CAR T cell therapy, often referred to as a “living drug,” has successfully treated blood cancers such as leukemia and lymphoma. The approach, which was pioneered at Memorial Sloan Kettering Cancer Center (MSK), involves equipping a person’s own immune cells (called T cells) with special receptors that can find cancer in the body and launch an immune attack against it.
To make the therapy even more precise and powerful, a clinical trial is now starting at MSK testing CAR T cells that were made using a genome-editing tool called CRISPR/Cas9.
The phase 1 clinical trial led by Dr. Park uses the CRISPR-edited CAR T cells to treat diffuse large B cell lymphoma, the most common type of non-Hodgkin lymphoma. The CAR T cells target a protein on the surface of the cancer cells called CD19.
“We gave the T cells an additional boost by inserting a molecule called 1XX in the CAR and introducing it in the TRAC location,” Dr Rivière says. “This makes the cells more effective and prolongs their potency.”
“The trial will test three different doses of CRISPR CAR T cells to see what is the lowest dose that is both effective and safe,” Dr. Park says. The trial will enroll around 20 patients and is expected to take about two years.
The interim phase 1 results were reported in December 2022 at the annual meeting of the American Society of Hematology.
“Results showed the approach to be safe and very effective, with relatively low doses of CAR T cells, which is extremely encouraging,” Dr. Park says.
https://www.mskcc.org/news/crispr-edited-car-cell-therapy-clinical-trial-lymphoma
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