Gene-Engineered Stem Cell Therapy Breakthrough: Sustained Production of Anti-Cancer T Cells in Humans
Beijing, August 5 (Science and Technology Daily) — Scientists at the University of California, Los Angeles, have made a breakthrough in the field of gene-engineered stem cell research: by modifying patients' hematopoietic stem cells, they can enable the continuous production of anti-cancer T cells in the patient's body. This clinical trial, carried out by a collaboration of multiple top scientists, has for the first time verified in humans a new therapeutic strategy — the continuous generation of tumor-targeting immune cells through a "internal factory" in stem cells. The 成果，published in the latest issue of Nature Communications, marks a major advancement in the ability of the human immune system to regenerate anti-cancer capabilities.
The research team conducted trials on patients with aggressive sarcomas that express the NY-ESO-1 antigen. This cancer-testis antigen, present in 80% of synovial sarcomas, can serve as both a safe therapeutic target and effectively distinguish tumors from normal tissues. By inserting cancer-specific receptors into the patients' own hematopoietic stem cells and then implanting them through bone marrow transplantation, these engineered stem cells can continuously generate T cells carrying antigen receptors in the body. One of the patients, after receiving the treatment, not only showed signs of tumor regression but also had detectable levels of engineered T cells in the body for several months.
This therapy solves the persistence problem of traditional immunotherapy through gene reprogramming technology. Existing T cell therapies often fail due to cell exhaustion, while stem cells, as "immortalized" "cell factories", can continuously produce fresh anti-cancer cells. Studies have shown that after the genetically modified stem cells were successfully implanted into the patient's bone marrow, their stable production system was confirmed through in vivo visualization technology.
The research team stated that this strategy of building a persistent immune response through hematopoietic stem cells can be extended to HIV infection treatment in the future, and even used to reset the immune system of patients with autoimmune diseases. Currently, the therapy requires complex processes such as stem cell collection, gene editing, and high-dose chemotherapy pretreatment, but with technological optimization, it is expected to become a new approach for preventing cancer recurrence and radical treatment.
This research, which lasted more than ten years and was promoted by more than 30 scientists, is still in the experimental stage and has not yet entered clinical application. However, it has shown extensive potential, providing a brand-new strategic direction for the treatment of solid tumors, and indicating that future cancer treatment is shifting from a single course of treatment to the construction of a persistent immune defense system.