pediatric solid tumors.
We will use tumor antigen-directed T cells that have demonstrated exquisite specificity and minimal to no toxicity in clinical trials. These T cells have already proved highly effective for the treatment of lymphoma, nasopharyngeal cancer (NPC) and melanoma, producing complete and sustained remissions in up to 55% of individuals whose disease had recurred after all other treatment options were exhausted. Because we have now developed technologies that enable us to extend this approach to the commonest solid tumors of children, we will be able to make a significant contribution to the treatment of pediatric cancer. Our initial impact would be in children with relapsed or resistant disease, but subsequently -- once safety and efficacy are confirmed - we believe these therapies could make a significant contribution as front line therapies, providing an effective treatment that offers a high subsequent quality of life.