PROJECT SUMMARY

Myelodysplastic syndrome (MDS) is a form of blood cancer where cells in the bone marrow are damaged and no longer function properly. Without normal growth of bone marrow cells, blood is not produced and patients can become weakened or even die. In addition, left untreated, MDS can often progress to a serious form of blood cancer known as acute myeloid leukemia (AML). The focus of our work is to harness the power of the human immune system to fight MDS. To achieve this goal, we have developed a new method that allows us to isolate immune cells (known as “T cells”) from an MDS patient, and to engineer those cells to more effectively target and destroy MDS cells. In the past year, we have demonstrated that T cell engineered to recognize and destroy MDS cells can be successfully produced and are highly effective in laboratory tests. In the coming year, we will continue to refine this method with the goal of beginning human clinical trials when this grant project is completed. Ideally, if we can eradicate MDS cells using a patient’s own T cells, we can restore normal blood production and reduce the risk of progression to AML.