MDR1-transduced peripheral blood progenitor cells

MDR1-transduced peripheral blood progenitor cells are an experimental ex vivo gene therapy product consisting of autologous hematopoietic stem and progenitor cells (HSPCs) that have been genetically modified to express the human multidrug resistance 1 (MDR1) gene. The MDR1 gene encodes P-glycoprotein (P-gp), also known as ATP-binding cassette sub-family B member 1 (ABCB1), which functions as an ATP-dependent efflux pump. This pump actively removes a wide array of cytotoxic compounds, including taxanes, anthracyclines, and vinca alkaloids, from the intracellular environment. The primary therapeutic goal of this cell therapy is 'chemoprotection': by overexpressing P-gp in the patient's bone marrow compartment prior to autologous transplantation, the hematopoietic system is rendered resistant to the dose-limiting myelosuppressive effects of high-dose chemotherapy. This strategy was developed to allow for intensified chemotherapy regimens in patients with advanced solid tumors, potentially enhancing tumor clearance while mitigating severe hematologic toxicities.

Other names

MDR1-transduced CD34+ cellsABCB1-transduced hematopoietic progenitor cellsABCB-1-transduced hematopoietic progenitor cellsABCB 1-transduced hematopoietic progenitor cellsP-glycoprotein-modified peripheral blood stem cells