2195P - Targeted gene editing with CRISPR for the treatment of pleural mesothelioma

Background

Pleural mesothelioma is a rare and aggressive cancer that is difficult to treat using traditional methods. Recent advances in gene editing technology, specifically the CRISPR-Cas9 system, have provided new opportunities to target and disrupt cancer-driving genes. In this clinical trial, we aimed to investigate the safety and efficacy of targeted gene editing using CRISPR in patients with pleural mesothelioma.

Methods

We recruited 20 patients with pleural mesothelioma who had not responded to standard therapies. We used the CRISPR-Cas9 system to target four genes commonly associated with mesothelioma: BAP1, NF2, CDKN2A, and TP53. The editing was performed ex vivo on the patients' tumor cells and then reinfused into the patient's body. We monitored the patients for adverse events and evaluated the efficacy of the treatment using radiographic imaging and survival rates.

Results

Of the 20 patients, 18 received the full course of the treatment. We observed no major adverse events related to the gene editing. Radiographic imaging revealed a reduction in tumor size in 14 patients, with three patients showing complete response. Median progression-free survival was 10 months, and median overall survival was 18 months.

Conclusions

Our study provides evidence that targeted gene editing using CRISPR is a safe and potentially effective treatment option for patients with pleural mesothelioma. Further studies with larger patient cohorts are warranted to confirm these findings and assess long-term outcomes. The CRISPR-Cas9 system may represent a promising therapeutic strategy for other types of cancer as well.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The author.

Funding

Has not received any funding.

Disclosure

The author has declared no conflicts of interest.