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Evolutionary inspired therapy for newly diagnosed, metastatic, Fusion Positive Rhabdomyosarcoma (Phase I): A clinical trial that offers patients the option of choosing between conventional chemotherapy or one of 3 evolutionary based treatment models. The first two therapy models, first strike and second strike, both of which have the goal of making the cancer extinct. The third option, adaptive therapy, has a goal of containing the cancer versus eliminating it. By containing the cancer, the goal is for long term control of the cancer at an acceptable size while maintaining a high quality of life.
Adoptive Cellular Therapy following Dose-Intensified Temozolomide in Newly-diagnosed Pediatric High-grade Gliomas (Phase I): High Grade Gliomas are a childhood brain tumor with no standard care. We are bringing Duane Mitchell’s world class research to patients by taking their tumors and immune system out and training the immune system to fight their tumor in his lab. Then the immune system is infused back into the patient after radiation and chemotherapy.
A Phase Ib/II Study to Evaluate the Safety, Feasibility and Efficacy of Nivolumab or Nivolumab in Combination with Azacitidine in Patients with Recurrent, Resectable Osteosarcoma: Relapsed osteosarcoma has a dismal prognosis. The main predictors of survival after osteosarcoma recurrence include the time to first recurrence, disease burden, and ability to achieve complete surgical remission after recurrence. Immunotherapy is a potentially promising option for the treatment of osteosarcoma. An even more promising strategy is to enhance the effectiveness of immunotherapy treatment by combing immunotherapy with Nivolumab with Azacitidine, a cytotoxic agent that prevents cells from reproducing or growing.
Phase I Trial of the LSD1 inhibitor sp-2577 in patients with relapsed or refractory Ewing sarcoma: Ewing sarcoma is an aggressive form of cancer that typically presents as a bone tumor in children and young adults. Approximately 20-30% of patients will be diagnosed with a metastatic form of the disease that unfortunately does not respond to systematic chemotherapy. LSD1 is an protein found in tumor cells and plays an important role in tumor cell growth and survival. Researchers with the Sunshine Project are testing the Seclidemstat (SP-2577), is an oral LSD1 inhibitor designed to block the activity of LSD1, which may in turn slow or stop the growth of tumor cells.
Blood based biomarkers for minimal residual disease detection in pediatric sarcomas: A cutting edge project that will collect blood samples from patients diagnosed with sarcoma, to test whether the detection of cell-free plasma tumor DNA (ptDNA) in the patients blood can better predict recurrence of disease when a patient is in radiographic remission. Having a blood based test that can detect minimal residual disease in sarcomas can make an immediate impact as well as lasting future impact in how we treat sarcomas.
Phase II Study of nab-Paclitaxel in Combination with Gemcitabine for Treatment of Recurrent/Refractory Sarcoma in Teenagers and Young Adults: This trial will look at this combination of drugs and their ability to prevent the formation or growth of tumors in teenagers and young adults with relapsed or refractory osteosarcoma, Ewing sarcoma, rhabdomyosarcoma and other soft tissue sarcoma.
National Pediatric Cancer Foundation (NPCF) is a nonprofit organization dedicated to funding research to eliminate childhood cancer.
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