CAR-T Cell Therapy: New Gene Editing Method Boosts Cancer Fight in Mice
Scientists at the University of California, San Francisco (UCSF) have developed a new gene editing method that reprograms immune cells to fight cancer directly inside the body, a process that could significantly lower the cost and complexity of CAR-T cell therapy. The breakthrough, detailed in a recent study published in the journal Nature, has demonstrated the ability to treat leukemia, myeloma, and sarcoma in mice with humanized immune systems.
Current CAR-T cell therapy, a powerful immunotherapy, requires extracting a patient’s immune cells, genetically modifying them in a laboratory to target cancer cells, and then re-infusing them back into the patient. This process is lengthy, expensive, and limited to specialized centers. The UCSF team’s new approach bypasses the need for external cell manipulation, enabling the body to generate its own CAR-T cells.
The method utilizes a system of double particles and CRISPR-Cas9 technology to integrate a long sequence of DNA into a specific location within human T cells without removing them from the body. According to UCSF, this new integration method “surpassed the standard method of random DNA integration using viruses,” representing a significant advancement not only for CAR-T therapy but also for the broader fields of cell and gene therapy.
In trials conducted on mice implanted with aggressive leukemia, a single injection of the reprogrammed cells eliminated detectable cancer in nearly all subjects within two weeks, according to reports from El Periodico. The research also suggests the potential to eliminate the need for strong preparatory chemotherapy, often a necessary but harsh component of traditional CAR-T treatment.
The “in vivo manufacturing” of T cells, as the process is being called, could also democratize access to these life-saving therapies, extending their reach beyond specialized medical centers. Researchers believe the technique could facilitate the widespread adoption of CAR-T therapy in hospitals across Europe and globally, according to reporting from Medgadget. The approach also appears to enhance the proliferative capacity of the CAR-T cells generated within the body.
The research team, led by scientists at UCSF, has not yet announced a timeline for human clinical trials. The university has not released a statement regarding potential partnerships for commercialization or further development of the technology.