Gene Therapy Shown to Fight Leukemia; T-Cells Taken Out, Tweaked and Reinserted

New advanced genetically engineered cell therapies could benefit people suffering from blood disorders including leukemia and genetic conditions.

More than half of patients newly diagnosed with these types of blood condition don't respond to treatment of experience a relapse, an American Society of Hematology news release reported. These factors can negatively affect the patients' prognosis.

This new approach, called "precision medicine," aims to elevate treatment success. The method employs the use of the patient's own re-engineered cells to target the disease.

The method uses the patient's immune system to transform healthy cells into "super cells" that can better battle the disease.

In chimeric antigen receptor (CAR) cell engineering naturally occurring immune cells, known as T-cells, are harvested from the patients' blood and enhanced with two new features: "a receptor on the outer cell surface that recognizes a protein called CD19 present on most leukemic cells and a powerful mechanism inside the cell that triggers it to expand and proliferate once attached to the targeted protein," the news release reported. Once enhanced, the T-cells are injected back into the patient's blood stream, where they are expected to battle existing cancer cells.

Both adult and pediatric leukemia patients were found to respond well to the innovative treatment. About two-thirds of patients benefited from the treatment when other options had failed.

Another benefit is that because the cells originated in the patient's own body, they are less likely to attack healthy tissue than cells taken from elsewhere.

"With the right technology and laboratory expertise, the process of cell engineering is feasible for many patients. One remaining challenge is determining why some patients benefit and others have less durable responses. Does 'one size fits all' therapy work or do we need personalized or individualized T cell treatments? Further, we need to extend these studies to other tumor types, particularly solid tumors, to evaluate their potential in other clinical settings," Laurence Cooper, MD, of The University of Texas MD Anderson Cancer Center in Houston, said.

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