Development of novel cell and gene therapies

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The cell processing laboratory is developing new natural killer (NK) cell, dendritic cell (DC), bone marrow stromal cell (BMSC) and chimeric antigen receptor (CAR) T cell therapies. NK cells are an important part of the innate immune system and they are thought play an important in host defense against foreign and abnormal cells including cancer and hematological malignancies. Preliminary work suggests that hematological malignancies can be treated by the administration of NK cells. The Cell Processing Laboratory has been expanding autologous and allogeneic NK cells to treat Clinical Center patients with cancer and hematological malignancies. Dendritic cells (DCs) are an important adjuvant for cancer vaccines. The Cell Processing Laboratory is currently is producing DCs expressing HER2/neu to treat cancer patients whose tumor express this antigen and is developing a new protocol to treat patients with stage 0 prostate cancer Bone marrow stromal cells (BMSCs) can inhibit in function and support tissue regeneration Preliminary studies suggest that acute graft versus host disease and autoimmune diseases can be treated with BMSCs. In addition, BMSCs secrete cytokines and growth factor that improve the healing of damaged tissue and BMSCs have used to treat patients with ischemic heart disease and peripheral vascular disease. The Cell Processing Laboratory has developed a bank of BMSCs products collected from healthy subjects that is being used to treat hematopoietic stem transplant patients with acute graft versus host disease or graft failure and inflammatory bowel disease. The lab has also developed a process to produce autologous BMSCs to treat patients with ischemic heart disease. The cell processing laboratory has developed CAR T cells expressing anti-CD19. These cells are now being used to treat pediatric patents with B cell leukemias. The laboratory has also developed and is manufacturing anti-GD2 CAR T cells to treat patients with sarcoma, anti-B Cell Maturation Antigen (BCMA) to treat patients with multiple myeloma and anti-CD22 CAR T cells to treat patient with CD19-negative B cell leukemia. The lab is working on manufacturing engineered T cells which express a high affinity T cell receptor specific for papilloma virus. The laboratory completed the development of a protocol to treat and prevent infections to CMV, EBV, adenovirus and BK virus using multiple virus specific T cells. A protocol to treat patients with progressive multifocal leukoencephalopathy (PML) using BK-specific viral T cells is also being developed.