Engager T Cells: A Novel Immunotherapeutic for AML
Background
Pediatric acute myeloid leukemia (AML) is a type of blood cancer that is in need of new therapies, as children suffering from high risk disease have little chance of cure. Aggressive treatment with traditional chemotherapy drugs can lead to complications and severe toxicities. Targeted therapy may have fewer side effects, and immune therapies in particular are a new class of treatments that offers great promise. Infusion of a type of immune cell, the T cell, modified to specifically recognize a cancer cell has been successful against pediatric acute lymphoblastic leukemia (ALL) in early clinical studies.
Project Goal
We have developed a new type of modified T cell, genetically engineered to secrete a targeting engager molecule that directs these cells, as well as unmodified T cells, to attack AML cells. Preliminary studies have shown strong activity against AML in a model that imitates human disease. Because this is an original, innovative therapy that we plan to translate into the clinic, we propose the incorporation of a 'safety-switch' that will allow rapid destruction of AML-specific T cells if unexpected toxicity is seen. We plan to test these T cells in our preclinical models that closely mimic human disease. If this modified approach to AML therapy is successful, and further clinical development is justified, our center has the infrastructure in place to develop such a clinical trial for children with AML.
