Tue. Jun 25th, 2024

Gene Editing Cure for Cancer First Successful Human Trials Completed

By Miya J May 29, 2024

Recent advancements in CRISPR-based gene editing have shown promising results in the fight against cancer, particularly in personalized treatment approaches. One notable study demonstrated the clinical feasibility and general safety of using CRISPR-edited, PD-1-ablated, patient-derived T cells for treating late-stage lung cancer.

This study, along with two other significant clinical studies, marks an important milestone in CRISPR-based gene therapy. These trials collectively provided the first pieces of evidence supporting the safety and feasibility of CRISPR-based cell therapy in human patients, establishing protocols for CRISPR editing and off-target event tracking in clinical settings, valuable for future trials.

The first human trial specifically aimed at late-stage lung cancer patients involved evaluating the safety concerns around off-target editing by CRISPR-Cas9 in edited T cells. The results suggested that off-target editing was rare, and infusions of PD-1-edited T cells were generally well-tolerated by patients, with no grade 3 or higher adverse events reported over a two-year follow-up period.

This indicates a strong potential for the clinical application of CRISPR technology in cancer treatment, although it also highlighted several limitations, such as varying gene-editing efficiency across target genes and patients, dependency on guide RNA (gRNA) selection, and the potential long-term safety concerns of off-target editing effects​​.

Further research and development are focused on newer generations of CRISPR-based gene-editing technologies, such as base editing and prime editing. These aim to improve editing precision and safety by generating predictable gene-editing results without creating double-strand DNA breaks, thus potentially reducing the most toxic form of DNA damage in mammalian cells​​.

These findings underscore the transformative potential of CRISPR technology in developing personalized cancer treatments, yet also emphasize the need for ongoing research to refine these approaches for broader clinical application.

By Miya J

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