We've been talking about how genetic engineering is now become a part of cancer treatment as well ; here we'll discuss how 1st gene therapy application was done on cancer patients!
In 2019, the University of Pennsylvania initiated the first trial in the United States to evaluate a groundbreaking CRISPR-made cancer therapy. This study, partially funded by NCI, aims to assess a form of immunotherapy that involves genetically modifying patients' immune cells to enhance their ability to identify and destroy cancer cells.
The therapy process entails implementing four genetic modifications to T cells, which are capable of targeting and eliminating cancer. Initially, a synthetic gene is introduced to equip the T cells with a receptor that recognizes NY-ESO-1, a molecule present in certain cancer cells.
Subsequently, CRISPR technology is utilized to eliminate three genes: two genes that may impede the function of the NY-ESO-1 receptor and another gene that restricts the cells' cancer-fighting capabilities. The resulting product, termed NYCE T cells, is cultivated in significant quantities and then administered to patients through infusion.
The trial's leader, Dr. Edward Stadtmauer, and his colleagues conducted a study to assess whether removing three genes with CRISPR would enhance the effectiveness of NY-ESO-1–directed T cells.
The CRISPR-made treatment was tested in two patients with advanced multiple myeloma and one with metastatic sarcoma, and initial findings indicate that the treatment is safe. Although some side effects occurred, they were likely due to the chemotherapy the patients received before the infusion of NYCE cells.
There was no evidence of an immune reaction to the CRISPR-edited cells.
The T cell therapy resulted in only about 10% of the T cells having all four of the intended genetic edits. Additionally, off-target edits were identified in the modified cells of all three patients. However, it's worth noting that none of the cells with off-target edits exhibited any signs of developing into cancer, as observed by Dr. Stadtmauer.
While the treatment had a limited impact on the patients’ cancers, it did temporarily halt the progression of tumors in two patients (one with multiple myeloma and one with sarcoma),
although the tumors resumed growing at a later stage. Regrettably, the treatment was not effective for the third patient. It's really promising that the initial treatment showed positive results for the sarcoma patient because it has been historically challenging to effectively treat solid tumors with cellular therapy.
Dr. Stadtmauer mentioned that CRISPR techniques may potentially improve our ability to treat solid tumors with cell therapies.
However, it's important to note that while the trial demonstrates the possibility of CRISPR-edited cell therapy, the long-term effects still require careful monitoring. According to Dr. Stadtmauer, the NYCE cells have appeared safe during the study period, but continual monitoring for years, possibly even decades, is planned to ensure long-term safety and efficacy.
While the study of NYCE T cells marked the first trial of a CRISPR-based cancer treatment, there are likely more to come
“This [trial] was really a proof-of-principle, feasibility, and safety thing that now opens up the whole world of CRISPR editing and other techniques of [gene] editing to hopefully make the next generation of therapies,” Dr. Stadtmauer said.
The landscape of CRISPR-made cancer treatments is rapidly evolving with exciting new developments. Ongoing trials are exploring CRISPR-engineered CAR T-cell therapies for B-cell cancers and multiple myeloma. It's an incredibly dynamic field, with researchers continuously enhancing CRISPR methods and exploring its potential for broader applications in the future. The pace of progress is truly exhilarating!
So that was all about gene therapy application on cancer 🙌🏻😄 hope you guys liked it let me know in comments
wow!