CAR T-Cell Therapy Targets Cells That Cause Leukemia Relapse
Genetically engineered immune cells successfully target the specific cancer cells that may be responsible for relapse of acute myeloid leukemia (AML), a type of blood cancer, and proved effective in animal models of the disease, according to a preclinical study by investigators at Weill Cornell Medicine.
In the study, published April 28 in Nature Communications, the researchers used an approach in which immune cells known as T cells are directed to produce proteins called chimeric antigen receptors, or CARs, that enable the T cells to recognize specific markers on cancer cells. In this case, the CAR is a receptor that binds to the CD123 molecule on leukemia stem cells, enabling the T cells to seek out and attack the cancer cells.
The CAR T cells—called UCART123 cells—used in this study have several very important features,” said Dr. Guzman. “They target a leukemia stem cell marker, they are derived from healthy donors and manufactured to be ‘off the shelf’ and ready-to-go for patients when needed, they are specially designed to try to minimize toxicity, and they can be eliminated using a drug called rituximab in case of excessive proliferation.”
When the team tested the UCART123 cells in a mouse model of AML, they found that the therapy effectively eliminated leukemia cells and prolonged survival. Finally, they demonstrated that UCART123 cells have specificity against leukemia cells, with minimal toxicity to normal blood cells in mice.
The results of the preclinical study suggest that UCART123 cells are highly selective and specific in targeting AML.
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