What Are the New Possibilities for Using CAR-T Cell Therapies

How Patients’ Immune Cells Fight

The essence of current CAR-T therapies lies in using a patient’s own immune cells to precisely target carcinogenic cells. T-lymphocytes are collected from the patient via leukapheresis and are then genetically modified. A viral vector is used to insert genetic information that enables the synthesis of a chimeric antigen receptor (CAR). Thanks to this artificially introduced receptor, the immune system cells gain the ability to specifically bind to a suitable target molecule, such as a protein typically found on the surface of pathologically altered cells.

The currently used therapies target either the CD19 marker or the BCMA marker (B-cell maturation antigen). Theoretically, however, any suitable marker unique to the target cells can be used. T-lymphocytes modified ex vivo are then multiplied and reintroduced into the patient’s body, where they can help effectively eliminate target cells, such as cancer cells carrying the recognized antigen.

Expanding Therapy to Other Diseases

CAR-T cell therapies have attracted the attention of the scientific community due to their effectiveness, precise targeting of pathologically altered cells, and ability to provide long-term protection. It is therefore not surprising that many research teams have begun to explore the use of this highly effective immunotherapy for the treatment of other diseases.

First and foremost, there is the potential to treat not only hematological malignancies but also solid tumors. At the same time, research is being conducted into its use in the treatment of autoimmune diseases (such as systemic lupus erythematosus, myasthenia gravis, or multiple sclerosis), fibrotic disorders, and infectious diseases.

Although scientific teams face many challenges when treating solid tumors with CAR-T therapies—such as cellular heterogeneity within the tumor mass, the tumor microenvironment suppressing immune response, and limited T-cell infiltration—clinical trials already indicate great potential for this approach.

Partial successes have been reported, for example, in the treatment of neuroblastoma, glioblastoma, and certain types of gastrointestinal cancers. Research efforts are therefore focusing on the development of CAR-T therapies more resistant to tumor microenvironment influences, new administration methods, or therapies in which T-cells are engineered to recognize two or even three target antigens.

Attention is also being paid to mechanisms ensuring the safety of these therapies and minimizing adverse effects. For example, CAR-T cells with controllable activity are being researched.

Hope for Thousands of Patients

From the above, it is clear that the future of CAR-T cell therapy holds great promise—not only as standalone treatment but also in combination with other treatment modalities such as chemotherapy, radiotherapy, or immunotherapy, which can further enhance overall efficacy.

However, it will be crucial to address the manufacturing and economic challenges associated with this type of therapy. While new innovative solutions continue to emerge, a major obstacle remains the complex and time-consuming manufacturing process.

It is also important to consider that these therapies are no longer being developed for a handful of individuals, as was the case with past gene therapies, but represent potential treatment options for thousands of patients.

Editorial Team, Medscope.pro

Sources:

1. Zhu C., Wang F., Cheng Z., Zhang L., Li H. Precise CAR-T cell therapy targeting non-cancerous diseases: Advances in precision medicine and bioengineering. Precision Medicine and Engineering 2025; 2(1): 100024, doi: 10.1016/j.preme.2025.100024.

2. Uslu U., June C. H. Beyond the blood: expanding CAR T cell therapy to solid tumors. Nat Biotechnol 2025; 43(6): 506–515, doi: 10.1038/s41587-024-02446-2.