Scientists at the University of California, San Francisco, have developed a groundbreaking approach to cancer treatment that enables the reprogramming of immune cells directly inside a patient’s body, eliminating the need to extract and process them in a laboratory. This innovative CAR-T cell therapy method represents a significant advancement in the treatment of blood cancers and could dramatically reduce both the cost and time required for this life-saving treatment.
According to a new study published in Nature, researchers have successfully introduced precise genetic modifications inside the body, directing T cells to produce chimeric antigen receptors without external processing. This marks the first time scientists have managed to insert a large DNA sequence into a specific location within human T cells while they remain in the body, avoiding the random integration associated with traditional viral-based methods.
How the New CAR-T Cell Therapy Works
Traditional CAR-T cell therapy has long relied on a complex and expensive process. Immune cells are extracted from patients, sent to specialized centers for genetic modification to recognize and kill cancer cells, then reinjected into the patient’s body. However, this procedure takes several weeks and costs hundreds of thousands of dollars, making it inaccessible to many patients who could benefit from the treatment.
The new technique employs a dual-particle delivery system carrying CRISPR-Cas9 gene editing tools, which function like molecular scissors capable of precisely modifying DNA. According to the researchers, one particle specifically targets T cells through the CD3 protein found on their surface, while the other carries the genetic code necessary to create CAR receptors along with instructions for integrating them into a specific location within the genome.
Promising Results in Preclinical Studies
In experiments conducted on mice with humanized immune systems, scientists successfully used this CAR-T cell therapy technology to treat multiple types of cancer, including acute lymphoblastic leukemia, multiple myeloma, and even some solid tumors. The results showed the elimination of detectable cancer cells within no more than two weeks in most animals, with notable formation of CAR-T cells inside the body.
Additionally, researchers indicate that this approach may surpass traditional methods not only in precision but also in efficiency. Cells produced inside the body appear more capable of proliferating and retaining their characteristics compared to those prepared in the laboratory, according to the study findings.
Advantages of In-Body CAR-T Cell Therapy Programming
This development could contribute to reducing the need for preparatory chemotherapy, lowering costs, and accelerating access to treatment for cancer patients worldwide. Meanwhile, the technology ensures that CAR receptors are activated only in T cells by integrating them at a specific genomic location, enhancing both safety and effectiveness.
The scientists note that cells generated through this method demonstrate superior proliferation capabilities and maintain their therapeutic properties better than conventionally manufactured CAR-T cells. In contrast to the weeks-long manufacturing process of traditional therapy, this approach could potentially deliver treatment much more rapidly.
Path to Clinical Application
Despite these promising results, the technology remains in the research and development phase and requires additional clinical trials to confirm its safety and efficacy in humans. However, the research team, in collaboration with scientific partners and a specialized company, is working to bring this technology into clinical use in the future.
The research team has not announced a specific timeline for human clinical trials, though they are actively pursuing partnerships to advance the technology toward eventual patient care. Authorities have not confirmed when the first human studies might begin, but the development represents a potentially transformative step in making cancer immunotherapy more accessible and affordable.
