A technique manufactures CAR-T cells directly in the patient

A team of U.S. researchers has developed a revolutionary method for generating CAR-T cells directly in the body , opening up new possibilities for the treatment of cancer and autoimmune diseases.

CAR-T cell therapy, which involves genetically modifying immune system cells to recognize and destroy tumor cells, has transformed the treatment of certain types of cancer. However, its current application requires extracting the cells from the patient, modifying them in specialized laboratories, and reintroducing them into the body, which makes its use expensive and complicated.

To overcome these barriers, researchers at the University of Pennsylvania Perelman School of Medicine designed a new system that uses targeted lipid nanoparticles (tLNPs) to deliver mRNA to T cells directly in the body.

Unlike DNA-based therapies, the use of mRNA reduces the risk of permanent genetic modifications, as it does not integrate into the cell genome.

One of the main challenges of this technology was to prevent the nanoparticles from being rapidly eliminated by the liver.

To address this, the scientists developed a special ionizable lipid , called L829, that allowed tLNPs to be directed toward T cells via the CD5 protein, achieving more precise delivery and lower hepatic uptake.

Tests in mice, rats, and monkeys showed that a single dose of these tLNPs can generate functional CAR T cells within a few hours, with effects lasting up to two weeks.

In leukemia models, repeated application was able to almost completely eliminate tumor cells.

Furthermore, in samples from patients with autoimmune diseases, the method was just as effective as in healthy donors, successfully eliminating pathological B lymphocytes.

Researchers led by Carl H. June, the father of CAR-T therapies, acknowledge that this breakthrough represents a paradigm shift in immunotherapy , eliminating the need for specialized laboratories and allowing for more accessible and scalable therapies. While the results are preliminary and based on preclinical models, the clinical potential is enormous.