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This investigational cell therapy approach, developed by Columbia University, utilizes allogeneic hematopoietic stem cells—specifically cord blood-derived stem cells—as a form of immunotherapy for high-risk neuroblastoma. The treatment protocol involves a sequential transplant strategy: patients first undergo high-dose myeloablative chemotherapy followed by autologous stem cell transplantation (autoSCT) to reduce tumor burden, followed by a reduced-intensity (non-myeloablative) allogeneic stem cell transplant (RI alloSCT). The primary therapeutic mechanism is the induction of a 'graft-versus-tumor' (GVT) effect, where the donor-derived immune system recognizes and eliminates residual neuroblastoma cells. The conditioning regimen for the allogeneic transplant typically includes busulfan and fludarabine, designed to allow donor engraftment with lower toxicity than traditional myeloablative allogeneic transplants.
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