New Horizons in Translational Medicine,2015,2,2,60.
Conventional cancer therapies are limited by their toxicity and lack of specificity. To achieve targeted immunotherapy of cancer, we have chosen Wilm's Tumour antigen (WT1) as a target as it is over-expressed in most leukemia and many solid cancers. Using sophisticated WT1-TCR retroviral constructs, we have performed in vivo engraftment studies with CD34þ leukemic progenitor cells. In our model, treatment with WT1-TCR engineered patiens' T cells had cleared patients own leukemic cells. As the analysis of bone marrow indicated that control group showed evident engraftment of human leukemia cells, while the WT1-TCR treated group had none detectable. These data have provided a solid basis for a phase I/II clinical trial, demonstrating that WT1-TCR engineering of patient's T cells offers a simple and efficient way of producing tumor specific T cells to cure human leukemia.