First human trial of CRISPR-edited T cells for personalized cancer therapy

First human trial of CRISPR-edited T cells for personalized cancer therapy

Scientists have always hoped to harness the power of the body’s immune system to treat cancer.

T-cell surface receptors, a key part of the immune system involved in recognizing and responding to specific antigens, can detect cancer cells because a single mutation in the genome of a cancer cell alters the cell surface protein.

Isolating such cancer-detecting T-cell receptors and using them to generate therapeutic T cells may open up a new avenue for treating refractory cancers.

A recent study published in Nature reports the development and human clinical trials of the first non-viral engineered modified immune cells for cancer treatment.

Using the CRISPR-Cas9 genome editing system, researchers inserted cancer-specific T-cell receptors into T cells from cancer patients to generate personalized cancer-fighting immune cells.

In a phase I clinical trial, 16 patients with metastatic solid tumors (mostly colorectal cancer) that had failed to respond to standard therapies were treated with genetically engineered T cells that expressed personalized T cell receptors targeting individual cancer mutations.

Of 16 subjects, the therapy resulted in stable disease in 5 and progressive disease in the other 11 patients.

Only two patients experienced adverse reactions resulting from T-cell therapy, and all patients experienced the expected adverse reactions associated with concurrent chemotherapy.

Although the clinical benefit regarding patient response was limited, this study demonstrates the potential feasibility of this treatment strategy.

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